A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

Not Applicable

Completed

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Other: Visit frequency

• Registration Number: NCT03760029
• Lead Sponsor: Pfizer

Brief Summary

This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below:

* Group 1, Ambulatory subjects aged \<6 years, approximately 100 subjects;

* Group 2, Ambulatory subjects aged \>=6 years, approximately 180 subjects;

* Group 3, Non-ambulatory subjects, approximately 50 subjects. Subjects will visit sites every 6 months. Each subject will be observed for at least 24 months. All subjects will remain enrolled until the study completion date, such that some will have data collected after Month 24. Subjects, who complete Visit 5/Month 24 at least 6 months prior to study completion, will be asked to complete an additional visit at Month 30.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-11-15
• Study First Submitted QC: 2018-11-28
• Study First Posted: 2018-11-30
• Study Start: 2019-07-24
• Primary Completion: 2023-03-21
• Study Completion: 2023-03-21
• Status Verified: 2023-04
• Last Update Submitted: 2023-04-03
• Last Update Posted: 2023-04-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 312

Inclusion Criteria

1. Chinese male patients with any age, diagnosed with DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing obtained during routine clinical care for diagnostic purposes as reported from an appropriate regulated laboratory using a clinically validated genetic test (genetic testing is not provided by the sponsor).
2. Subjects who are >=4 years old must be receiving glucocorticosteroids for a minimum of 6 months prior to signing informed consent. There should be no significant change (<0.2 mg/kg) in dosage or dose regimen (not related to body weight change) for at least 3 months immediately prior to signing the informed consent. Subjects who are aged >4 years will be exempt from this requirement; those not taking GC will be eligible if the initiation of GC treatment in these subjects is considered inappropriate in the opinion of Investigators.

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Exclusion Criteria

1. Any injury which may impact functional testing. Previous injuries must be fully healed prior to consenting. Prior lower limb fractures must be fully healed and at least 3 months from injury date.
2. Presence or history of other musculoskeletal or neurologic disease or somatic disorder not related to DMD including pulmonary, cardiac, and cognitive diseases.
3. Subjects >=4 years old who have not completed the varicella vaccination.
4. Participation in other studies involving investigational drug(s) for a minimum of 90 days prior to signing the informed consent and/or during study participation.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Study arm	Visit frequency	All subjects in this study will be observed for 24-30 months.

Primary Outcome Measures

Name	Time	Method
Age of Participants When They Failed to Walk	Up to Month 30	Participant's age at life-altering clinical milestones- failure to walk was calculated based on the birthdate and the date of failure to walk as reported by caregiver during 30 months of this study. Participants who were not reported being failure to walk by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.
Age of Participants When They Failed to Stand	Up to Month 30	Participant's age at life-altering clinical milestones- failure to stand was calculated based on the birthdate and the date of failure to stand as reported by caregiver during 30 months of this study. Participants who were not reported being failure to stand by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.
Age of Participants When They Failed to Self-feed	Up to Month 30	Participant's age at life-altering clinical milestones- failure to self-feed during 30 months of this study was analyzed using the Kaplan-Meier method. Age was summarized in years.
Change From Baseline in Northstar Ambulatory Assessment (NSAA) Total Score at Month 6: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 6	NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (example \[e.g.\] standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.
Change From Baseline in NSAA Total Score at Month 12: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 12	NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.
Change From Baseline in NSAA Total Score at Month 18: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 18	NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.
Change From Baseline in NSAA Total Score at Month 24: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 24	NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.
Change From Baseline in NSAA Total Score at Month 30: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 30	NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.
Change From Baseline in Performance of Upper Limb (PUL) 2.0 Total Score at Month 6: Participants Aged >=10 Years	Baseline (Day 1) and Month 6	PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.
Change From Baseline in PUL 2.0 Total Score at Month 12: Participants Aged >=10 Years	Baseline (Day 1) and Month 12	PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.
Change From Baseline in PUL 2.0 Total Score at Month 18: Participants Aged >=10 Years	Baseline (Day 1) and Month 18	PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.
Change From Baseline in PUL 2.0 Total Score at Month 24: Participants Aged >=10 Years	Baseline (Day 1) and Month 24	PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.
Change From Baseline in PUL 2.0 Total Score at Month 30: Participants Aged >=10 Years	Baseline (Day 1) and Month 30	PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.
Change From Baseline in Rise From Floor Velocity at Month 6: Ambulatory Participants Aged >=3 Years Only	Baseline (Day 1) and Month 6	The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.
Change From Baseline in Rise From Floor Velocity at Month 12: Ambulatory Participants Aged >=3 Years Only	Baseline (Day 1) and Month 12	The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.
Change From Baseline in Rise From Floor Velocity at Month 18: Ambulatory Participants Aged >=3 Years Only	Baseline (Day 1) and Month 18	The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.
Change From Baseline in Rise From Floor Velocity at Month 24: Ambulatory Participants Aged >=3 Years Only	Baseline (Day 1) and Month 24	The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.
Change From Baseline in Rise From Floor Velocity at Month 30: Ambulatory Participants Aged >=3 Years Only	Baseline (Day 1) and Month 30	The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 6: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 6	The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 12: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 12	The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 18: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 18	The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 24: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 24	The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 30: Ambulatory Participants Aged >=3 Years	Baseline (Day 1) and Month 30	The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.
Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years	Baseline (Day 1) and Month 6	Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years	Baseline (Day 1) and Month 12	Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years	Baseline (Day 1) and Month 18	Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years	Baseline (Day 1) and Month 24	Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years	Baseline (Day 1) and Month 30	Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years	Baseline (Day 1) and Month 6	Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years	Baseline (Day 1) and Month 12	Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years	Baseline (Day 1) and Month 18	Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years	Baseline (Day 1) and Month 24	Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years	Baseline (Day 1) and Month 30	Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years	Baseline (Day 1) and Month 6	Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years	Baseline (Day 1) and Month 12	Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years	Baseline (Day 1) and Month 18	Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years	Baseline (Day 1) and Month 24	Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years	Baseline (Day 1) and Month 30	Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years	Baseline (Day 1) and Month 6	Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years	Baseline (Day 1) and Month 12	Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years	Baseline (Day 1) and Month 18	Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years	Baseline (Day 1) and Month 24	Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years	Baseline (Day 1) and Month 30	Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.
Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6	Baseline (Day 1) and Month 6	Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.
Change From Baseline in ROM at Bilateral Ankles at Month 12	Baseline (Day 1) and Month 12	Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.
Change From Baseline in ROM at Bilateral Ankles at Month 18	Baseline (Day 1) and Month 18	Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.
Change From Baseline in ROM at Bilateral Ankles at Month 24	Baseline (Day 1) and Month 24	Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.
Change From Baseline in ROM at Bilateral Ankles at Month 30	Baseline (Day 1) and Month 30	Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.
Change From Baseline in ROM at Bilateral Elbows at Month 6	Baseline (Day 1) and Month 6	Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.
Change From Baseline in ROM at Bilateral Elbows at Month 12	Baseline (Day 1) and Month 12	Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.
Change From Baseline in ROM at Bilateral Elbows at Month 18	Baseline (Day 1) and Month 18	Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.
Change From Baseline in ROM at Bilateral Elbows at Month 24	Baseline (Day 1) and Month 24	Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.
Change From Baseline in ROM at Bilateral Elbows at Month 30	Baseline (Day 1) and Month 30	Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.
Change From Baseline in Percent Predicted Forced Vital Capacity (%pFVC) at Month 12: Participants Aged >=6 Years	Baseline (Day 1) and Month 12	Forced vital capacity (FVC) is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.
Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years	Baseline (Day 1) and Month 24	FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.
Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years	Baseline (Day 1) and Month 30	FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.
Change From Baseline in %pFVC at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 12	FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 24	FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 30	FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%pFEV1) at Month 12: Participants Aged >=6 Years	Baseline (Day 1) and Month 12	Forced expiratory volume in one second (FEV1) is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years	Baseline (Day 1) and Month 24	FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years	Baseline (Day 1) and Month 30	FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in %pFEV1 at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 12	FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 24	FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 30	FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in Maximum Inspiratory Pressure at Month 12: Participants Aged >=6 Years	Baseline (Day 1) and Month 12	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in Maximum Inspiratory Pressure at Month 24: Participants Aged >=6 Years	Baseline (Day 1) and Month 24	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in Maximum Inspiratory Pressure at Month 30: Participants Aged >=6 Years	Baseline (Day 1) and Month 30	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in Maximum Expiratory Pressure at Month 12: Participants Aged >=6 Years	Baseline (Day 1) and Month 12	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.
Change From Baseline in Maximum Expiratory Pressure at Month 24: Participants Aged >=6 Years	Baseline (Day 1) and Month 24	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.
Change From Baseline in Maximum Expiratory Pressure at Month 30: Participants Aged >=6 Years	Baseline (Day 1) and Month 30	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.
Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years	Baseline (Day 1) and Month 12	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years	Baseline (Day 1) and Month 24	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years	Baseline (Day 1) and Month 30	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 12	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 24	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 30	The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 12: Participants Aged >=6 Years	Baseline (Day 1) and Month 12	LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years	Baseline (Day 1) and Month 24	LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years	Baseline (Day 1) and Month 30	LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.
Change From Baseline in LVEF at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 12	LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 24	LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	Baseline (Day 1) and Month 30	LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.
Change From Baseline in Wechsler Intelligence Scale for Children (WISC)-IV Score at Month 24: Ambulatory Participants >= 6 to <=16 Years	Baseline (Day 1) and Month 24	WISC-IV is an individually administered intelligence test for children between the ages of 6 and 16. The WISC-IV Composites are: Verbal Comprehension, Perceptual Reasoning, Working Memory, and Processing Speed. Scores from the Composites constitute the WISC-IV Full Scale IQ score which ranges from 40 (Exceptionally Low) to 160 (Exceptionally Superior), higher scores indicated more intelligence. The WISC was only performed in ambulatory participants \>= 6 to \<=16 years old as pre-specified in the protocol.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Type of DMD Mutation	Up to Month 30	Number of participants as per type of mutation: exon deletion, exon duplication, point mutation, small insertion, small deletion and others is presented in this outcome measure. One participant could have more than 1 mutation type.
Number of Participants With Each Affected Exon by Mutation Types	Up to Month 30	Number of participants with each affected exon by mutation types is presented in this outcome measure. Only those categories with non-zero values have been reported.
Number of Participants With DMD Mutations Affecting Any Exon Between Exon 9 and Exon 13 or Deletion That Affects Both Exon 29 and Exon 30	Up to Month 30	Number of participants with DMD mutations affecting any exon between exon 9 and exon 13 or deletion that affects both exon 29 and exon 30 is presented in this outcome measure.
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30	Baseline (Day 1) and Months 6, 12, 18, 24 and 30	The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30	Baseline (Day 1) and Months 6, 12, 18, 24 and 30	The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30	Baseline (Day 1) and Months 6, 12, 18, 24 and 30	The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12	Months 12	EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants \>= 16 years old as pre-specified in protocol.
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24	Month 24	EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants \>= 16 years old as pre-specified in protocol.
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30	Month 30	EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants \>= 16 years old as pre-specified in protocol.
Change From Baseline in EQ-5D-3L Visual Analogue Score (VAS) Assessment at Months 12, 24 and 30	Baseline (Day 1) and Months 12, 24 and 30	EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state), where higher scores indicated better quality of life. EQ-VAS assessment was only performed in the participants \>= 16 years old.
Change From Baseline in EQ-5D-3L Index Score at Months 12, 24 and 30	Baseline (Day 1) and Months 12, 24, and 30	EQ-5D-3L index score, participants rated their current health state on 5 single-item dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression with each dimension having three levels of severity: 1=no problems, 2=some problems and 3=extreme problems. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total index score range of 0 to 1.00. Higher scores indicating a better quality of life. EQ-5D-3L was only performed in participants \>= 16 years old.
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12	Month 12	EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24	Month 24	EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30	Month 30	EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.
Change From Baseline in EQ-5D-Y VAS Assessment at Months 12, 24, and 30	Baseline (Day 1) and Months 12, 24, and 30	EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).
Change From Baseline in EQ-5D-Y Index Score at Months 12, 24, and 30	Baseline (Day 1) and Months 12, 24, and 30	EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<=16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems. Participants indicated their health state by choosing the appropriate level from each dimension. The 5 digit health states thus obtained for each dimension were then converted into a single median index value. A health state index score was calculated from individual health profiles using a USA scoring algorithm. Health state index scores generally ranged from -0.109 to 1, where, -0.109= the worst health status, 1= full health. Higher the score the better the better quality of life.
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30	Baseline (Day 1) and Months 12, 24, and 30	HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to their child's use of healthcare professionals, emergency room visits, and hospitalizations in past 3 months. Change from baseline in mean number of visits to primary care physician, emergency room and office visits is presented in this outcome measure.
Change From Baseline in HRU Survey Responses: Number of Nights in Hospital Due to Disease/Medication for Disease at Months 12, 24, and 30	Baseline (Day 1) and Months 12, 24, and 30	HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to number of nights in hospital due to disease/dedication for disease in past 3 months. Change from baseline in mean number of nights in hospital due to disease or medication for disease is presented in this outcome measure.
Change From Baseline in Out-of-Pocket Money of HRU Survey at Months 12, 24 and 30	Baseline (Day 1) and Months 12, 24, and 30	Caregivers were asked to estimate out-of-pocket costs related to healthcare resource utilization. The number of out of pocket money was defined as the total spent of the past three months in managing child's Duchenne muscular dystrophy.
Change From Baseline in Percent Activity Impairment as Per Work Productivity and Activity Impairment Questionnaire Adapted for Caregiving (WPAI:CG) Caregiver Unchanged at Months 12, 24, and 30	Baseline (Day 1) and Months 12, 24, and 30	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 12	Baseline (Day 1) and Month 12	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 24	Baseline (Day 1) and Month 24	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 30	Baseline (Day 1) and Month 30	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30	Baseline (Day 1) and Months 12, 24, and 30	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Percent Activity Impairment Score as Per WPAI: CG at Months 12, 24, and 30	Months 12, 24, and 30	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Percent Work Time Missed Score as Per WPAI: CG at Month 12	Month 12	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Percent Work Time Missed Score as Per WPAI: CG at Month 24	Month 24	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Percent Work Time Missed Score as Per WPAI: CG at Month 30	Month 30	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30	Months 12, 24, and 30	WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Trial Locations

Locations (8)

Children's Hospital of Chongqing Medical University (Liangjiang Branch); 🇨🇳 Chongqing, Chongqing, China

Children's Hospital of Chongqing Medical University; 🇨🇳 Chongqing, Chongqing, China

Huashan Hospital, Fudan University; 🇨🇳 Shanghai, China

Children's Hospital of Fudan University; 🇨🇳 Shanghai, China

Beijing Children's Hospital, Capital Medical University; 🇨🇳 Beijing, China

The First Affiliated Hospital Of Fujian Medical University; 🇨🇳 Fuzhou, Fujian, China

Peking University First Hospital; 🇨🇳 Beijing, China

Affiliated children's hospital of fudan university; 🇨🇳 Shanghai, China