A Study of TAK-771 in Japanese Participants With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal Motor Neuropathy (MMN)
- Conditions
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)Multifocal Motor Neuropathy (MMN)
- Interventions
- Registration Number
- NCT05084053
- Lead Sponsor
- Takeda
- Brief Summary
The main aim of the study is to check for side effects from TAK-771, and to check how well TAK-771 controls symptoms in Japanese participants with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and multifocal motor neuropathy (MMN)
The participants will be treated with TAK-771 for 45 months as a maximum.
There will be many clinic visits. The number of visits will depend on the infusion cycles of study drug (every 2, 3, or 4 weeks).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 26
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Cohort 1: TAK-771 for CIDP Participants TAK-771 TAK-771 includes Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20). Participants will receive subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 2, 3, or 4 weeks. Cohort 2: TAK-771 for MMN Participants TAK-771 TAK-771 includes IGI 10% and rHuPH20. Participants will receive subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution, every 2, 3, or 4 weeks.
- Primary Outcome Measures
Name Time Method Epoch 1: Percentage of Participants With CIDP Who Experienced Relapse Epoch 1: Baseline up to 6 months Relapse was defined as worsening of functional disability defined as an increase of \>=1 point relative to the pre-subcutaneous (pre-SC) treatment baseline score in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score. The INCAT disability scale was the most widely used assessment tool to measure the functional activity level of participants with CIDP. The INCAT disability scale consisted of upper and lower extremity components, with a maximum of 5 points for the upper extremities (arm disability) and a maximum of 5 points for the lower extremities (leg disability), which were summed for an overall INCAT disability score ranging from 0 to 10 points, where 0 was normal and 10 was severely incapacitated. An adjusted INCAT disability score was the same as the INCAT disability score, with the only exception in the exclusion of changes from 0 (normal) to 1 (minor symptoms) (or vice versa) in upper limb function.
Epoch 1: Change From Baseline in Maximum Grip Strength in the More Affected Hand in Participants With MMN Epoch 1: Baseline up to 6 months The Martin Vigorimeter was used to assess grip strength in both hands. The instrument consisted of a compressible rubber ball that was connected to a manometer. When the rubber ball was squeezed, the force of compression was measured in kilopascal (kPa) ranging from 0 to 160 kPa.
- Secondary Outcome Measures
Name Time Method Epoch 1 and 2 (6 Months): Number of Participants With Related SAEs and TEAEs Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Related SAES and TEAEs: that followed a reasonable temporal sequence from administration of a drug (including the course after withdrawal of the drug), or for which possible involvement of the drug cannot be ruled out, although factors other than the drug, such as underlying diseases, complications, concomitant medications and concurrent treatments, may also be responsible.
Epoch 1 and 2 (6 Months): Number of Participants With Serious and Non-serious Adverse Reactions (ARs) Plus Suspected ARs Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Adverse reactions plus suspected ARs were defined as TEAEs that were considered by the investigator to be related to IP administration, or for which the causality was indeterminate or missing, or that began during infusion of IP or within 72 hours following the end of IP infusion.
Epoch 1 and 2 (6 Months): Number of Infusions for Which the Infusion Rate Was Reduced and/or the Infusion Was Interrupted or Stopped Due to Intolerability and/or AEs Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Number of infusions for which the infusion rate was reduced and/or the infusion was interrupted or stopped due to intolerability and/or AEs were reported.
Epoch 1 and 2 (6 Months): Number of Participants With Positive Binding Antibodies, and Positive Neutralizing Antibodies to rHuPH20 Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Plasma samples for the detection of anti-rHuPH20 binding and neutralizing antibodies were collected. Participants were monitored for the formation of anti-rHuPH20 antibodies using validated anti-rHuPH20 antibody detection assay (also known as the Screening and Confirmatory Binding Assay). Positive antibodies were defined as participants who had anti rHuPH20 antibody titer greater than or equal to (\>=) 1:160 at least one time during treatment.
Epoch 1 and 2 (6 Months): Time to Relapse in Participants With CIDP Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Time to relapse was defined as time from the date of the first SC administration of TAK-771 in Epoch 1 or 2 to the date of relapse.
Epoch 1 and 2 (6 Months): Number of Participants With Treatment-emergent Adverse Events (TEAEs) Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 TEAEs were defined as adverse events that occurred during or after administration of the first dose of investigational product (IP). TEAEs caused in Epoch 1 were defined as AEs that occurred on or after the start of study drug administration, and before the first dose of IP in Epoch 2 (6 months). TEAEs caused in Epoch 2 (6 months) were defined as AEs that began during or after administration of the first dose of IP in Epoch 2 (6 months).
Epoch 1 and 2 (6 Months): Number of Participants With Serious Adverse Events (SAEs) Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 SAEs were defined as any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) at any dose: resulted in death, was life-threatening, required inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, or any other important medical event.
Epoch 1 and 2 (6 Months): Number of Participants With SAEs and/or TEAEs Associated With Infusions Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Infusion associated SAEs and/or TEAEs were defined as SAEs and/or TEAEs considered to be "infusion-related reactions" by investigators.
Epoch 1 and 2 (6 Months): Number of Participants With Related SAEs and TEAEs Associated With Infusions Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Related SAES and TEAEs: that followed a reasonable temporal sequence from administration of a drug (including the course after withdrawal of the drug), or for which possible involvement of the drug cannot be ruled out, although factors other than the drug, such as underlying diseases, complications, concomitant medications and concurrent treatments, may also be responsible. SAEs and TEAEs associated with infusions were the events considered to be "infusion-related reactions" by investigators.
Epoch 1 and 2 (6 Months): Number of Participants With TEAEs Temporally Associated With Infusions Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 TEAEs were defined as adverse events that occurred during or after administration of the first dose of IP. TEAEs caused in Epoch 1 were defined as AEs that occurred on or after the start of study drug administration, and before the first dose of IP in Epoch 2 (6 months). TEAEs caused in Epoch 2 (6 months) were defined as AEs that began during or after administration of the first dose of IP in Epoch 2 (6 months). TEAEs temporally associated with infusions defined as AEs occurring during or within 72 hours after completion of an infusion.
Epoch 1 and 2 (6 Months): Number of Participants With Serious and Non-serious ARs Plus Suspected ARs Temporally Associated With Infusions Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Adverse reactions plus suspected ARs were defined as TEAEs that were considered by the investigator to be related to IP administration, or for which the causality was indeterminate or missing, or that began during infusion of IP or within 72 hours following the end of IP infusion. AEs temporally associated with infusions are defined as AEs occurring during or within 72 hours after completion of an infusion.
Epoch 1 and 2 (6 Months): Number of Participants With Systemic TEAEs Associated With Infusions Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Any TEAEs other than local TEAEs were considered a systemic TEAE. Infusion associated TEAEs were defined as TEAE considered to be "infusion-related reactions" by investigators.
Epoch 1 and 2 (6 Months): Number of Participants With Treatment-emergent Local Infusion Site Reactions Associated With Infusions Epoch 1: Baseline up to 6 months; Epoch 2: Up to 6 months post-Epoch 1 Any reaction with the medical dictionary for regulatory activities (MedDRA) high-level group term (HLGT) of "Administration site reactions" were considered a local infusion site reaction (local TEAE). In addition, any TEAE with Injection Site Reaction Flag = "Yes" was considered a local TEAE. Infusion associated reaction were defined as event considered to be "infusion-related reactions" by investigators.
Epoch 1: Percentage of Participants With Clinical Worsening of CIDP Epoch 1: Baseline until end of Epoch 1 or relapse (up to 6 months) Clinical Worsening of CIDP was defined as a \>=8 kPa decrease in hand grip strength in the more affected hand OR \>=4 points decrease in Rasch Built Overall Disability Scale (R-ODS) relative to the pre-SC treatment baseline score at 2 consecutive time points. Hand grip strength was measured using the Martin vigorimeter by quantifying air pressure. The R-ODS was a participant self-reported, linearly-weighted overall disability scale that was specifically designed to capture activity and social participation limitations in participants with immune-mediated peripheral neuropathies including CIDP. R-ODS was comprised of 24 items that rate participant's functioning related to a variety of everyday tasks at moment of completion on a scale of 0 to 2 (where 0 indicates it is not possible for the respondent to perform the task and 2 means that task can be performed without difficulty). Total scores range from 0 to 48 and higher scores indicate greater ability to perform daily and social tasks.
Epoch 1: Change From Pre-subcutaneous Treatment (Baseline) in R-ODS Total Score in Participants With CIDP Epoch 1: Baseline up to 6 months The R-ODS was a participant self-reported, linearly-weighted overall disability scale that was specifically designed to capture activity and social participation limitations in participants with immune-mediated peripheral neuropathies including CIDP. R-ODS was comprised of 24 items that rate participant's functioning related to a variety of everyday tasks at moment of completion on a scale of 0 to 2 (where 0 indicates it is not possible for the respondent to perform the task and 2 means that task can be performed without difficulty). Total scores range from 0 to 48 and higher scores indicate greater ability to perform daily and social tasks.
Epoch 1: Change From Pre-subcutaneous Treatment (Baseline) in an Average of Handgrip Strength of Both Hands in Participants With CIDP and MMN Epoch 1: Baseline up to 6 months The Martin Vigorimeter was used to assess grip strength in both hands. The instrument consisted of a compressible rubber ball that was connected to a manometer. When the rubber ball was squeezed, the force of compression was measured in kPa ranging from 0 to 160 kPa. Average of grip strength was defined as an average of the two maximum values: maximum of the 3 measurements in the more affected hand and the maximum of the 3 measurements in the less affected hand.
Epoch 1: Change From Pre-subcutaneous Treatment (Baseline) Total Medical Research Council (MRC) Sum Score in Participants With MMN Epoch 1: Baseline up to 6 months The MRC sum score served as a measure of muscle strength. The following muscles on each side of the body were examined and the strength of each muscle was rated according to the MRC scale: deltoids, biceps, wrist extensors, iliopsoas, quadriceps, and anterior tibialis. The MRC scale ranged from 0 to 5, where: 0 = no visible contraction; 1 = visible contraction without movement of the limb; 2 = movement of the limb but not against gravity; 3 = movement against gravity over (almost) the full range; 4 = movement against gravity and resistance; and 5 = normal. All scores from both left and right side of the body were summed to obtain the total MRC sum score. The total MRC sum score ranged from 0 (paralysis) to 60 (normal strength) with higher score indication normal strength.
Epoch 1: Number of MMN Participants With Increased Guy's Neurological Disability Scale (GNDS) Score in Upper Limb and Lower Limb Categories Epoch 1: Baseline up to 6 months GNDS was a questionnaire which consisted of 12 separate categories (4 to 8 questions per category). The categories included: cognition, mood, vision, speech, swallowing, upper limb function, lower limb function, bladder function, bowel function, sexual function, fatigue, and others. In the current study, only 2 categories; upper limb function and the lower limb function was used for assessment of the disability of participants with MMN. The severity of each subscale (including upper limb and lower limb) was graded from 0 (normal function) to 5 (total loss of function) based according to severity and impact on the individual. The total GNDS score was the sum of the 12 separate scores ranging between 0 and 60 with higher scores indicating loss of function.
Trial Locations
- Locations (23)
Asahikawa Medical Center
🇯🇵Asahikawa, Hokkaido, Japan
Tokushima National Hospital
🇯🇵Yoshinogawa, Tokushima, Japan
Chiba University Hospital
🇯🇵Chiba, Japan
Tokyo Women's Medical University Hospital
🇯🇵Shinjuku-ku, Tokyo, Japan
Kyushu University Hospital
🇯🇵Fukuoka, Japan
Hiroshima University Hospital
🇯🇵Hiroshima, Japan
Toyama University Hospital
🇯🇵Toyama, Japan
Aichi Medical University Hospital
🇯🇵Nagakute, Aichi, Japan
Chubu Rosai Hospital
🇯🇵Nagoya, Aichi, Japan
Fujita Health University Hospital
🇯🇵Toyoake, Aichi, Japan
St.Marianna University School of Medicine Hospital
🇯🇵Kawasaki, Kanagawa, Japan
Hyogo College of Medicine Hospital
🇯🇵Nishinomiya, Hyogo, Japan
Kansai Rosai Hospital
🇯🇵Amagasaki, Hyogo, Japan
Tohoku Medical and Pharmaceutical University Hospital
🇯🇵Sendai, Miyagi, Japan
Shiga University of Medical Science Hospital
🇯🇵Otsu, Shiga, Japan
Nara Medical University Hospital
🇯🇵Kashihara, Nara, Japan
Higashimatsuyama Municipal Hospital
🇯🇵Higashimatsuyama, Saitama, Japan
National Center of Neurology and Psychiatry
🇯🇵Kodaira, Tokyo, Japan
Juntendo University Hospital
🇯🇵Bunkyo-ku, Tokyo, Japan
Yamaguchi University Hospital
🇯🇵Ube, Yamaguchi, Japan
Toho University Omori Medical Center
🇯🇵Ota-ku, Tokyo, Japan
Kumamoto University Hospital
🇯🇵Kumamoto, Japan
Tokushima University Hospital
🇯🇵Tokushima, Japan