Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
- Registration Number
- NCT02146378
- Lead Sponsor
- Pfizer
- Brief Summary
The purpose of this study is to understand safety (e.g., occurrence of adverse drug reactions \[ADRs\]) and efficacy data on the long-term use of Vyndaqel Capsules (hereinafter referred to as Vyndaqel) in all patients who received this drug under actual use conditions after its marketing.
- Detailed Description
Both prospective and retrospective is acceptable All the patients whom an investigator prescribes Vyndaqel should be registered
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 500
The subjects of this surveillance are all patients who received Vyndaqel.
Patients not receive Vyndaqel.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Vyndaqel Vyndaqel -
- Primary Outcome Measures
Name Time Method Percentage of Participants With ADRs in This Study 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) An adverse drug reaction (ADR) was any untoward medical occurrence attributed to Vyndaqel capsules 20mg in a participant who received Vyndaqel capsules 20mg.
A serious adverse drug reaction (SADR) was ADR resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect.
Relatedness to Vyndaqel capsules 20mg was assessed by the physician.
- Secondary Outcome Measures
Name Time Method Change From Baseline in the NIS-LL Score Week 78, Week 156 The amounts of change from baseline in the lower limbs (NIS-LL) was investigated for assessment of neurological function.The NIS-LL (Lower Limb) assessed muscle weakness, reflexes, and sensation (possible range: 0 to 88). Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated.Higher scores indicate more impaired neurological function.The mean changes from baseline in the NIS-LL at Week 78 and Week 156 were presented with standard deviation.
Change From Baseline in the TQOL Score of the Norfolk QOL-DN Week 78, Week 156 The amounts of change from baseline in the TQOL score of the QOL-DN were used to assess quality of life .The Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire which consists of symptoms items (1 to 7) and activities of daily living items (8 to 35) . Total Quality of Life (TQOL) (possible range: -4 to 136) in the QOL-DN was evaluated. Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher scores indicate decreased QOL.The mean changes from baseline in the TQOL score at Week 78 and Week 156 were presented with standard deviation.
Change From Baseline in mBMI Week 26, Week 52, Week 78, Week 104, Week 130, Week 156 Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher mBMI scores indicate better nutrition status.The mean changes from baseline in modified Body Mass Index (mBMI) at Week 26, Week 52, Week 78, Week 104, Week 130, and Week 156 were presented with standard deviation.mBMI can be calculated by multiplying BMI by the serum albumin level.In Transthyretin Familial Amyloid Polyneuropathy patients, oedema and subsequent weight gain may occur, but weight changes can be corrected with a modified BMI.
Time Course of Ambulatory Status Week 156 The percentage of participants were calculated in each group of ambulatory status change from the start of treatment with Vyndaqel capsules 20mg to the final evaluation time point (week 156). Ambulatory status (0, 1, 2, 3a, 3b, 4) was assessed using the ambulatory ability scale. The ambulatory ability scale in the polyneuropathy dysfunction score was defined as follows. Ambulatory status 0：Walking ability is Good. Ambulatory status 1：Walking ability is able to walk without difficulty despite of sensory disorder in the lower extremities. Ambulatory status. Ambulatory status 2：Walking ability is able to walk without assistance despite of some difficulties. Ambulatory status 3a：Walking ability is able to walk with one stick or crutch. Ambulatory status 3b：Walking ability is able to walk with two sticks or crutches. Ambulatory status 4: Walking ability is unable to walk, restricted to wheelchair or bedridden.
Change From Baseline in the Total Neurological Assessment Score Week 52, Week 104, Week 156 The total score of neurological assessment (possible range: 0 to 294), scores of Motor, Reflexes and sensation were evaluated and summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher scores indicate more decreased function.The mean changes from baseline in the total neurological assessment at Week 52 , Week 104, and Week 156 were presented with standard deviation.
Assessment of Survival Time for Death From Any Cause 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) By setting death from any cause as events, the survival probability was estimated using the Kaplan-Meier method that treats cases who didn't have events within the observation period as censoring.
Assessment of Survival Time for Death Associated With ATTR-PN 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) By setting death associated with transthyretin familial amyloid polyneuropathy (ATTR-PN) as events, the survival probability was estimated using the Kaplan-Meier method that treats cases who didn't have events within the observation period as censoring.