An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
Summary
This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.
Timeline
- Start
- 2016-10-13
- Primary completion
- 2018-11-22
- Completion
- 2018-11-22
Outcome
Missed primary endpoint
Stopped: “Termination Date: 30Aug2018; Reason for termination: Lack of Efficacy”
paper Difference in mean (95% CI) change from baseline in 4SC at week 49 for domagrozumab vs placebo was 0.27 (-7.4 to 7.9) seconds (p = 0.94). PMID 32522498 ↗
paper Efficacy measures did not support a significant treatment effect. PMID 32522498 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | domagrozumab | Monoclonal antibody | 5 mg/kg | Intravenous |
| Subject | domagrozumab | Monoclonal antibody | 20 mg/kg | Intravenous |
| Subject | domagrozumab | Monoclonal antibody | 40 mg/kg | Intravenous |