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PF-06939926 · 1 trial · 1 indication
The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. Baseline NSAA total score is defined as the last non-missing NSAA total score collected prior to Year 1 drug administration.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | OTHER | Approximately two thirds of participants will be randomized to Cohort 1. |
| Cohort 2 | OTHER | Approximately one third of participants will be randomized to Cohort 2. |
| Name | Type | Description |
|---|---|---|
| PF-06939926 | GENETIC | PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1. |
| Placebo | OTHER | Placebo will be administered as a single IV infusion at Year 1 for Cohort 2. |
Key inclusion criteria: 1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing 2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening 3. Ambulatory, as assessed by ...
PF-06939926 is an investigational gene therapy being developed for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
PF-06939926 is being developed by Pfizer, Inc. (NYSE: PFE). The company is conducting a Phase 3 clinical trial to evaluate the safety and efficacy of this gene therapy for Duchenne Muscular Dystrophy.
PF-06939926 is currently in Phase 3 clinical development. It is an investigational gene therapy for Duchenne Muscular Dystrophy and has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 3 trial is active but not recruiting participants.
PF-06939926 is being evaluated in a Phase 3 clinical trial with the identifier NCT04281485. This randomized, double-blind, controlled study is assessing the safety and efficacy of the gene therapy in male patients aged 4 years and older with Duchenne Muscular Dystrophy. The trial has an enrollment of 114 participants and is being conducted across multiple countries.
PF-06939926 is a gene therapy designed to deliver a functional version of the dystrophin gene to muscle cells. This approach aims to address the underlying genetic cause of Duchenne Muscular Dystrophy by enabling the production of a shortened but functional dystrophin protein, which is essential for muscle stability.