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NCT03362502 · ClinicalTrials.gov registry record · Phase 1
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
A Phase 1 study, sponsored by Pfizer.
- Terminated
- Registry status
- Phase 1
- Development phase
- 23
- Enrollment target
NCT03362502: Clinical Trial Phase 1 study, sponsored by Pfizer.
NCT03362502 is a Phase 1 study that was terminated before completion, run by Pfizer. The registered enrollment target is 23 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (62% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03362502, a Phase 1 study, was terminated before completion, sponsored by Pfizer.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 23 participants
- Enrollment target
Study Summary
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. A total of approximately 22 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.
Primary Outcome
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Any AE that started following start of treatment of PF-06939926 was counted as a treatment-emergent AE (TEAE). A serious AE (SAE) was an untoward medical occurrence that resulted in death, was life-threatening (immediate risk of death), required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disabil
Interventions
- GENETIC PF-06939926
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 23 participants |
| Start Date | 2018-01-23 |
| Est. Completion | 2025-07-28 |
| Phase | Phase 1 |
Why NCT03362502 stopped before completion
NCT03362502 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 23 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (62% lower).
The record links to 0 conditions, and to 1 intervention - of which PF-06939926 is the first listed.
NCT03362502 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03362502 about?
NCT03362502 is a clinical study titled "A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy". This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of...
What is the current status of trial NCT03362502?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 23 participants. The study started on 2018-01-23. Estimated completion is 2025-07-28.
What interventions are being tested in trial NCT03362502?
The interventions under investigation include: PF-06939926 (GENETIC).
Who is sponsoring clinical trial NCT03362502?
This trial is sponsored by Pfizer, which has 1,845 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03362502's enrollment target sits among peer trials
23 1596th of 2000 higher than 400 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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