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PF-06939926 Gene Therapy for Duchenne Muscular Dystrophy
Study Summary
This trial is a safety and tolerability study of a new drug for Duchenne muscular dystrophy. The drug will be given to 22 people, some of whom will also take sirolimus. The trial will be conducted in two phases, with a review by an external data monitoring committee before progressing to the next phase.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- Group 1: PF-06939926
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Has PF-06939926 earned regulatory authorization from the Food and Drug Administration?
"PF-06939926's safety was ranked 1 due to its Phase 1 status, indicating that there is only limited data regarding the drug's efficacy and security."
Is this research actively recruiting participants?
"Clinicaltrials.gov shows that this medical trial is not presently enrolling patients, despite having been added to the directory on January 23rd 2018 and amended most recently in November of 2022. Although 86 other trials are currently recruiting participants, this one is unfortunately closed for now."
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