Stopped: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
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.
Age range
4 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With All-Causality TEAEs by System Organ Class (SOC) and Preferred Term (PT) for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Treatment-Related TEAEs for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Treatment-Related TEAEs by SOC and PT for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With All-Causality SAEs by SOC and PT for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Treatment-Related SAEs by SOC and PT for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Hematology
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Clinical Chemistry
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Laboratory Test Abnormalities Without Regard to Baseline Abnormality for 1-Year Follow-Up - Urinalysis
Timeframe: Baseline up to 1 year post dose of PF-06939926
Change From Baseline on Left Ventricular Ejection Fraction as Measured by Cardiac Magnetic Resonance Imaging (MRI) for 1-Year Follow-Up
Timeframe: Baseline, 1 year post dose of PF-06839926 (Day 360)
Number of Participants With Electrocardiograms (ECGs) Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926
Number of Participants With Positive Responses on Columbia-Suicide Severity Rating Scale (C-SSRS) According to the Columbia Classification Algorithm of Suicide Assessment (C-CASA) Categories
Timeframe: Baseline, Day 7, Day 14, Day 180, and Day 360
Number of Participants With Vital Signs Meeting the Pre-Defined Categorical Criteria for 1-Year Follow-Up
Timeframe: Baseline up to 1 year post dose of PF-06939926