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Gene Therapy

PF-06939926 Gene Therapy for Duchenne Muscular Dystrophy

Phase 1
Waitlist Available
Research Sponsored by Pfizer
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age as follows, based on ambulatory status: FOR AMBULATORY PARTICIPANTS: Between 4 and 12 years, FOR NON-AMBULATORY PARTICIPANTS: No age restrictions so long as loss of ambulation occurs prior to the subject's 17th birthday; Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing; Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry; Ability to tolerate magnetic resonance imaging (MRI) without sedation and with no contraindications; Ability to tolerate muscle biopsies under anesthesia with no contraindications; Body weights as follows, based on ambulatory status: FOR AMBULATORY PARTICIPANTS: Between 15 kg and 50 kg, FOR NON-AMBULATORY PARTICIPANTS: Less than 75 kg; Functional performance as follows, based on ambulatory status: FOR AMBULATORY PARTICIPANTS: Ability to rise from floor within seven (7) seconds, FOR NON-AMBULATORY PARTICIPANTS: Percent predicted forced vital capacity greater than 40% as part of pulmonary function tests, as well as adequate upper limb function; > 8 years of age for Sirolimus Cohort
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through 5 years post-treatment
Awards & highlights

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.

Who is the study for?
This trial is for boys with Duchenne muscular dystrophy (DMD), both walking and non-walking, who can handle MRI scans without sedation. Participants must meet specific genetic criteria, have a certain level of muscle function, and be on stable glucocorticoids. Boys should weigh under 50 kg if walking or under 75 kg if not. Those in the Sirolimus Cohort must be over 8 years old.Check my eligibility
What is being tested?
The study tests PF-06939926 gene therapy's safety in DMD patients by giving a single IV dose to about 22 participants. It includes staggered enrollment and reviews by an external committee before increasing doses. The trial also measures dystrophin levels, muscle strength, quality, and function.See study design
What are the potential side effects?
Specific side effects are not listed but may include reactions related to immune response against the viral vector used for gene therapy or general risks associated with intravenous infusions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through 5 years post-treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and through 5 years post-treatment for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of dose-limiting safety or intolerability, as measured by treatment-related adverse events
Secondary outcome measures
Evidence of mini-dystrophin expression and distribution assessed by immunohistochemistry, western blot, and/or LC-MS using muscle biopsies
Incidence and magnitude of abnormal laboratory findings
Incidence and severity of abnormal and clinical relevant changes in Columbia Suicide Severity Rating Scale (C-SSRS)
+7 more
Other outcome measures
Percent change from baseline of NAb antibody titers
Percent change from baseline of nadir in C4 levels
Percent change from baseline of nadir in platelet levels

Trial Design

1Treatment groups
Experimental Treatment
Group I: PF-06939926Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor
4,556 Previous Clinical Trials
10,907,660 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,467 Previous Clinical Trials
8,089,097 Total Patients Enrolled

Media Library

PF-06939926 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03362502 — Phase 1
Duchenne Muscular Dystrophy Research Study Groups: PF-06939926
Duchenne Muscular Dystrophy Clinical Trial 2023: PF-06939926 Highlights & Side Effects. Trial Name: NCT03362502 — Phase 1
PF-06939926 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03362502 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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."

Answered by AI
~3 spots leftby Mar 2025