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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy

Phase 3

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing

Ambulatory, as assessed by protocol-specified criteria

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 52

Awards & highlights

Study Summary

This trial will test whether gene therapy is safe and effective for boys with Duchenne Muscular Dystrophy. Two thirds of participants will get the therapy, while the other third will get a placebo. Those in the placebo group will have the opportunity to get the therapy at the beginning of the second year.

Who is the study for?

This trial is for boys with Duchenne muscular dystrophy (DMD) confirmed by genetic testing, who can walk and have been on a stable dose of steroids for at least 3 months. Boys with antibodies to AAV9, recent treatments increasing dystrophin expression, prior gene therapy, non-healed injuries affecting tests or certain genetic abnormalities in the dystrophin gene cannot participate.Check my eligibility

What is being tested?

The study tests the safety and effectiveness of PF-06939926, a gene therapy for DMD. Participants are randomly assigned to receive either this gene therapy or a placebo in a blinded manner. Two-thirds will get the actual treatment while one-third receives placebo with an option to switch after one year.See study design

What are the potential side effects?

Potential side effects may include immune reactions due to viral vector used in gene therapy (like fever or chills), liver enzyme elevations indicating stress on the liver, blood count changes suggesting bone marrow impact, and possible allergic responses.

Eligibility Criteria

Inclusion Criteria

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

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I have been genetically tested and confirmed to have Duchenne muscular dystrophy.

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I can walk and move around on my own.

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I have been on a stable dose of steroids for at least 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from Baseline in North Star Ambulatory Assessment (NSAA)

Secondary outcome measures

Change from Baseline in distribution of mini-dystrophin expression in the muscle

Change from Baseline in mini-dystrophin expression level in muscle

Change from Baseline in serum creatine kinase (CK)

+6 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment2 Interventions

Approximately one third of participants will be randomized to Cohort 2.

Group II: Cohort 1Experimental Treatment2 Interventions

Approximately two thirds of participants will be randomized to Cohort 1.

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,567 Previous Clinical Trials

10,911,775 Total Patients Enrolled

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,474 Previous Clinical Trials

8,092,943 Total Patients Enrolled

Media Library

PF-06939926 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04281485 — Phase 3

Duchenne Muscular Dystrophy Research Study Groups: Cohort 1, Cohort 2

Duchenne Muscular Dystrophy Clinical Trial 2023: PF-06939926 Highlights & Side Effects. Trial Name: NCT04281485 — Phase 3

PF-06939926 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04281485 — Phase 3

Frequently Asked Questions

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

Are elderly patients able to participate in this trial?

"Eligibility for this trial is based on age, specifically participants must be between 4 and 7 years old."

Answered by AI

Has this cure been cleared by the FDA?

"The safety of this Phase 3 trial's treatment was estimated to be a 3."

Answered by AI

Does my profile fit the requirements for this experiment?

"Duchenne muscular dystrophy patients, aged 4-7, who are taking a stable daily dose of prednisone (0.5mg/kg), prednisolone (0.75mg/kg), or deflazacort (0.75mg/kg) for the 3 months preceding the study are welcome to enroll. Up to 99 participants are needed."

Answered by AI

Can people with this condition still enroll in the trial?

"Yes, the clinical trial is currently recruiting patients. It was first posted on 11/5/2020 and was last updated on 10/27/2022. There are 20 locations admitting a total of 99 patients."

Answered by AI

Are there a lot of places conducting this research study in North America?

"20 hospitals are running this clinical trial, these include Childrens Hospital of Eastern Ontario in Ottawa, The Hospital For Sick Children in Toronto and KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow in Kansas City. The other 20 locations can be found."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

2020. "Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04281485.

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