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Gene Therapy

Gene Therapy (PF-06939926) for Duchenne Muscular Dystrophy

Phase 2

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 DMD by prior genetic testing

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at week 9, week 52 and year 5 (if available)

Awards & highlights

Study Summary

This trialwill study gene therapy to help boys with Duchenne Muscular Dystrophy (DMD) with safety and muscle strength. No randomization, open to all.

Who is the study for?

This trial is for boys with a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) who haven't had gene therapy before. They must have normal blood counts and liver/kidney function, no specific genetic mutations in the dystrophin gene, and no recent treatments aimed at increasing dystrophin expression.Check my eligibility

What is being tested?

The study tests Fordadistrogene Movaparvovec (PF-06939926), a gene therapy for DMD. It's an open-label study where all participants receive the treatment to assess safety and how well it promotes dystrophin production.See study design

What are the potential side effects?

Potential side effects may include immune reactions to the therapy vector AAV9, changes in liver enzymes indicating liver stress, possible allergic responses, or other unforeseen issues related to gene therapy.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have a confirmed genetic diagnosis of Duchenne Muscular Dystrophy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at week 9, week 52 and year 5 (if available)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at week 9, week 52 and year 5 (if available)

This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 9, week 52 and year 5 (if available) for reporting.

Treatment Details

Study Objectives

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

Secondary outcome measures

Distribution of mini-dystrophin expression in muscle

Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events

Level of mini-dystrophin expression in muscle

+9 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: PF-06939926Experimental Treatment1 Intervention

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,567 Previous Clinical Trials

10,911,864 Total Patients Enrolled

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,474 Previous Clinical Trials

8,093,032 Total Patients Enrolled

Media Library

PF-06939926 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05429372 — Phase 2

Duchenne Muscular Dystrophy Research Study Groups: PF-06939926

Duchenne Muscular Dystrophy Clinical Trial 2023: PF-06939926 Highlights & Side Effects. Trial Name: NCT05429372 — Phase 2

PF-06939926 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05429372 — Phase 2

Frequently Asked Questions

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

Is enrollment open to those over 20 years of age for this clinical experiment?

"This clinical trial's eligibility criteria necessitates that participants are aged between 2 and 3. Individuals who fall below the age of consent can take part in 59 separate studies, while those above 65 can enrol into 50 different trials."

Answered by AI

Am I able to join this clinical investigation?

"Applicants aged 2 - 3 with a formally diagnosed case of becker muscular dystrophy are currently being sought for this study. Critically, those hoping to join must have prior genetic testing certifying their diagnosis."

Answered by AI

Is this experiment actively accepting new participants?

"According to clinicaltrials.gov, the recruitment period for this medical trial is ongoing; it first went live on 8th August 2022 and was recently revised on 12th December 2022."

Answered by AI

Has the FDA authorized PF-06939926 for general use?

"We at Power have determined that the safety of PF-06939926 can be rated a 2, as this is a Phase 2 clinical trial and while there exists some evidence demonstrating its security, no data has yet been provided suggesting efficacy."

Answered by AI

How many participants have volunteered for this research experiment?

"Affirmative. Clinicaltrials.gov shows that this medical study, which was launched on August 8th 2022, is actively enrolling patients. At six different trial sites across the nation, 10 participants must be recruited for the clinical investigation to progress as planned."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

2022. "Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05429372.

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