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Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Phase 3
Recruiting
Research Sponsored by Pfizer
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up annually from 5 through 10 years after dosing in the interventional study
Awards & highlights
Pivotal Trial
No Placebo-Only Group
Summary
This trial is testing a new gene therapy called fordadistrogene movaparvovec. It aims to see if the therapy is safe and effective over a long period. The treatment works by fixing or replacing problematic genes in the body.
Who is the study for?
This trial is for individuals who have Duchenne Muscular Dystrophy and were part of previous Pfizer studies where they received the gene therapy fordadistrogene movaparvovec. Participants will be monitored for 10 years with annual onsite visits and additional remote check-ins.
What is being tested?
The study focuses on the long-term safety and effects of an experimental gene therapy called fordadistrogene movaparvovec, which was administered in earlier trials. The follow-up includes yearly in-person assessments alongside several virtual visits.
What are the potential side effects?
Since this is a follow-up study, it primarily monitors ongoing or late-appearing side effects from the initial treatment with fordadistrogene movaparvovec, which may include immune reactions or muscle-related symptoms.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ annually from 5 through 10 years after dosing in the interventional study
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~annually from 5 through 10 years after dosing in the interventional study
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Change from pre-dose in the North Star Ambulatory Assessment total score
Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: All participantsExperimental Treatment1 Intervention
All participants enrolled in the study.
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Duchenne Muscular Dystrophy (DMD) include glucocorticoids, exon-skipping drugs, and gene therapy. Glucocorticoids help reduce inflammation and slow muscle degeneration.
Exon-skipping drugs, such as eteplirsen, golodirsen, and viltolarsen, work by skipping over specific exons in the DMD gene to produce a shorter but functional dystrophin protein. Gene therapy, like the experimental fordadistrogene movaparvovec, aims to introduce a functional version of the dystrophin gene into muscle cells, potentially restoring dystrophin production.
These treatments are crucial for DMD patients as they target the underlying genetic defects, aiming to improve muscle function and slow disease progression.
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Who is running the clinical trial?
PfizerLead Sponsor
4,675 Previous Clinical Trials
28,716,931 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,556 Previous Clinical Trials
25,757,787 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have previously received fordadistrogene movaparvovec in a Pfizer study.
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.