Gene Therapy for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+11 other locations

Age: Any Age

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Pfizer

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Do I need to stop my current medications for this trial?

The trial protocol does not specify whether you need to stop taking your current medications.

What data supports the idea that Gene Therapy for Duchenne Muscular Dystrophy (also known as: fordadistrogene movaparvovec, BMB-D-001, PF 06939926) is an effective treatment?

The available research shows that fordadistrogene movaparvovec, a gene therapy, was effective in a rat model of Duchenne Muscular Dystrophy. The study found improvements in muscle strength, heart function, and muscle structure after treatment. This suggests that the therapy could be beneficial for patients at different stages of the disease. Compared to other treatments like drisapersen, which was tested in a placebo-controlled trial, gene therapy offers a more direct approach by aiming to correct the underlying genetic issue. ^{1 2 3 4 5}

What safety data exists for gene therapy in Duchenne Muscular Dystrophy?

The safety data for gene therapy in Duchenne Muscular Dystrophy includes findings from various studies. In a nonclinical study using a rat model, fordadistrogene movaparvovec showed therapeutic effectiveness with a correlation between dose and muscle improvement. In a Phase 1/2a trial, delandistrogene moxeparvovec was evaluated for long-term safety and functional outcomes, with common treatment-related adverse events being vomiting, decreased appetite, and nausea, all of which resolved. Another study reported robust expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment, with most adverse events occurring within the first 90 days and resolving. Delandistrogene moxeparvovec has been approved in the USA for certain pediatric patients, indicating a recognized safety profile for this age group. ^{1 2 3 4 5}

Is the treatment fordadistrogene movaparvovec a promising treatment for Duchenne Muscular Dystrophy?

Yes, fordadistrogene movaparvovec is a promising treatment for Duchenne Muscular Dystrophy. It has shown positive results in improving muscle strength and heart function in animal studies, suggesting it could be effective for patients at all stages of the disease. ^{1 2 3 4 5}

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

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.

Inclusion Criteria

I have previously received fordadistrogene movaparvovec in a Pfizer study.

Exclusion Criteria

Investigator site staff directly involved in the study and their family members

Treatment Details

Interventions

• fordadistrogene movaparvovec (Gene Therapy)

Trial Overview 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.

Participant Groups

Group I: All participants Experimental Treatment 1 Intervention

All participants enrolled in the study.

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Findings from Research

In a Phase 2 study involving 51 ambulant Duchenne muscular dystrophy (DMD) patients, drisapersen at a dose of 6 mg/kg/week showed a potential benefit in improving the 6-minute walking distance (6MWD) compared to placebo, with a mean difference of 27.1 meters at week 24.

Drisapersen was generally well-tolerated, with the most common side effects being injection site reactions and subclinical proteinuria, indicating a manageable safety profile for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. McDonald, CM., Wong, B., Flanigan, KM., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy. [2023]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy. [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Delandistrogene Moxeparvovec: First Approval. [2023]