What side effects are associated with this type of intervention?

Common treatments for Duchenne Muscular Dystrophy (DMD) include gene therapies like eteplirsen, golodirsen, and casimersen, as well as glucocorticoids such as prednisone and deflazacort. Gene therapies often have side effects like upper respiratory tract infections, cough, fever, headache, joint pain, and hypersensitivity reactions including bronchospasm and chest pain. Glucocorticoids can cause weight gain, decreased linear growth, cushingoid appearance, delayed puberty, bone fractures, gastrointestinal symptoms, cataracts, and behavioral changes. Monitoring and managing these side effects are crucial for optimizing patient outcomes.

What prior FDA approvals exist?

Several gene therapies and related drugs have received FDA approval for the treatment of Duchenne Muscular Dystrophy (DMD). Eteplirsen (ExonDys 51) is approved for patients with a confirmed mutation amenable to exon 51 skipping, showing increased dystrophin production and some clinical benefits. Golodirsen (Vyondys 53) and Viltolarsen are approved for patients with mutations amenable to exon 53 skipping, both demonstrating increased dystrophin levels. Casimersen targets exon 45 skipping and has shown similar efficacy. These treatments aim to increase dystrophin production, a key protein deficient in DMD, though their long-term clinical benefits are still being evaluated. Fordadistrogene movaparvovec, an experimental gene therapy, is being studied for its long-term safety and effects in DMD patients.

What other types of research exist?

Promising novel alternatives to fordadistrogene movaparvovec for Duchenne Muscular Dystrophy patients include: 1) Exon-skipping therapies such as eteplirsen and golodirsen, which aim to restore the production of functional dystrophin protein. 2) CRISPR-Cas9 gene editing, which is being explored to correct the genetic mutations causing DMD. 3) Utrophin modulation therapies, which aim to upregulate utrophin, a protein similar to dystrophin, to compensate for its deficiency. 4) Anti-inflammatory and fibrosis-targeting drugs, which aim to reduce muscle damage and improve muscle function. These alternatives are in various stages of clinical development and may offer additional options for DMD treatment by 2024.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and annually from 5 through 10 years after dosing in the interventional study for reporting.

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.

Do I qualify?Apply below to find out

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.