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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy

Q: How many enrollees are currently part of this clinical investigation?

Indeed, per the information on clinicaltrials.gov this trial is presently enrolling qualified individuals. It was first published on March 13th 2023 and subsequently amended June 1st of that same year. A total of 250 volunteers are wanted from 2 separate sites for participation in the study.

Q: Has this therapy been given the green light by the FDA?

We rate the safety of this intervention as a 3 due to evidence acquired from Phase 3 trials that indicate both efficacy and multiple rounds of supporting data.

Q: Is recruitment currently active for this study?

Affirmative. Clinicaltrials.gov lists this medical trial, which first appeared on March 13th 2023, as currently recruiting participants. The study aims to recruit 250 patients from 2 separate sites.

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 at least annually from 5 through 10 years after dosing in the interventional study

Awards & highlights

Study Summary

This trial looks at the safety and effects of an experimental gene therapy. Participants who have taken part in a previous Pfizer study are invited to take part and be monitored for 10 years. Participants will have 1 annual onsite visit and some remote visits with their doctor.

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.Check my eligibility

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.See study design

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 ~ at least annually from 5 through 10 years after dosing in the interventional study.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at least annually from 5 through 10 years after dosing in the interventional study.

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

Treatment Details

Study Objectives

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

Primary outcome measures

Number of participants with adverse events considered related to treatment

Number of participants with clinically significant findings in cardiac troponin I laboratory examinations

Number of participants with clinically significant findings in echocardiogram parameters

+5 more

Secondary outcome measures

Age at death

Age at loss of ambulation

Age when percent predicted forced vital capacity <30%

+10 more

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

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,570 Previous Clinical Trials

10,915,776 Total Patients Enrolled

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,476 Previous Clinical Trials

8,091,971 Total Patients Enrolled

Media Library

fordadistrogene movaparvovec (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05689164 — Phase 3

Duchenne Muscular Dystrophy Clinical Trial 2023: fordadistrogene movaparvovec Highlights & Side Effects. Trial Name: NCT05689164 — Phase 3

Frequently Asked Questions

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

How many enrollees are currently part of this clinical investigation?

"Indeed, per the information on clinicaltrials.gov this trial is presently enrolling qualified individuals. It was first published on March 13th 2023 and subsequently amended June 1st of that same year. A total of 250 volunteers are wanted from 2 separate sites for participation in the study."

Answered by AI

Has this therapy been given the green light by the FDA?

"We rate the safety of this intervention as a 3 due to evidence acquired from Phase 3 trials that indicate both efficacy and multiple rounds of supporting data."

Answered by AI

Is recruitment currently active for this study?

"Affirmative. Clinicaltrials.gov lists this medical trial, which first appeared on March 13th 2023, as currently recruiting participants. The study aims to recruit 250 patients from 2 separate sites."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

2023. "A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05689164.

All > Muscular Dystrophy > Experimental