Gene Therapy for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+83 other locations

Age: < 18

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 tests a gene therapy treatment for boys aged 4 to 7 with muscle problems. The treatment aims to fix the genetic issues causing their muscle problems. Participants will receive the treatment and be monitored for safety and effectiveness. The treatment involves using a specific gene therapy, which has been researched for its potential to correct muscle issues in children with muscle problems.

Research Team

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

This trial is for boys with Duchenne muscular dystrophy (DMD) confirmed by genetic testing, who can walk and have been on a stable dose of steroids for at least 3 months. Boys with antibodies to AAV9, recent treatments increasing dystrophin expression, prior gene therapy, non-healed injuries affecting tests or certain genetic abnormalities in the dystrophin gene cannot participate.

Inclusion Criteria

I have been genetically tested and confirmed to have Duchenne muscular dystrophy.

I can walk and move around on my own.

I have been on a stable dose of steroids for at least 3 months.

Exclusion Criteria

I do not have any injuries that would affect my ability to perform physical tests.

Your blood, liver, or kidney tests show abnormal results.

I have never had gene therapy before.

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Treatment Details

Interventions

PF-06939926 (Gene Therapy)
Placebo (Other)

Trial OverviewThe study tests the safety and effectiveness of PF-06939926, a gene therapy for DMD. Participants are randomly assigned to receive either this gene therapy or a placebo in a blinded manner. Two-thirds will get the actual treatment while one-third receives placebo with an option to switch after one year.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment2 Interventions

Approximately one third of participants will be randomized to Cohort 2.

Group II: Cohort 1Experimental Treatment2 Interventions

Approximately two thirds of participants will be randomized to Cohort 1.