Gene Therapy for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

Overseen byMegan Waldrop, MD

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Megan Waldrop

No Placebo Group

Trial Summary

What is the purpose of this trial?

This trial uses a special virus to deliver genetic material to boys with Duchenne muscular dystrophy who have a specific gene issue. The treatment helps their bodies make a better version of an important muscle protein.

Research Team

Megan Waldrop, MD

Principal Investigator

Nationwide Children's Hospital

Eligibility Criteria

This trial is for boys aged 6 months to less than 14 years with Duchenne muscular dystrophy due to a specific genetic change (duplication of exon 2). Participants can be walking or not and must have been on stable corticosteroid therapy if over age 4. They cannot join if they have heart problems, certain infections like HIV or hepatitis, autoimmune diseases, abnormal blood counts, or high antibodies against the treatment vector.

Inclusion Criteria

I am between 6 months and 14 years old.

My test shows a specific genetic change in the DMD gene.

I can walk 10 meters without help or I cannot walk yet.

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Exclusion Criteria

Your test results are outside the healthy range according to the Nationwide Children's Hospital Laboratory.

You have high levels of AAV9 binding antibodies in your blood.

Your white blood cell count is too low or too high, or your neutrophil count is too low.

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Treatment Details

Interventions

scAAV9.U7.ACCA (Gene Therapy)

Trial OverviewThe trial tests a single dose of gene therapy called scAAV9.U7.ACCA delivered through an injection into a vein in the arm. It's designed for boys with Duchenne muscular dystrophy who have duplication of exon 2 in their genes. The study is open-label, meaning everyone knows what treatment is being given.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Cohort 1 (Minimal Efficacious Dose)Experimental Treatment1 Intervention

The Minimal Effective Dose (MED) will be delivered.