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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy (HORIZON Trial)

Phase 1

Recruiting

Research Sponsored by Sarepta Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein with exception of a mutation in exon 8 and/or 9

Ability to cooperate with motor assessment testing

Must not have

Has reduced left ventricular ejection fraction on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a gene transfer therapy to see if it is safe and effective in boys with Duchenne muscular dystrophy who have antibodies to a specific virus. The study will last for 58 weeks

Who is the study for?

This trial is for ambulatory male participants with Duchenne Muscular Dystrophy (DMD) who already have antibodies to AAVrh74. The study spans 58 weeks and involves a gene transfer therapy followed by plasmapheresis, a process that filters the blood.

What is being tested?

The trial tests delandistrogene moxeparvovec's safety and its ability to express dystrophin protein in patients with DMD after they undergo therapeutic plasma exchange (plasmapheresis).

What are the potential side effects?

Potential side effects may include reactions related to the infusion of delandistrogene moxeparvovec, changes in blood components due to plasmapheresis, or immune responses against the introduced gene.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My genetic test shows a specific mutation in the DMD gene, not including exon 8 or 9.

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I can participate in tests that assess my muscle movements.

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I have been officially diagnosed with Duchenne Muscular Dystrophy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My heart's pumping ability is lower than normal.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

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: Delandistrogene Moxeparvovec After Plasmapheresis ProcedureExperimental Treatment2 Interventions

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1 after plasmapheresis procedure if AAVrh74 antibodies are sufficiently low.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Plasmapheresis

2016

Completed Phase 3

~390

delandistrogene moxeparvovec

2018

Completed Phase 2

~50