SGT-003 Gene Therapy for Duchenne Muscular Dystrophy

Overseen By Solid Bio Clinical Trials

Age: < 18

Sex: Male

Trial Phase: Phase 3

Sponsor: Solid Biosciences Inc.

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.

Are You a Good Fit for This Trial?

This trial is for ambulant males with Duchenne Muscular Dystrophy (DMD) who can walk, have a confirmed diagnosis and specific gene mutation, are on stable steroids for at least 6 months, can rise from lying down within a set time, weigh ≤50 kg, and don't have antibodies against adeno-associated virus.

Inclusion Criteria

I have been diagnosed with DMD and have a gene mutation linked to it.

I don't have antibodies against adeno-associated virus.

I have been on a stable dose of prednisone or deflazacort for at least 6 months.

What Are the Treatments Tested in This Trial?

Interventions

• SGT-003

Trial Overview The study tests SGT-003 gene therapy's effectiveness in improving DMD symptoms. Participants will receive either SGT-003 or placebo via IV infusion in a randomized order across two parts of the trial. They'll be monitored for at least five years after dosing.

How Is the Trial Designed?

Group I: SGT-003 followed by Placebo Experimental Treatment 2 Interventions

Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2.

Group II: Placebo followed by SGT-003 Experimental Treatment 2 Interventions

Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2.

Who Is Running the Clinical Trial?

Solid Biosciences Inc.

Lead Sponsor

Trials

4

Recruited

130+