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Purpose

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.

Condition

Eligibility

Eligible Ages
Between 7 Years and 11 Years
Eligible Sex
Male
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Participant is ambulatory. - Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype. - Negative for antibodies against adeno-associated virus. - On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice. - Meet 10-meter walk/run time criteria. - Meet time to rise from supine criteria. - Participant has bodyweight ≤50 kg.

Exclusion Criteria

  • Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy. - Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment. - Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. Other Inclusion/Exclusion criteria to be applied as per protocol.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
SGT-003 followed by Placebo 		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.
  • Drug: SGT-003
    Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
  • Drug: Placebo
    IV infusion
Experimental
Placebo followed by SGT-003 		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.
  • Drug: SGT-003
    Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
  • Drug: Placebo
    IV infusion

More Details

Status
Recruiting
Sponsor
Solid Biosciences Inc.

Study Contact

Solid Bio Clinical Trials
6173374680
clinicaltrials@solidbio.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.