A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Phase 2RecruitingNCT06138639

Solid Biosciences Inc.60 enrolled

Overview

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

Eligibility

Sex: MALEMin age: 0 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Cohort 1: 4 to \<7 years of age * Cohort 2: 7 to \<12 years of age * Cohort 3: 0 to \< 4 years of age * Cohort 4: 12 to \< 18 years of age * Cohort 5: 10 to \< 18 years of age * Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in \< 30 seconds: * Cohorts 1, 2, and 4: Ambulatory * Cohort 3: Either ambulatory or non-ambulatory * Cohort 5: Non-ambulatory, but having been previously ambulatory by history * Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion. * Negative for AAV antibodies. * Steroid regimen: * Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice. * Cohort 3: N/A * Meet 10-meter walk/run time criteria * Meet time to rise from supine criteria * Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria * Participant has body weight: ≤ 90 kg Exclusion Criteria: * Treatment with dystrophin modifying drugs within 3 months prior to screening. * Current or prior treatment with an approved or investigational gene transfer drug. * Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer. * Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing. Other inclusion or exclusion criteria apply.

Locations (15)

Arkansas Children's Hospital

Little Rock, Arkansas, United States

RECRUITING

University of California, Los Angeles Medical Center

Los Angeles, California, United States

RECRUITING

University of California, Davis

Sacramento, California, United States

RECRUITING

University of California

San Diego, California, United States

RECRUITING

Rare Disease Research

Atlanta, Georgia, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

RECRUITING

Washington University in St. Louis

St Louis, Missouri, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

RECRUITING

Oregon Health and Sciences University

Portland, Oregon, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

RECRUITING

...and 5 more locations

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events (AEs)

Time frame: Day 360

Change from baseline in Microdystrophin Protein Levels

Microdystrophin expression evaluation in muscle biopsies

Time frame: Day 90

Secondary Outcomes

Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF)

Time frame: Day 90, Day 360

Change from baseline in Microdystrophin Protein Levels

Microdystrophin expression evaluation in muscle biopsies

Time frame: Day 360

Change from Baseline in Time to Rise Velocity

The Time to rise from supine (TTR) (s) will be captured as part of item 12 of the North Star Ambulatory Assessment (NSAA). Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.

Time frame: Day 360, Day 540

Change from baseline in Stride Velocity 95th Centile (SV95C)

Assessment of peak ambulatory performance captured by wearable activity monitoring device.

Time frame: Day 360, Day 540

Change from baseline in 10-meter walk/run velocity

The 10MWR (s) will be captured as part of item 17 of the NSAA. Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.

Time frame: Day 360, Day 540

Change from baseline in 4-stair climb velocity

Time frame: Day 360, Day 540

Change from baseline in North Star Ambulatory Assessment (NSAA) total score

Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.

Time frame: Day 360, Day 540

Change from baseline in 6-minute walk test (6MWT) distance

Time frame: Day 360, Day 540

Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters

Time frame: Through Day 360 and Day 540

Number of Participants with Clinically Significant Abnormalities in Vital Signs

Time frame: Through Day 360 and Day 540

Number of Participants with Clinically Significant Abnormalities in Physical Examinations

Time frame: Through Day 360 and Day 540

Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) or Echocardiography (ECHO)

Time frame: Through Day 360 and Day 540

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Overview

Conditions

Interventions

Eligibility

Locations (15)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts