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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT06138639
Recruitment Status : Not yet recruiting
First Posted : November 18, 2023
Last Update Posted : November 18, 2023
Sponsor:
Information provided by (Responsible Party):
Solid Biosciences Inc.

Study Description
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Brief Summary:
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study, dosed sequentially. Cohort 1 will include participants 4 to <6 years of age, inclusive. Cohort 2 will only be opened after dosing and monitoring a subset of participants in Cohort 1. Cohort 2 will include participants 6 to <8 years of age, inclusive. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: SGT-003 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Estimated Study Start Date : January 15, 2024
Estimated Primary Completion Date : January 15, 2026
Estimated Study Completion Date : January 15, 2030

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1: SGT-003
All participants will receive a single IV infusion of SGT-003 on Day 1.
 Genetic: SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Experimental: Cohort 2: SGT-003
All participants will receive a single IV infusion of SGT-003 on Day 1.
 Genetic: SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (AEs) [ Time Frame: Day 360 ]

Secondary Outcome Measures :
  1. Change from baseline in microdystrophin protein levels [ Time Frame: Day 90, Day 360 ]
    Microdystrophin expression evaluation in muscle biopsies

  2. Change from baseline in North Star Ambulatory Assessment (NSAA) total score [ Time Frame: Day 360 ]
    Assessment of muscle function using a 17-item scale

  3. Change from baseline in stride velocity 95th centile (SV95C) [ Time Frame: Day 360 ]
    Assessment of peak ambulatory performance captured by wearable activity monitoring device


Eligibility Criteria
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Ages Eligible for Study:   4 Years to 7 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Cohort 1: 4 to <6 years of age, inclusive
  • Cohort 2: 6 to <8 years of age, inclusive
  • Ambulatory as defined as "being able to walk without the use of an assistive device."
  • Negative for AAV antibodies.
  • On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study.
  • Cohort 1: <18 kg body weight
  • Cohort 2: <30 kg body weight

Exclusion Criteria:

  • Current or prior treatment with approved or investigational dystrophin modifying drugs such as eteplirsen, golodirsen, casimersen, and viltolarsen.
  • Current or prior treatment with an approved or investigational gene transfer drug.
  • Exposure to another investigational drug 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 in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.

Other inclusion or exclusion criteria apply.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06138639


Contacts
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Contact: Solid Bio Clinical Trials 617-337-4680 clinicaltrials@solidbio.com

Locations
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United States, California
University of California, Los Angeles Medical Center
Los Angeles, California, United States, 90095
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43215
Sponsors and Collaborators
Solid Biosciences Inc.
Investigators
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Study Director: Solid Bio Clinical Trials Solid Biosciences
More Information
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Responsible Party: Solid Biosciences Inc.
ClinicalTrials.gov Identifier: NCT06138639    
Other Study ID Numbers: SGT-003-101
First Posted: November 18, 2023    Key Record Dates
Last Update Posted: November 18, 2023
Last Verified: November 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Solid Biosciences Inc.:
DMD
Gene Therapy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked