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

A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

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 Overview

• Status: Recruiting
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Genetic: SGT-003

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Solid Bio Clinical Trials; Phone Number: 617-337-4680; Email: clinicaltrials@solidbio.com

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 0A4
      • Recruiting
      • The Hospital for Sick Children
      • Principal Investigator: Hernan Gonorazky, MD
      • Contact: Ana Stosic; Phone Number: 416-648-6831; Email: Ana.Stosic@SickKids.ca
• Italy
  • Rome, Italy, 00168
    • Recruiting
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
    • Contact: Lorenzo Stivala; Phone Number: +39 063-015-6330; Email: lorenzo.stivala@policlinicogemelli.it
    • Principal Investigator: Eugenio Mercuri, MD, PhD
• United Kingdom
  • London, United Kingdom, WC1N 3JH
    • Recruiting
    • Great Ormond Street Hospital
    • Principal Investigator: Francesco Muntoni, MD
    • Contact: Lucinda Furtado; Phone Number: +44 (0)20 7762 6932; Email: Lucinda.furtado@gosh.nhs.uk
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Recruiting
      • Arkansas Children's Hospital
      • Principal Investigator: Aravindhan Veerapandiyan, MD
      • Contact: Amber Kellogg; Phone Number: 501-364-3389; Email: aveerapandiyan@uams.edu
  • California
    • Los Angeles, California, United States, 90095
      • Recruiting
      • University of California, Los Angeles Medical Center
      • Contact: Merve Kaleli; Phone Number: 310-825-3264; Email: mkaleli@mednet.ucla.edu
      • Principal Investigator: Perry Shieh, MD, PhD
    • Sacramento, California, United States, 95817
      • Recruiting
      • University of California, Davis
      • Principal Investigator: Craig McDonald, MD
      • Contact: Neuromuscular Research Lab; Phone Number: 916-734-5057; Email: NMRL@ucdavis.edu
    • San Diego, California, United States, 92037
      • Recruiting
      • University of California
      • Principal Investigator: Chamindra Laverty, MD
      • Contact: Gabriella Penner; Phone Number: 858-382-3061; Email: gpenner@health.ucsd.edu
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Recruiting
      • Rare Disease Research
      • Principal Investigator: Renata Shih, MD
      • Contact: Neona Ni; Phone Number: 470-666-1884; Email: neona.ni@rarediseaseresearch.com
  • Illinois
    • Chicago, Illinois, United States, 60611-2605
      • Recruiting
      • Ann & Robert H. Lurie Children's Hospital of Chicago
      • Contact: Alka Maheshwari; Phone Number: 312-227-3813; Email: amaheshwari@luriechildrens.org
      • Contact: Nancy Kuntz, MD
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Recruiting
      • Washington University in St. Louis
      • Principal Investigator: Craig Zaidman, MD
      • Contact: Natalie Goedeker; Phone Number: 314-362-4919; Email: NeuromusclePediatricResearch@wustl.edu
  • Ohio
    • Columbus, Ohio, United States, 43215
      • Recruiting
      • Nationwide Children's Hospital
      • Principal Investigator: Kevin Flanigan, MD
      • Contact: Destany McCain; Phone Number: 614-722-6961; Email: dbs001@nationwidechildrens.org
  • Oregon
    • Portland, Oregon, United States, 97239
      • Recruiting
      • Oregon Health and Sciences University
      • Principal Investigator: Erika Finanger, MD
      • Contact: Beata Dyar; Phone Number: 503-494-8216; Email: dyar@ohsu.edu
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Principal Investigator: John Brandsema, MD
      • Contact: Brandt Parlanti; Phone Number: 215-850-7769; Email: parlantib@chop.edu
  • Virginia
    • Norfolk, Virginia, United States, 23510
      • Recruiting
      • Children's Hospital of The King's Daughters
      • Principal Investigator: Crystal Proud, MD
      • Contact: E.Ann Walker; Phone Number: 757-668-6543; Email: Proud.Research@CHKD.org
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Seattle Children's Hospital
      • Contact: Janaki O'Brien; Phone Number: 206-987-1642; Email: Janaki.OBrien@seattlechildrens.org
      • Principal Investigator: Alicia Henriquez, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: SGT-003; All ambulatory participants from age 4 to < 7 years 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 ambulatory participants from age 7 to < 12 years 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 3: SGT-003; All participants from age 0 to < 4 years 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 4: SGT-003; All ambulatory participants from age 12 to < 18 years 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 5: SGT-003; All non-ambulatory participants from age 10 to < 18 years 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events (AEs)		Day 360
Change from baseline in Microdystrophin Protein Levels	Microdystrophin expression evaluation in muscle biopsies	Day 90

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF)		Day 90, Day 360
Change from baseline in 4-stair climb velocity		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.	Day 360, Day 540
Change from baseline in 6-minute walk test (6MWT) distance		Day 360, Day 540
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters		Through Day 360 and Day 540
Number of Participants with Clinically Significant Abnormalities in Vital Signs		Through Day 360 and Day 540
Number of Participants with Clinically Significant Abnormalities in Physical Examinations		Through Day 360 and Day 540
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) or Echocardiography (ECHO)		Through Day 360 and Day 540
Change from baseline in Microdystrophin Protein Levels	Microdystrophin expression evaluation in muscle biopsies	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.	Day 360, Day 540
Change from baseline in Stride Velocity 95th Centile (SV95C)	Assessment of peak ambulatory performance captured by wearable activity monitoring device.	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.	Day 360, Day 540

Collaborators and Investigators

• Sponsor: Solid Biosciences Inc.
• Investigators: Study Director: Solid Bio Clinical Trials, Solid Biosciences

Study record dates

Study Major Dates

• Study Start (Actual): May 6, 2024
• Primary Completion (Estimated): May 6, 2027
• Study Completion (Estimated): May 6, 2031

Study Registration Dates

• First Submitted: November 14, 2023
• First Submitted That Met QC Criteria: November 14, 2023
• First Posted (Actual): November 18, 2023

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 6, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Gene Therapy; DMD
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: SGT-003-101; 2024-514501-57-00 (Ctis); 1010251 (Other Identifier: UK IRAS ID)

Drug and device information, study documents

• 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