Duchenne Muscular DystrophyPhase 1/2NCT06138639
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
A Phase 1/2 study testing SGT-003 in people with Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 15 sites in 4 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 0 to 17 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- SGT-003
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 15 sitesin 4 countries
- Study dates
- 6 May 2024to 6 May 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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:
Show all 18Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
15 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
15 sites in 4 countries
United States12 sites, 12 recruiting
- Recruiting
Rare Disease Research
Atlanta, Georgia, 30329
678-883-6897, subjectrecruiting@rarediseaseresearch.com
Renata Shih, MD
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605
Alka Maheshwari, 312-227-3813, amaheshwari@luriechildrens.org
Nancy Kuntz, MD
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43215
Destany McCain, 614-722-6961, dbs001@nationwidechildrens.org
Kevin Flanigan, MD
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Amber Kellogg, 501-364-3389, aveerapandiyan@uams.edu
Aravindhan Veerapandiyan, MD
- Recruiting
University of California, Los Angeles Medical Center
Los Angeles, California, 90095
Ummi Qasim, 310-825-3264, UQasim@mednet.ucla.edu
Perry Shieh, MD, PhD
- Recruiting
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
E.Ann Walker, 757-668-6543, Proud.Research@CHKD.org
Crystal Proud, MD
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Brandt Parlanti, 215-850-7769, parlantib@chop.edu
John Brandsema, MD
- Recruiting
Oregon Health and Sciences University
Portland, Oregon, 97239
Beata Dyar, 503-494-8216, dyar@ohsu.edu
Erika Finanger, MD
- Recruiting
University of California, Davis
Sacramento, California, 95817
Neuromuscular Research Lab, 916-734-5057, NMRL@ucdavis.edu
Craig McDonald, MD
- Recruiting
University of California
San Diego, California, 92037
Gabriella Penner, 858-382-3061, gpenner@health.ucsd.edu
Chamindra Laverty, MD
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Janaki O'Brien, 206-987-1642, Janaki.OBrien@seattlechildrens.org
Alicia Henriquez, MD
- Recruiting
Washington University in St. Louis
St Louis, Missouri, 63110
Natalie Goedeker, 314-362-4919, neuromusclepediatricresearch@wustl.edu
Craig Zaidman, MD
Canada1 site, 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 0A4
Ana Stosic, 416-648-6831, Ana.Stosic@SickKids.ca
Hernan Gonorazky, MD
Italy1 site, 1 recruiting
- Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, 00168
Lorenzo Stivala, +39 063-015-6330, lorenzo.stivala@policlinicogemelli.it
Eugenio Mercuri, MD, PhD
United Kingdom1 site, 1 recruiting
- Recruiting
Great Ormond Street Hospital
London, WC1N 3JH
Lucinda Furtado, +44 (0)20 7762 6932, Lucinda.furtado@gosh.nhs.uk
Francesco Muntoni, MD
About this study
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 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- SGT-003
- Keywords
- DMD, Gene Therapy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SGT-003Genetic
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: SGT-003 | Experimental | All ambulatory participants from age 4 to < 7 years will receive a single IV infusion of SGT-003 on Day 1. Receives: Genetic: SGT-003 |
| Cohort 2: SGT-003 | Experimental | All ambulatory participants from age 7 to < 12 years will receive a single IV infusion of SGT-003 on Day 1. Receives: Genetic: SGT-003 |
| Cohort 3: SGT-003 | Experimental | All participants from age 0 to < 4 years will receive a single IV infusion of SGT-003 on Day 1. Receives: Genetic: SGT-003 |
| Cohort 4: SGT-003 | Experimental | All ambulatory participants from age 12 to < 18 years will receive a single IV infusion of SGT-003 on Day 1. Receives: Genetic: SGT-003 |
| Cohort 5: SGT-003 | Experimental | All non-ambulatory participants from age 10 to < 18 years will receive a single IV infusion of SGT-003 on Day 1. Receives: Genetic: SGT-003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (AEs)
Measured: Day 360
Change from baseline in Microdystrophin Protein Levels
Microdystrophin expression evaluation in muscle biopsies
Measured: Day 90
Secondary outcomes (12)
Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF)
Measured: Day 90, Day 360
Change from baseline in Microdystrophin Protein Levels
Microdystrophin expression evaluation in muscle biopsies
Measured: 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.
Measured: Day 360, Day 540
Change from baseline in Stride Velocity 95th Centile (SV95C)
Assessment of peak ambulatory performance captured by wearable activity monitoring device.
Measured: 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.
Measured: Day 360, Day 540
Change from baseline in 4-stair climb velocity
Measured: 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.
Measured: Day 360, Day 540
Change from baseline in 6-minute walk test (6MWT) distance
Measured: Day 360, Day 540
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters
Measured: Through Day 360 and Day 540
Number of Participants with Clinically Significant Abnormalities in Vital Signs
Measured: Through Day 360 and Day 540
Number of Participants with Clinically Significant Abnormalities in Physical Examinations
Measured: Through Day 360 and Day 540
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) or Echocardiography (ECHO)
Measured: Through Day 360 and Day 540
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2023
- Study start6 May 2024
- Primary completion6 May 2027 (estimated)
- Study completion6 May 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Solid Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Solid Bio Clinical Trials, Study director, Solid Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06138639
- Sponsor's protocol ID
- SGT-003-101
- Other IDs
- 2024-514501-57-00, 1010251
- Status last verified
- Jul 2026
- First submitted
- 14 Nov 2023
- Last updated
- 8 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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 Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
- ClinicalTrials.gov ID
- NCT06138639
- Acronym / sponsor ID
- SGT-003-101
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Solid Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 6 May 2024 (actual)
- Primary completion
- 6 May 2027 (estimated)
- Study completion
- 6 May 2031 (estimated)
- First posted
- 18 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: SGT-003 | Experimental | Genetic: SGT-003All ambulatory participants from age 4 to < 7 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 2: SGT-003 | Experimental | Genetic: SGT-003All ambulatory participants from age 7 to < 12 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 3: SGT-003 | Experimental | Genetic: SGT-003All participants from age 0 to < 4 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 4: SGT-003 | Experimental | Genetic: SGT-003All ambulatory participants from age 12 to < 18 years will receive a single IV infusion of SGT-003 on Day 1. |
| Cohort 5: SGT-003 | Experimental | Genetic: SGT-003All non-ambulatory participants from age 10 to < 18 years will receive a single IV infusion of SGT-003 on Day 1. |
Who may qualify
- Ages
- 0 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (18 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SGT-003Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5) | Genetic | Cohort 1: SGT-003, Cohort 2: SGT-003, Cohort 3: SGT-003, Cohort 4: SGT-003, Cohort 5: SGT-003 |
Primary outcome
Incidence of treatment-emergent adverse events (AEs)
Time frame: Day 360
Change from baseline in Microdystrophin Protein Levels
Time frame: Day 90
Microdystrophin expression evaluation in muscle biopsies
12 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
Time frame: Day 360
Change from Baseline in Time to Rise Velocity
Time frame: Day 360, Day 540
Change from baseline in Stride Velocity 95th Centile (SV95C)
Time frame: Day 360, Day 540
Change from baseline in 10-meter walk/run velocity
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Solid Bio Clinical Trials study director
Solid Biosciences
15 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SGT-003 in people with Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 15 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Canada, Italy, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Solid Bio Clinical Trials (617-337-4680, clinicaltrials@solidbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (AEs) (time frame: Day 360). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2026.