Duchenne Muscular DystrophyPhase 3NCT07160634
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
A Phase 3 study testing SGT-003 in people with Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 7 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 7 to 11 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?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 7 sitesin 3 countries
- Study dates
- 22 Oct 2025to Jan 2034
- Phase
- Phase 3
- 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 7 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Participant is ambulatory.
- Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
- Negative for antibodies against adeno-associated virus serotype 9 (AAV9).
- 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.
Who cannot join
- 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.
Trial locations and contacts
7 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 3 countries
United States3 sites, 3 recruiting
- Not yet recruiting
Neurology Rare Disease Center
Flower Mound, Texas, 75082
Jennifer Avelar, CRC, Research@neuromdcenter.com
- Not yet recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Amber Kellogg, RN, KelloggA@archildrens.org
- Not yet recruiting
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
Jennifer Beachum, BSHS, Proud.research@chkd.org
Canada3 sites, 3 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Israt Yasmeen, 587-943-0851, Israt.Yasmeen@ahs.ca
Jean Mah, MD
- Recruiting
The Hospital for Sick Children (SickKids)
Toronto, Ontario
Ana Stosic, 416-648-6831, Ana.Stosic@SickKids.ca
Hernan Gonorazky, MD
- Recruiting
BC Children's Hospital
Vancouver, British Columbia
Nela Martic, 604-875-2345 ext. 6549, nmartic3@cw.bc.ca
Kathryn Selby, MD
Australia1 site, 1 recruiting
- Recruiting
The Children's Hospital of Westmead
Sydney, New South Wales
CHW CRC clinical trials team, (02) 7825 1387, SCHN-ClinicalTrials@health.nsw.gov.au
Michelle Lorentzos, MD
About this study
Official title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- SGT-003, Placebo
- Keywords
- SGT-003, Duchenne Muscular Dystrophy (DMD), adeno-associated virus (AAV), IMPACT DUCHENNE
- 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-003Drug
Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
PlaceboDrug
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| SGT-003 in Part 1, Placebo in Part 2 | Experimental | Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria. Receives: Drug: SGT-003; Drug: Placebo |
| Placebo in Part 1, SGT-003 in Part 2 | Experimental | 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 per protocol-defined eligibility criteria. Receives: Drug: SGT-003; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540
Measured: Baseline, Day 540
Secondary outcomes (15)
Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540
Measured: Baseline, Day 540
Change From Baseline in 4-Stair Climb (4SC) Velocity (Tasks/s) at Day 540
Measured: Baseline, Day 540
Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540
Measured: Baseline, Day 540
Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540
Measured: Baseline, Day 540
Cumulative Loss of Function in NSAA Items at Day 540
Measured: At Day 540
Change from Baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540
Measured: Baseline, Day 540
Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540
Measured: Baseline, Day 540
Change from Baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540
Measured: Baseline, Day 540
Change from Baseline in Left Ventricular Ejection Fraction (LVEF) at Day 540[
Measured: Baseline, Day 540
Change from Baseline in Global Longitudinal Strain (GLS) at Day 540
Measured: Baseline, Day 540
Change from Baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540
Measured: Baseline, Day 540
Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of special interest (AESIs), and clinically significant changes in Electrocardiogram (ECG) and Echocardiography (ECHO)
Measured: From first dose up to Day 540
Change From Baseline in Viral Shedding as Measured by Polymerase Chain Reaction (PCR) at Day 360
Measured: Baseline, Day 360
Change From Baseline in Anti-Adeno-Associated Virus (AAV) Antibody Titers at Day 540
Measured: Baseline, Day 540
Change from Baseline in Microdystrophin T-cell Reactivity by Enzyme-Linked Immunospot at Day 540
Measured: Baseline, Day 540
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
- Study start22 Oct 2025
- Primary completionJan 2029 (estimated)
- Study completionJan 2034 (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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Solid Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07160634
- Sponsor's protocol ID
- SGT-003-301
- Other IDs
- 2025-522949-22, 1013075, 2025
- Status last verified
- Sep 2026
- First submitted
- 29 Aug 2025
- Last updated
- 28 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT07160634
- Acronym / sponsor ID
- SGT-003-301
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- SGT-003, Duchenne Muscular Dystrophy (DMD), adeno-associated virus (AAV), IMPACT DUCHENNE
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Solid Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Oct 2025 (actual)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jan 2034 (estimated)
- First posted
- 8 Sep 2025 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| SGT-003 in Part 1, Placebo in Part 2 | Experimental | Drug: SGT-003, Drug: PlaceboEnrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria. |
| Placebo in Part 1, SGT-003 in Part 2 | Experimental | Drug: SGT-003, Drug: PlaceboEnrolled 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 per protocol-defined eligibility criteria. |
Who may qualify
- Ages
- 7 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Participant is ambulatory.
- Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
- Negative for antibodies against adeno-associated virus serotype 9 (AAV9).
- 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.
Key exclusion criteria (4 listed)
- 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.
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 (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5) | Drug | Placebo in Part 1, SGT-003 in Part 2, SGT-003 in Part 1, Placebo in Part 2 |
| PlaceboIV infusion | Drug | Placebo in Part 1, SGT-003 in Part 2, SGT-003 in Part 1, Placebo in Part 2 |
Primary outcome
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540
Time frame: Baseline, Day 540
15 secondary outcomes
Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540
Time frame: Baseline, Day 540
Change From Baseline in 4-Stair Climb (4SC) Velocity (Tasks/s) at Day 540
Time frame: Baseline, Day 540
Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540
Time frame: Baseline, Day 540
Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540
Time frame: Baseline, Day 540
Cumulative Loss of Function in NSAA Items at Day 540
Time frame: At Day 540
Change from Baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540
Time frame: Baseline, Day 540
Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540
Time frame: Baseline, Day 540
Change from Baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540
Time frame: Baseline, Day 540
Change from Baseline in Left Ventricular Ejection Fraction (LVEF) at Day 540[
Time frame: Baseline, Day 540
Change from Baseline in Global Longitudinal Strain (GLS) at Day 540
Time frame: Baseline, Day 540
Change from Baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540
Time frame: Baseline, Day 540
Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of special interest (AESIs), and clinically significant changes in Electrocardiogram (ECG) and Echocardiography (ECHO)
Time frame: From first dose up to Day 540
Change From Baseline in Viral Shedding as Measured by Polymerase Chain Reaction (PCR) at Day 360
Time frame: Baseline, Day 360
Change From Baseline in Anti-Adeno-Associated Virus (AAV) Antibody Titers at Day 540
Time frame: Baseline, Day 540
Change from Baseline in Microdystrophin T-cell Reactivity by Enzyme-Linked Immunospot at Day 540
Time frame: Baseline, 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 28 Sep 2026.
- Individual participant dataSharing planned: No
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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.
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SGT-003 in people with Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 7 sites in 3 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 7 inclusion and 4 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 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Canada, Australia. 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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 (6173374680, clinicaltrials@solidbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 (time frame: Baseline, Day 540). 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 28 Sep 2026.