Duchenne Muscular DystrophyPhase 1/2IGNITE DMDNCT03368742
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
A Phase 1/2 study testing SGT-001 in people with Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 2 sites in United States.
Trial summary
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. Read more about this study
- Who is it for?
- Ages 4 to 17 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- SGT-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 6 Dec 2017to 30 Nov 2026
- 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 4 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
- Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
- Anti-AAV9 antibodies below protocol-specified thresholds
- Stable cardiac and pulmonary function
- Adolescents: non-ambulatory by protocol-specified criteria
- Children: ambulatory by protocol-specified criteria
- Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks
Who cannot join
- Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
- Abnormal liver function
- Abnormal renal function
- Clinically significant coagulation abnormalities
- Impaired cardiovascular function based on cardiac MRI or ECHO
- Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
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- Significant spinal deformity or presence of spinal rods
- Body mass index ≥ 95th percentile for age
- Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
- Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening
- Additional inclusion/exclusion criteria may apply.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Florida
Gainesville, Florida, 32610
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095
About this study
Official title: A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.
The protocol was amended to drop the control arm after 4 participants were dosed.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- SGT-001
- 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-001Genetic
AAV9 vector containing muscle-specific promoter and microdystrophin construct
| Study group | Role | What they receive |
|---|---|---|
| SGT-001 - Dose Level 1 | Experimental | Single IV infusion of SGT-001 at starting dose Receives: Genetic: SGT-001 |
| SGT-001 - Dose Level 2 | Experimental | Single IV infusion of SGT-001 at next ascending dose Receives: Genetic: SGT-001 |
| Untreated Control | No intervention | Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Measured: Up to 5 years
Secondary outcomes (14)
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters
Measured: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Vital Signs
Measured: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Physical Examinations
Measured: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)
Measured: Up to 5 years
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)
Measured: Baseline, 12 months
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)
Measured: Baseline, 12 months
Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants
Measured: Baseline, 12 months
Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants
Measured: Baseline, 12 months
Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score
Measured: Baseline, 12 months
Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted
Measured: Baseline, 12 months
Change from Baseline in Ejection Fraction, As Measured by Echocardiography
Measured: Baseline,12 months
Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography
Measured: Baseline,12 months
Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography
Measured: Baseline,12 months
Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module
Measured: Baseline, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2017
- Study start6 Dec 2017
- Primary completion30 Nov 2026 (estimated)
- Study completion30 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
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
- NCT03368742
- Sponsor's protocol ID
- GX1001
- Acronym
- IGNITE DMD
- Status last verified
- Oct 2026
- First submitted
- 5 Dec 2017
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Boehler JF, Brown KJ, Ricotti V, Morris CA. N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy. Skelet Muscle. 2024 Jan 16;14(1):2. doi: 10.1186/s13395-023-00334-y.
PubMed 38229112
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03368742
- Acronym / sponsor ID
- IGNITE DMD / GX1001
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Solid Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Dec 2017 (actual)
- Primary completion
- 30 Nov 2026 (estimated)
- Study completion
- 30 Nov 2026 (estimated)
- First posted
- 11 Dec 2017 (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 controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.
The protocol was amended to drop the control arm after 4 participants were dosed.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SGT-001 - Dose Level 1 | Experimental | Genetic: SGT-001Single IV infusion of SGT-001 at starting dose |
| SGT-001 - Dose Level 2 | Experimental | Genetic: SGT-001Single IV infusion of SGT-001 at next ascending dose |
| Untreated Control | No intervention | –Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose. |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
- Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
- Anti-AAV9 antibodies below protocol-specified thresholds
- Stable cardiac and pulmonary function
- Adolescents: non-ambulatory by protocol-specified criteria
Key exclusion criteria (11 listed)
- Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
- Abnormal liver function
- Abnormal renal function
- Clinically significant coagulation abnormalities
- Impaired cardiovascular function based on cardiac MRI or ECHO
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-001AAV9 vector containing muscle-specific promoter and microdystrophin construct | Genetic | SGT-001 - Dose Level 1, SGT-001 - Dose Level 2 |
Primary outcome
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 5 years
14 secondary outcomes
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Vital Signs
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Physical Examinations
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)
Time frame: Up to 5 years
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)
Time frame: Baseline, 12 months
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)
Time frame: Baseline, 12 months
Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants
Time frame: Baseline, 12 months
Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants
Time frame: Baseline, 12 months
Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score
Time frame: Baseline, 12 months
Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted
Time frame: Baseline, 12 months
Change from Baseline in Ejection Fraction, As Measured by Echocardiography
Time frame: Baseline,12 months
Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography
Time frame: Baseline,12 months
Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography
Time frame: Baseline,12 months
Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module
Time frame: Baseline, 12 months
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 15 Oct 2026.
- Derived from this study: PubMed 38229112Boehler JF, Brown KJ, Ricotti V, Morris CA. N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy. Skelet Muscle. 2024 Jan 16;14(1):2. doi: 10.1186/s13395-023-00334-y.
- 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. This study is not recruiting at the moment.
Solid Bio Clinical Trials study director
Solid Biosciences
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SGT-001 in people with Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 2 sites in United States.
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 11 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: under way, not looking for participants, as of the sponsor's last update on 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Treatment Emergent Adverse Events (TEAEs) (time frame: Up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03368742, which also lists 1 linked publication.
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 15 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.