A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Solid Biosciences Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Decision date (initial)

2026-05-28

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Solid Biosciences Inc.

External identifiers

EU CT number

2025-522949-22-00

ClinicalTrials.gov

NCT07160634

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing change in muscle function.

Secondary objectives 6

1. To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing changes in muscle function and strength
2. To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing microdystrophin expression in muscle biopsies
3. To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing changes in respiratory function
4. To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing changes in patient-reported outcome measures
5. To investigate the safety and tolerability of a single intravenous dose of SGT-003
6. To investigate the efficacy of a single IV dose of SGT-003, compared to placebo, by assessing additional changes in muscle function and strength.

Conditions and MedDRA coding

Duchenne muscular dystrophy

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-000024-PIP97-84

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Participant 7 to <12 years of age.
2. Participant is ambulatory. Ambulatory is defined as “being able to walk without the use of an assistive device.”
3. Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
4. Negative for AAV antibodies.
5. On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 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.
6. Meet 10-meter walk/run time criteria.
7. Meet time to rise from supine criteria.
8. Participant has bodyweight ≤50 kg
9. Participant is genetically male.
10. Participant is able to understand and comply with all study procedures as appropriate by age and has a parent(s) or legal guardian(s) (i.e., legally authorized representative [LAR]) who is (are) able to understand and comply with the study procedure requirements. Be willing to provide informed assent and have an LAR(s) who is (are) willing to provide written informed consent for the participant to participate in the study.
11. If participant is of reproductive potential, participant and partner of childbearing potential are willing to use 2 highly effective forms of contraception for 12 months following study drug administration.

Exclusion criteria 13

1. Prior or ongoing medical condition, medical history, or physical finding that in the Investigator's opinion could adversely affect the safety of the participant, make it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
2. Major surgery within 3 months prior to recruitment or planned surgery any time during this study that would have the potential to interfere with the ability or performance on outcome measures.
3. 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.
4. Sponsor employees and their family members are ineligible to participate in this study.
5. Known liver disease, evidence of active viral hepatitis, or abnormal liver function.
6. Abnormal renal function
7. Clinically significant abnormalities of coagulation
8. Impaired cardiovascular function
9. Pulmonary function predictive of or requiring the use of daytime ventilatory support outside of acute illnesses.
10. History of severe hypersensitivity reactions, including anaphylaxis, to the product or its components.
11. Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.
12. 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.
13. Any active infection.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in time to rise velocity at Day 540

Secondary endpoints 16

1. Change from baseline in stride velocity 95th centile (SV95C) by activity monitoring using the Syde wearable device at Day 540
2. Change from baseline in 10-meter walk/run velocity at Day 540
3. Change from baseline in 4-stair climb velocity at Day 540
4. Change from baseline in absolute NSAA score at Day 540
5. Cumulative loss of function in North Star Ambulatory Assessment (NSAA) items at Day 540
6. Change from baseline in microdystrophin protein levels at Day 90
7. Change from baseline in microdystrophin tissue distribution at Day 90
8. Change from baseline in % predicted forced vital capacity (FVC) at Day 540
9. Change from baseline in % predicted peak expiratory flow (PEF) at Day 540
10. Change from baseline in % predicted forced expiratory volume in 1 second (FEV1) at Day 540
11. Incidence of treatment-emergent adverse events through Day 540
12. Incidence of serious adverse events through Day 540
13. Incidence of adverse events of special interest through Day 540
14. Incidence of clinically significant changes in ECGs through Day 540
15. Incidence of clinically significant changes in ECHOs through Day 540
16. Change from baseline in the PODCI Global score at Day 540.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Solid Biosciences Inc.

Sponsor organisation

Solid Biosciences Inc.

Address

500 Rutherford Avenue

City

Charlestown

Postcode

02129-1647

Country

United States

Scientific contact point

Organisation

Solid Biosciences Inc.

Contact name

Amber Conklin

Public contact point

Organisation

Solid Biosciences Inc.

Contact name

Amber Conklin

Third parties 22

Locations

6 EU/EEA countries · 12 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 101 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications