SGT-003 (human microdystrophin) for Duchenne muscular dystrophy

Inclusion criteria

• {"criterion_text":"- Participant age at the time of signing the informed consent form (ICF): Cohort 1: 4 to <7 years Cohort 2: 7 to <12 years Cohort 3: 0 to < 4 years Cohort 4: 12 to < 18 years Cohort 5: 10 to < 18 years"}
• {"criterion_text":"- Participant is male."}
• {"criterion_text":"- Able to understand and comply with all study procedures as appropriate by age and have 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"}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- Negative for AAV antibodies"}
• {"criterion_text":"- Steroid regimen: a. 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. b. Cohort 3: N/A"}
• {"criterion_text":"- Meet 10-meter walk/run time criteria"}
• {"criterion_text":"- Meet time to rise from supine criteria"}
• {"criterion_text":"- Cohort 5: meet Performance of Upper Limb (PUL) 2.0 criteria"}
• {"criterion_text":"- Participant has body weight ≤ 90 kg."}

Exclusion criteria

• {"criterion_text":"- Any prior or ongoing medical condition, medical history, 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."}
• {"criterion_text":"- Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer."}
• {"criterion_text":"- Major surgery within 3 months prior to recruitment or planned orthopedic surgery for any time during this study which would interfere with the ability to perform outcome measures."}
• {"criterion_text":"- Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted to not express exons 1 to 11, 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."}
• {"criterion_text":"- Sponsor employees and their family members are ineligible to participate in this study."}
• {"criterion_text":"- Any active infection"}
• {"criterion_text":"- Abnormal liver function, evidence of active viral hepatitis"}
• {"criterion_text":"- Abnormal renal function"}
• {"criterion_text":"- Clinically significant abnormalities of coagulation"}
• {"criterion_text":"- Impaired cardiovascular function"}
• {"criterion_text":"- Pulmonary function predictive of (or requiring) the use of daytime ventilatory support"}
• {"criterion_text":"- Have severe hypersensitivity reactions, including anaphylaxis, to SGT-003 or its components."}
• {"criterion_text":"- Treatment with dystrophin modifying drugs within 3 months prior to screening."}
• {"criterion_text":"- Current or prior treatment with an approved or investigational gene transfer drug."}

Primary endpoints

• {"endpoint_text":"- Incidence of treatment-emergent adverse events (AEs) through Day 360","definition_or_measurement_approach":"Assessment of incidence (count and classification) of treatment-emergent adverse events occurring up to Day 360."}
• {"endpoint_text":"- Change from baseline of microdystrophin protein levels at Day 90","definition_or_measurement_approach":"Change from baseline in microdystrophin protein levels measured at Day 90 (microdystrophin expression assessed in muscle biopsies per study objectives)."}

Secondary endpoints

• {"endpoint_text":"- Change from baseline of microdystrophin tissue distribution at Day 90","definition_or_measurement_approach":"Assessment of microdystrophin tissue distribution change from baseline at Day 90 (likely via muscle biopsy/histology)."}
• {"endpoint_text":"- Change from baseline of microdystrophin tissue distribution at Day 360","definition_or_measurement_approach":"Assessment of microdystrophin tissue distribution change from baseline at Day 360 (likely via muscle biopsy/histology)."}
• {"endpoint_text":"- Change from baseline of microdystrophin protein levels at Day 360","definition_or_measurement_approach":"Quantitative change from baseline in microdystrophin protein levels at Day 360 (measured in muscle biopsies)."}
• {"endpoint_text":"- Change from baseline in time to rise velocity at Day 360 and Day 540","definition_or_measurement_approach":"Change in time to rise velocity from baseline measured at Day 360 and Day 540 (functional assessment)."}
• {"endpoint_text":"- Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540","definition_or_measurement_approach":"Change in SV95C from baseline at Day 360 and Day 540 (gait/ambulatory assessment metrics)."}
• {"endpoint_text":"- Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540","definition_or_measurement_approach":"Change in 10-meter walk/run velocity from baseline at Day 360 and Day 540 (ambulatory performance measure)."}
• {"endpoint_text":"- Change from baseline in 4-stair climb velocity at Day 360 and Day 540","definition_or_measurement_approach":"Change in 4-stair climb velocity from baseline at Day 360 and Day 540 (functional performance measure)."}
• {"endpoint_text":"- Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540","definition_or_measurement_approach":"Change in NSAA score from baseline at Day 360 and Day 540 (standard DMD functional scale)."}
• {"endpoint_text":"- Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540","definition_or_measurement_approach":"Change in 6MWT distance from baseline at Day 360 and Day 540 (endurance/functional assessment)."}
• {"endpoint_text":"- Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540","definition_or_measurement_approach":"Monitoring and reporting incidence of clinically significant lab abnormalities through Day 360 and Day 540 (laboratory tests and thresholds per protocol)."}
• {"endpoint_text":"- Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540","definition_or_measurement_approach":"Monitoring incidence of clinically significant vital sign abnormalities through Day 360 and Day 540."}
• {"endpoint_text":"- Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540","definition_or_measurement_approach":"Monitoring incidence of clinically significant physical exam abnormalities through Day 360 and Day 540."}
• {"endpoint_text":"- Incidence of cardiac abnormalities by electrocardiogram (ECG) or echocardiography (ECHO) through Day 360 and Day 540","definition_or_measurement_approach":"Incidence of cardiac abnormalities detected by ECG or ECHO through Day 360 and Day 540 (cardiac imaging and ECG assessments)."}

Italy

Earliest CTIS Part Ii Submission Date

16-12-2024

Latest Decision Or Authorization Date

05-05-2026

Processing Time Days

505

Number Of Sites

1

Number Of Participants

8

Primary sponsor

Full Name

Solid Biosciences Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Parexel International (IRL) Limited

Responsibilities

Lab supplies management, Medical Monitoring; other clinical trial support functions (multiple sponsor duties codes listed)

Name

Worldwide Clinical Trials Early Phase Services LLC

Responsibilities

Microdystrophin Expression Analysis

Third parties

• {"country":"United States","full_name":"Diverge Translational Science Laboratory","duties_or_roles":"Muscle Pathology, Microsystrophin Expression Analysis","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Switzerland","full_name":"Voisin Consulting CH SARL","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Charles River Laboratories Inc.","duties_or_roles":"Viral vector Shedding Analysis","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Flagship Biosciences Inc.","duties_or_roles":"Microdystrophin Distribution Analysis","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"ViroClinics Biosciences B.V.","duties_or_roles":"AAV9 Antibody Testing","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eurofins Central Laboratory LLC","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Lab supplies management, Medical Monitoring","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Machaon Diagnostics Inc.","duties_or_roles":"sC5b-9 Testing","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Netherlands","full_name":"Eurofins Central Laboratory B.V.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Anti-SGT-003 antibody testing and cellular immune response testing to microdystrophin","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Worldwide Clinical Trials Early Phase Services LLC","duties_or_roles":"Microdystrophin Expression Analysis","organisation_type":"Pharmaceutical company"}