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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-522949-22 | EudraCT Number | ||
| 1013075 | Other Identifier | IRAS |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SGT-003 followed by Placebo | Experimental | Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2. |
|
| Placebo followed by SGT-003 | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SGT-003 | Drug | Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5) |
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| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 | Baseline, Day 540 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540 | Baseline, Day 540 | |
| Change From Baseline in 4-Stair Climb (4SC) Velocity (tasks/s) at Day 540 | Baseline, Day 540 | |
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Inclusion Criteria:
Exclusion Criteria:
Other Inclusion/Exclusion criteria to be applied as per protocol.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Solid Bio Clinical Trials | Contact | 6173374680 | clinicaltrials@solidbio.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Not yet recruiting | Little Rock | Arkansas | 72202 | United States |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| Placebo | Drug | IV infusion |
|
| Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540 |
| Baseline, Day 540 |
| Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540 | Baseline, Day 540 |
| Cumulative Loss of Function in NSAA Items at Day 540 | At Day 540 |
| Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90 | Baseline, Day 90 |
| Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90 | Baseline, Day 90 |
| Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540 | Baseline, Day 540 |
| Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540 | Baseline, Day 540 |
| Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540 | Baseline, Day 540 |
| Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540 | 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) | From first dose up to Day 540 |
| Neurology Rare Disease Center | Not yet recruiting | Flower Mound | Texas | 75082 | United States |
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| Children's Hospital of the King's Daughters | Not yet recruiting | Norfolk | Virginia | 23510 | United States |
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| The Children's Hospital of Westmead | Recruiting | Sydney | New South Wales | Australia |
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| BC Children's Hospital | Recruiting | Vancouver | British Columbia | Canada |
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |