A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

Официальное название

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

Обзор

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.

Вмешательства

• Препарат SGT-003
  Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
• Препарат Placebo
  IV infusion

Первичные конечные точки

• Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 [Срок оценки: Baseline, Day 540]

Вторичные конечные точки (12)

• Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540 [Срок оценки: Baseline, Day 540]
• Change From Baseline in 4-Stair Climb (4SC) Velocity (tasks/s) at Day 540 [Срок оценки: Baseline, Day 540]
• Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540 [Срок оценки: Baseline, Day 540]
• Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540 [Срок оценки: Baseline, Day 540]
• Cumulative Loss of Function in NSAA Items at Day 540 [Срок оценки: At Day 540]
• Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90 [Срок оценки: Baseline, Day 90]
• Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90 [Срок оценки: Baseline, Day 90]
• Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540 [Срок оценки: Baseline, Day 540]
• Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540 [Срок оценки: Baseline, Day 540]
• Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540 [Срок оценки: Baseline, Day 540]
• Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540 [Срок оценки: 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) [Срок оценки: From first dose up to Day 540]

Критерии участия

Критерии включения

• Participant is ambulatory.
• Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
• Negative for antibodies against adeno-associated virus.
• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 3 центра

• Arkansas Children's Hospital — Little Rock
• Neurology Rare Disease Center — Flower Mound
• Children's Hospital of the King's Daughters — Norfolk

Канада · 2 центра

• Alberta Children's Hospital — Calgary
• BC Children's Hospital — Vancouver

Австралия · 1 центр

• The Children's Hospital of Westmead — Sydney

Идентификаторы

NCT: NCT07160634 · SGT-003-301 · 2025-522949-22 · 1013075

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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