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New treatment option for Duchenne Muscular Dystrophy
Official title A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
ClinicalTrials.gov ID: NCT07160634
What this study is testing
What is SGT-003?
SGT-003 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 to 11, men only
You may be able to join if
- 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...
- Meet 10-meter walk/run time criteria.
You likely can't join if
- 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...
- 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...
See the full eligibility criteria
- 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...
- Meet 10-meter walk/run time criteria.
- Meet time to rise from supine criteria.
- Participant has bodyweight ≤50 kg.
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Flower Mound, Texas, United States
- Norfolk, Virginia, United States
- Sydney, New South Wales, Australia
- Calgary, Alberta, Canada
- Vancouver, British Columbia, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 7 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Flower Mound, Texas, United States; Norfolk, Virginia, United States; Sydney, New South Wales, Australia; Calgary, Alberta, Canada; Vancouver, British Columbia, Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.