BridgeMD
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy
EU CTIS ID: 2025-522949-22-00
What this study is testing
To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing change in muscle function.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participant 7 to <12 years of age.
- 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.
- 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.
- Participant is ambulatory. Ambulatory is defined as “being able to walk without the use of an assistive device.”
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
- Negative for AAV antibodies.
You likely can't join if
- 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.
- History of severe hypersensitivity reactions, including anaphylaxis, to the product or its components.
- 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.
- Any active infection.
- 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.
See the full eligibility criteria
Who can join
- Participant 7 to <12 years of age.
- 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.
- 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.
- Participant is ambulatory. Ambulatory is defined as “being able to walk without the use of an assistive device.”
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
- Negative for AAV antibodies.
- 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.
- Meet 10-meter walk/run time criteria.
- Meet time to rise from supine criteria.
- Participant has bodyweight ≤50 kg
- Participant is genetically male.
What rules you out
- 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.
- History of severe hypersensitivity reactions, including anaphylaxis, to the product or its components.
- 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.
- Any active infection.
- 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.
- 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.
- Sponsor employees and their family members are ineligible to participate in this study.
- Known liver disease, evidence of active viral hepatitis, or abnormal liver function.
- Abnormal renal function
- Clinically significant abnormalities of coagulation
- Impaired cardiovascular function
- Pulmonary function predictive of or requiring the use of daytime ventilatory support outside of acute illnesses.
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- United Kingdom
- 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, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; United Kingdom; 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 EU CTIS; the study team decides eligibility.