BridgeMD
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy
EU CTIS ID: 2024-514501-57-00
What this study is testing
To investigate the safety and tolerability of a single intravenous dose of SGT-003 and to investigate the efficacy of a single intravenous dose of SGT-003 by assessing microdystrophin expression in muscle biopsies.
- Phase I and Phase II (Integrated)- First administration to humans
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 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
- Participant is male.
- 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
- 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 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
- 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
You likely can't join if
- 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.
- Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
- 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.
- 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.
- Sponsor employees and their family members are ineligible to participate in this study.
- Any active infection
See the full eligibility criteria
Who can join
- 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
- Participant is male.
- 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
- 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 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
- 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
- Negative for AAV antibodies
- 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
- Meet 10-meter walk/run time criteria
- Meet time to rise from supine criteria
- Cohort 5: meet Performance of Upper Limb (PUL) 2.0 criteria
- Participant has body weight ≤ 90 kg.
What rules you out
- 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.
- Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
- 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.
- 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.
- Sponsor employees and their family members are ineligible to participate in this study.
- Any active infection
- Abnormal liver function, evidence of active viral hepatitis
- Abnormal renal function
- Clinically significant abnormalities of coagulation
- Impaired cardiovascular function
- Pulmonary function predictive of (or requiring) the use of daytime ventilatory support
- Have severe hypersensitivity reactions, including anaphylaxis, to SGT-003 or its components.
- Treatment with dystrophin modifying drugs within 3 months prior to screening.
- Current or prior treatment with an approved or investigational gene transfer drug.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- 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 United States; 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.