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New treatment option for Duchenne Muscular Dystrophy
Official title A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
ClinicalTrials.gov ID: NCT06138639
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 multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 17, men only
You may be able to join if
- Cohort 1: 4 to \<7 years of age
- Cohort 2: 7 to \<12 years of age
- Cohort 3: 0 to \< 4 years of age
- Cohort 4: 12 to \< 18 years of age
- Cohort 5: 10 to \< 18 years of age
You likely can't join if
- Treatment with dystrophin modifying drugs within 3 months prior to screening.
- Current or prior treatment with an approved or investigational gene transfer drug.
- Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is...
- Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or...
See the full eligibility criteria
- Cohort 1: 4 to \<7 years of age
- Cohort 2: 7 to \<12 years of age
- Cohort 3: 0 to \< 4 years of age
- Cohort 4: 12 to \< 18 years of age
- Cohort 5: 10 to \< 18 years of age
- 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...
- Negative for AAV antibodies.
- Steroid regimen:
- 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...
- 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
- Treatment with dystrophin modifying drugs within 3 months prior to screening.
- Current or prior treatment with an approved or investigational gene transfer drug.
- Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
- Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as...
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Los Angeles, California, United States
- Sacramento, California, United States
- San Diego, California, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- St Louis, Missouri, United States
- Columbus, Ohio, United States
- Portland, Oregon, United States
- Philadelphia, Pennsylvania, United States
- Norfolk, Virginia, United States
- Seattle, Washington, United States
- Toronto, Ontario, Canada
- Rome, Italy
- London, United Kingdom
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 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Los Angeles, California, United States; Sacramento, California, United States; San Diego, California, United States; Atlanta, Georgia, United States; Chicago, Illinois, United States and 9 more location(s). 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.