Phase
Condition
Muscular Dystrophy
Treatment
SGT-003
Clinical Study ID
Ages < 17 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion 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, asdefined 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 mutationpredictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where agenotype may be predictive of residual dystrophin production and/or a clear clinicaldiagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levelsin baseline muscle biopsies may be required to determine eligibility under thiscriterion.
Negative for AAV antibodies.
Steroid regimen:
Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior toScreening Part A or Rescreening, allowing for weight-based modificationsconsistent with clinical practice.
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
Exclusion
Exclusion Criteria:
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 toscreening or 5 half-lives since last administration, whichever is longer.
Established clinical diagnosis of DMD that is associated with any deletion mutationin exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a geneticreport and confirmed by Sponsor genetic testing.
Other inclusion or exclusion criteria apply.
Study Design
Connect with a study center
The Hospital for Sick Children
Toronto, Ontario M5G 0A4
CanadaActive - Recruiting
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, 00168
ItalyActive - Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas 72202
United StatesActive - Recruiting
University of California, Los Angeles Medical Center
Los Angeles, California 90095
United StatesActive - Recruiting
University of California, Davis
Sacramento, California 95817
United StatesActive - Recruiting
Rare Disease Research
Atlanta, Georgia 30329
United StatesActive - Recruiting
Washington University in St. Louis
Saint Louis, Missouri 63110
United StatesActive - Recruiting
Nationwide Children's Hospital
Columbus, Ohio 43215
United StatesActive - Recruiting
Children's Hospital of the King's Daughters
Norfolk, Virginia 23510
United StatesActive - Recruiting
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