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Solid - SGT - 001

  • Status
    Accepting Candidates
  • Age
    4 Years - 17 Years
  • Sexes
    Male
  • Healthy Volunteers
    No
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Objective

This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.

The protocol was amended to drop the control arm after 4 subjects were dosed. Subjects currently enrolling are assigned to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol.

Details

Full study title A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy
Protocol number OCR23423
ClinicalTrials.gov ID NCT03368742
Phase Phase 1/Phase 2

Eligibility

Inclusion Criteria:

  • Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype

  • Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory patients)

  • Anti-AAV9 antibodies below protocol-specified thresholds

  • Stable cardiac and pulmonary function

  • Adolescents: non-ambulatory by protocol-specified criteria

  • Children: ambulatory by protocol-specified criteria

  • Stable daily dose (or equivalent) of oral corticosteroids 鈮� 12 wks

Exclusion Criteria:

  • Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect subject safety, compromise completion of treatment and follow-up, or impair assessment of study results

  • Abnormal liver function

  • Abnormal renal function

  • Clinically significant coagulation abnormalities

  • Impaired cardiovascular function based on cardiac MRI or ECHO

  • Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support

  • Significant spinal deformity or presence of spinal rods

  • Body mass index 鈮� 95th percentile for age

  • Exposure to another investigational drug within 3 months or 5 half-lives prior to screening

  • Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening

Additional inclusion/exclusion criteria may apply. Patients over 30 kg will not be

eligible for treatment at this time. A weight limit of 鈮� 18 kg will be implemented for

the next two patients to be dosed.

Lead researcher

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