Skip to main content
Update Location

My Location

Update your location to show providers, locations, and services closest to you.

Enter a zip code
Or
Select a campus/region
(352) 733-0111

Solid - SGT - 001

  • Status
    Accepting Candidates
  • Age
    4 Years - 17 Years
  • Sexes
    Male
  • Healthy Volunteers
    No
I'm interested
Share this study
Facebook
Twitter
LinkedIn

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 ≥ 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 ≥ 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 ≤ 18 kg will be implemented for the next two

patients to be dosed.

Lead researchers

  • Barry J Byrne, MD, PhD
    Pediatric Cardiologist
    Barry J Byrne
  • Renata Shih, MD
    Pediatric Cardiologist, Pediatric Neuromuscular Rehabilitation Specialist
    Languages: Portuguese
    Renata Shih

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

    Renata Shih
    Barry Byrne
  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.