Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 4 - 17 |
| Updated: | 12/13/2017 |
| Start Date: | December 6, 2017 |
| End Date: | March 2021 |
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
This is a randomized, 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). Eligible patients will be randomized to an active treatment group
or an untreated control group. Patients in the active treatment group will receive a single
intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. Patients
in the untreated control group who continue to meet treatment criteria will receive SGT-001
after 1 year on study.
safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne
muscular dystrophy (DMD). Eligible patients will be randomized to an active treatment group
or an untreated control group. Patients in the active treatment group will receive a single
intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. Patients
in the untreated control group who continue to meet treatment criteria will receive SGT-001
after 1 year on study.
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 of oral corticosteroids ≥ 24 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.
We found this trial at
1
site
Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Barry Byrne, MD, PhD
Phone: 352-273-6582
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
Click here to add this to my saved trials
