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

Purpose

The purpose of this research study is to evaluate a new investigational product called “SGT-001” which is designed to target the underlying cause of DMD in male children and adolescents (ages 4-17), by producing a shortened version of the dystrophin protein.

Procedures

SGT-001 is considered to be an experimental drug. Experimental means that this study medicine is currently being tested. It also means that the U.S. Food and Drug Administration (FDA) and the European Medical Agency (EMA) and other regulatory authorities do not allow it to be sold for treating patients. The regulatory authorities only allow SGT-001 to be used in research. This study is the first in humans therefore the safety of the experimental drug has not been established.

If your child qualifies for the study your child will be randomly assigned (like flipping a coin) to either the active treatment group or the control (delayed treatment) group.

If your child is enrolled in the active treatment group, they will receive a single dose of SGT-001 soon after enrolling. If your child does gets randomized into the control (delayed treatment) group, he will still be able to participate in the study assessments, and provided your child still qualifies, he will receive a single dose of SGT-001 about a year after joining the study.

Eligibility

  • Male
  • 4-17 years old 
  • 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

Additional inclusion and exclusion criteria may apply. Please contact Stephanie Salabarria for more information: 

Age

18 and under

Gender

Male

Monetary compensation

Yes

Can be done from home

No

Keywords

Muscular dystrophy, Muscular dystrophy - resources, Neurology

Principal Investigator

Barry Byrne, MD, PhD

Department

Neurology

Contact Information

ssalabarria@ufl.edu

(352) 273-6582


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