Neuromuscular: Research and Clinical Trials

The Neuromuscular Disease Clinic at Children’s Medical Center is actively involved in research as well as clinical trials for patients with neuromuscular disorders. Clinical trials focus on increasing information regarding rare neuromuscular disorders as well as investigating promising treatment options.


In addition to helping advance scientific discoveries, this research may offer your child hope where other treatment options may not have been successful. Discuss these options with your doctor and our research team will go over the details to ensure that your child is carefully matched to the right research study.

Current pediatric neuromuscular clinical trials at Children’s Medical Center include:

Duchenne Muscular Dystrophy

  • An exploratory study to assess two doses of GSK24002968 in the Treatment of Ambulant boys with Duchenne Muscular Dystrophy. (Exon 51 Skipping)
  • An Open-Label Safety Study for Previously Treated Ataluren (PTC124) patients with nonsense mutation dystrophyinopathy.
  • A phase 3 efficacy and safety study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy.
  • A phase III, double-blind, randomized, placebo-controlled study of the efficacy, safety and tolerability of Idebenone in 10 – 18 year old boys with DMD.
  • A randomized, double-blind, placebo-controlled, phase 3 trial of Tadalafil for Duchenne Musucular Dystrophy.
  • Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys with DMD
  • Muscular Dystrophy Association Neuromuscular Disease Registry

Spinal Muscular Atrophy

  • An open-label, escalating dose study to assess the safety, tolerability and dose-range finding of a single intrathecal dose of ISIS 396443 in patients with Spinal Muscular Atrophy.
  • An open-label, escalating dose study to assess the safety, tolerability and dose-range finding of multiple intrathecal dose of ISIS 396443 in patients with Spinal Muscular Atrophy.
  • An open-label study to assess the safety and tolerability of a single intrathecal dose of ISIS 396443 in patients with Spinal Muscular Atrophy who previously participated in ISIS 396443-CS1.
  • Spinal Muscular Atrophy Biomarkers in the Immediate Postnatal period of development.
  • Muscular Dystrophy Association Neuromuscular Disease Registry

Additional information can be found at clinicaltrials.gov

If you are interested in learning more information regarding a clinical trial or enrolling your child in a study, please contact Research Coordinator, Stephanie Trest at 214-456-3686.

 

Contact the Neuromuscular Disease Clinic at Children’s Medical Center

(214) 456-2768