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Neurological Diseases Clinical Trials

 
 

St. Jude Clinical Trials

DEVOTE: Study of Nusinersen in Children with Spinal Muscular Atrophy

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy

Diseases Treated:

Spinal muscular atrophy

Eligibility:

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DYNEDMD: Study to Measure the Safety and Effectiveness of DYNE-251 in Boys with Duchenne Muscular Dystrophy

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Diseases Treated:

Duchenne muscular dystrophy

Eligibility:

  • Male age 4 to 16 years old
  • Diagnosed with Duchenne muscular dystrophy 
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FACOMS: Friedreich’s Ataxia Clinical Outcome Measures Study

Friedreich’s Ataxia Clinical Outcome Measures Study

Diseases Treated:

Friedreich’s Ataxia

Eligibility:

  • Patients with Friedreich’s Ataxia
  • Birth to 22 years
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INC: Pediatric Study of Charcot-Marie-Tooth Disease

Inherited Neuropathies Consortium Rare Disease Clinical Research Network

Diseases Treated:

Charcot-Marie-Tooth Disease

Eligibility:

  • Patients with Charcot-Marie-Tooth disease
  • Birth to 22 years
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SAPPHIRE: Efficacy and Safety of Apitegromab in Patients with Spinal Muscular Atrophy Receiving Nusinersen or Risdiplam Therapy

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Diseases Treated:

Spinal Muscular Atrophy Types 2 and 3

Eligibility:

  • Between 2 and 21 years old
  • Diagnosed with SMA type 2 or type 3 and not able to walk
  • Those taking Spinraza must have taken it for at least 10 months.
  • Those taking Evrsysdi must have taken it for at least 6 months.

Eligibility exclusions:

  • Those who have previously received Zolgensma gene therapy
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