St. Jude Clinical Trials
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:
Eligibility:
- Male age 4 to 16 years old
- Diagnosed with Duchenne muscular dystrophy
FACOMS: Friedreich’s Ataxia Clinical Outcome Measures Study
Friedreich’s Ataxia Clinical Outcome Measures Study
Diseases Treated:
Eligibility:
- Patients with Friedreich’s Ataxia
- Birth to 22 years
GENETX: Assessment for Gene Therapy Decisions
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-Aids for Gene Therapy for Rare Diseases (GENETX)
Diseases Treated:
Neurological Disorders
Sickle Cell Disease
Hemophilia
Inherited Genetic Diseases
Eligibility:
- Non-therapeutic clinical trial
- Interviews conducted remotely
- 18-35-year-olds with rare genetic diseases
- Parents or caregivers of patients under 21 with rare genetic diseases
- Health care workers for gene therapy patients
INC: Pediatric Study of Charcot-Marie-Tooth Disease
Inherited Neuropathies Consortium Rare Disease Clinical Research Network
Diseases Treated:
Eligibility:
- Patients with Charcot-Marie-Tooth disease
- Birth to 22 years
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