Rare Disease Research

The trials here

What the 14 trials at this site are about, from their registry records.

Conditions

• Duchenne muscular dystrophy9 trials
• Stroke2 trials
• Spinal muscular atrophy1 trial

Phase

• Phase 26 trials
• Phase 33 trials
• Phase 11 trial
• Phase 1/21 trial
• Phase 2/31 trial

Status

• Active, not recruiting6 trials
• Completed4 trials
• Enrolling by invitation1 trial
• Recruiting1 trial
• Terminated1 trial
• Withdrawn1 trial

Lead sponsor

• Edgewise Therapeutics, Inc.4 trials
• REGENXBIO Inc.2 trials
• Tisento Therapeutics2 trials
• Biohaven Pharmaceuticals, Inc.1 trial
• CureDuchenne1 trial
• and 4 more

Running trials

• Recruiting

  A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

  NCT06138639· Phase 1/2· Solid Biosciences Inc.· Source

  Contact: Renata Shih, MD (principal investigator)

• Active, not recruiting

  NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

  NCT05996003· Phase 2· NS Pharma, Inc.· Source

• Active, not recruiting

  A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS

  NCT06144645· Phase 3· Foundation for Prader-Willi Research· Source

• Enrolling by invitation

  Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy

  NCT06066580· Phase 2· Edgewise Therapeutics, Inc.· Source

• Active, not recruiting

  AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

  NCT05693142· Phase 2/3· REGENXBIO Inc.· Source

• Active, not recruiting

  AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

  NCT05683379· REGENXBIO Inc.· Source

• Active, not recruiting

  A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)

  NCT05540860· Phase 2· Edgewise Therapeutics, Inc.· Source

• Active, not recruiting

  A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

  NCT05337553· Phase 3· Biohaven Pharmaceuticals, Inc.· Source

Recruiting at the same time

How many trials recruit for the same indication here at once. A count, not a rating.

• Duchenne muscular dystrophy2 recruiting

Closed trials

• Terminated

  An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

  NCT06961344· Phase 2· Tisento Therapeutics· Source

• Completed

  A Phase 2b Study of Zagociguat in Patients With MELAS

  NCT06402123· Phase 2· Tisento Therapeutics· Source

• Recruiting

  A Study of Pitolisant in Patients With Prader-Willi Syndrome

  NCT06366464· Phase 3· Harmony Biosciences Management, Inc.· Source

  Contact: Ann Adee

• Completed

  Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

  NCT05291091· Phase 2· Edgewise Therapeutics, Inc.· Source

• Completed

  A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy

  NCT05160415· Phase 1· Edgewise Therapeutics, Inc.· Source

• Completed

  CureDuchenne Link®: A Resource for Research

  NCT04972604· CureDuchenne· Source