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Clinical Trial Summary

This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04256265
Study type Expanded Access
Source Genentech, Inc.
Contact
Status Approved for marketing
Phase

See also
  Status Clinical Trial Phase
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Completed NCT02908685 - A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants Phase 2
Recruiting NCT05575011 - A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115 Phase 1
Completed NCT05073133 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) Phase 4
Terminated NCT02240355 - A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) Phase 1
Recruiting NCT05042921 - Pediatric Spinal Muscular Atrophy (SMA) China Registry
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Recruiting NCT05861986 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy Phase 4
Completed NCT00466349 - International SMA Patient Registry
Completed NCT03920865 - A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function Phase 1
Recruiting NCT05481164 - Newborn Screening for Spinal Muscular Atrophy
Completed NCT04089566 - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Phase 3
Recruiting NCT05861999 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy Phase 4
Recruiting NCT05755451 - Natural History of SMA
Active, not recruiting NCT01233817 - Progressive Strength Training in Spinal Muscular Atrophy N/A
Recruiting NCT04317794 - Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
Active, not recruiting NCT05618379 - Adult Spinal Muscular Atrophy (SMA) China Registry
Active, not recruiting NCT04488133 - A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec Phase 4
Completed NCT03781479 - Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients Phase 2
Completed NCT00568802 - A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients Phase 1/Phase 2