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Clinical Trial Summary

The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA) who previously participated in study 232SM203 (NCT04089566). The secondary objective of this study is to evaluate the long-term efficacy of nusinersen administered intrathecally at higher doses to participants with SMA who previously participated in study 232SM203 (NCT04089566).


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04729907
Study type Interventional
Source Biogen
Contact
Status Enrolling by invitation
Phase Phase 3
Start date April 19, 2021
Completion date July 31, 2026

See also
  Status Clinical Trial Phase
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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
Completed NCT04419233 - Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
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 NCT00568698 - A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients Phase 1/Phase 2