Muscular Atrophy, Spinal Clinical Trial
Official title:
Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C). The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00533221 -
Pilot Study of Growth Hormon to Treat SMA Typ II and III
|
Phase 2 | |
| 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
|
||
| 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
|
||
| 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 | |
| Completed |
NCT00568802 -
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
|
Phase 1/Phase 2 |