Muscular Atrophy, Spinal Clinical Trial
Official title:
A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
A two-period, two-treatment, crossover study to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with spinal muscular atrophy (SMA) Type 3.
This randomized (1:1), double-blind, placebo-controlled, 2-period, 2-treatment, crossover, outpatient study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with SMA Type 3. The study is planned to include approximately 12 male and female SMA Type 3 patients. The planned duration of participation for each patient is approximately 2 months, based upon length of dose titration and excluding the screening period, which can last up to 14 days. Patients should only be taking the assigned investigational product (amifampridine phosphate 10 mg tablets or matching placebo tablets), no new therapies are permitted during the study. ;
| 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
|
||
| 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 |
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 |