Page last updated: 2024-11-08
amifampridine and Adult Spinal Muscular Atrophy
amifampridine has been researched along with Adult Spinal Muscular Atrophy in 1 studies
Amifampridine: 4-Aminopyridine derivative that acts as a POTASSIUM CHANNEL blocker to increase release of ACETYLCHOLINE from nerve terminals. It is used in the treatment of CONGENITAL MYASTHENIC SYNDROMES.
Research Excerpts
| Excerpt | Relevance | Reference |
|---|
| "Amifampridine treatment led to a statistically significant improvement in HFMSE (mean difference 0." | 3.11 | Amifampridine safety and efficacy in spinal muscular atrophy ambulatory patients: a randomized, placebo-controlled, crossover phase 2 trial. ( Baranello, G; Bonanno, S; Giossi, R; Iannacone, C; Ingenito, G; Iyadurai, S; Maggi, L; Peric, S; Porcelli, V; Stevic, Z; Zanin, R, 2022) |
Research
Studies (1)
| Timeframe | Studies, this research(%) | All Research% |
|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 0 (0.00) | 29.6817 |
| 2010's | 0 (0.00) | 24.3611 |
| 2020's | 1 (100.00) | 2.80 |
Authors
| Authors | Studies |
|---|
| Bonanno, S | 1 |
| Giossi, R | 1 |
| Zanin, R | 1 |
| Porcelli, V | 1 |
| Iannacone, C | 1 |
| Baranello, G | 1 |
| Ingenito, G | 1 |
| Iyadurai, S | 1 |
| Stevic, Z | 1 |
| Peric, S | 1 |
| Maggi, L | 1 |
Clinical Trials (1)
Trial Overview
| Trial | Phase | Enrollment | Study Type | Start Date | Status |
|---|
| 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[NCT03781479] | Phase 2 | 13 participants (Actual) | Interventional | 2019-01-21 | Completed |
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] |
Trial Outcomes
Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis
Hammersmith Functional Motor Scale Expanded (HFMSE) assesses motor function by functional item in order of progressive difficulty, with higher values showing higher function abilities. Each item is scored on a scale of 0-2 with 2 representing item achieved unaided and 0 representing inability to achieve item. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28. The total HFMSE score was calculated as the sum of each item score, with a maximum score of 66 (all items achieved unaided) and minimum score of 0 (all items failed). Change from baseline (CFB) will be assessed from Day 0 to Day 28. A mixed effects liner model was fit with the HFMSE change from baseline (CFB) scores at Day 28 as a response and treatment, sequence, and treatment by sequence as fixed effect terms and patient as a random effect. (NCT03781479)
Timeframe: Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28
| Intervention | Overall Score (Least Squares Mean) |
|---|
| Amifampridine Phosphate | 0.208 |
| Placebo | -0.583 |
Trials
1 trial available for amifampridine and Adult Spinal Muscular Atrophy