Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients

Phase 2 Completed 13 enrolled Catalyst Pharmaceuticals, Inc.

RandomizedCrossoverQuadruple-blindTreatment

Summary

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.

Timeline

Start: 2019-01-21
Primary completion: 2020-07-23
Completion: 2020-07-23

Outcome

Met primary endpoint

paper Amifampridine treatment led to a statistically significant improvement in HFMSE (mean difference 0.792; 95% CI from 0.22 to 1.37; p = 0.0083), compared to placebo PMID 35763114 ↗

Publications

Bonanno S, Giossi R, Zanin R, Porcelli V, Iannacone C, Baranello G, Ingenito G, Iyadurai S, Stevic Z, Peric S, Maggi L. Amifampridine safety and efficacy in spinal muscular atrophy ambulatory patients: a randomized, placebo-controlled, crossover phase 2 trial. J Neurol. 2022 Nov;269(11):5858-5867. doi: 10.1007/s00415-022-11231-7. Epub 2022 Jun 28.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Amifampridine	Small molecule	30 mg	Oral
Subject	Amifampridine	Small molecule	80 mg	Oral