NCT03781479

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Catalyst Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 6 Years – 50 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
DRUG	Amifampridine Phosphate	Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.
DRUG	Placebo Oral Tablet	Placebo Oral Tablet

Timeline

• Start date: 2019-01-21
• Primary completion: 2020-07-23
• Completion: 2020-07-23
• First posted: 2018-12-20
• Last updated: 2021-06-01
• Results posted: 2021-06-01

Locations

1 site across 1 country: Italy

Regulatory

• FDA-regulated drug study