NCT03304054

Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

A Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 93 (actual)
• Sponsor: Catalyst Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.

Detailed description

Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In addition, a sample of AChR-MG patients will be assess for efficacy and safety of amifampridine phosphate. Planned duration of participation for each patient is at least 38 days, excluding the screening period. Eligible patients will be titrated to an efficacious dose of amifampridine phosphate and those who demonstrate improvement will be randomized to either placebo or amifampridine, in a double-blind fashion, for 10 days.

Conditions

• Myasthenia Gravis, Generalized

Interventions

Type	Name	Description
DRUG	Amifampridine Phosphate	tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
DRUG	Placebo Oral Tablet	tablets matching amifampridine phosphate, 3 to 4 times a day

Timeline

• Start date: 2018-04-18
• Primary completion: 2020-01-31
• Completion: 2020-03-15
• First posted: 2017-10-06
• Last updated: 2024-03-12
• Results posted: 2021-08-13

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study