NCT05919407

Pyridostigmine and Amifampridine for Myasthenia Gravis

IMproving Symptomatic Treatment With Pyridostigmine and Amifampridine: a Randomized Double-blinded, Placebo Controlled Crossover Trial in Patients With Myasthenia Gravis (IMPACT-MG)

• Status: Unknown
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Leiden University Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A randomized, double-blind, placebo controlled, crossover intervention study evaluating the effect of pyridostigmine (part 1) and amifampridine (part 2) in Myasthenia Gravis (MG).

Detailed description

In the first part of the study, patients who are currently using pyridostigmine will be randomly allocated to one of two consecutive treatment periods in which patients either first receive placebo and then their usual dose of pyridostigmine, or vice versa. Each treatment period lasts 5 days with a 2-day wash-out period between each treatment period. Measurements will be performed at every last day of a treatment period (day 5 and day 12). In the second part of the study the effect of two doses of amifampridine as add-on to pyridostigmine will be studied. Patients will be randomly assigned to either one of three treatment sequences; 1) amifampridine 30 mg - amifampridine 60 mg - placebo or 2) amifampridine 60 mg - placebo - amifampridine 30 mg or 3) placebo - amifampridine 30 mg - amifampridine 60 mg. Again, each treatment period consists of 5 days and will be separated by a 2-day wash-out period. Measurements will be performed at every last day of treatment (day 19, day 26 and day 33). Patients will have the option to participate in a substudy to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of amifampridine in AChR positive MG patients.

Conditions

• Myasthenia Gravis

Interventions

Type	Name	Description
DRUG	Pyridostigmine	Participants will receive pyridostigmine 10 mg tablets.
DRUG	Amifampridine (base) with modified release	Participants will receive amifampridine (base) with modified release 15 mg or 30 mg tablets.
DRUG	Placebo	The placebo tablets will be identical apart from the active substance (pyridostigmine)
DRUG	Placebo	The placebo tablets will be identical apart from the active substance (amifampridine base)

Timeline

• Start date: 2023-03-22
• Primary completion: 2024-09-22
• Completion: 2024-09-22
• First posted: 2023-06-26
• Last updated: 2023-06-26

Locations

1 site across 1 country: Netherlands