Pyridostigmine and Amifampridine for Myasthenia Gravis (IMPACT-MG)
Myasthenia Gravis
About this trial
This is an interventional treatment trial for Myasthenia Gravis
Eligibility Criteria
Inclusion Criteria: Age >18 years AChR positive myasthenia gravis (ocular or generalized) Current use of pyridostigmine MGFA Clinical Classification I-IV Receiving a stable dose of MG treatment (other than pyridostigmine). If applicable: A stable steroid regimen for 1 month Nonsteroidal immunosuppressants: i. Azathioprine, mycophenolate mofetil, cyclosporine or other nonsteroid immunosuppressive agents start > 3 months ago and a stable regimen for 1 month. ii. Rituximab start > 6 months ago, complement inhibitors and Fc receptor inhibitors start > 6 months ago and a stable regimen for 3 months. Additional inclusion criteria for part 2 To be eligible for participation in part 2 of the study patients must score >10 points on the MGII questionnaire at inclusion. We will include a maximum of 5 patients with a MGFA class I (i.e. 20 percent of the total number of included patients) to ensure that this study accurately reflects the clinical population. Exclusion Criteria: Use of intravenous immunoglobulin or plasma exchange <4 weeks or planned during the trial. Thymectomy < 6 months, or thymectomy (expected) to take place during the trial Use of other acetylcholinesterase inhibitors than pyridostigmine Pregnancy, lactation or intention to become pregnant during the study Treatment with amifampridine is contraindicated. Contraindications include a history of epilepsy, uncontrolled asthma, inherited QT syndrome / a prolonged QT interval (as indicated by ECG), any drug known to cause QT c-prolongation, any drug known to lower the epileptic threshold, a known hypersensitivity reaction to the active substance or to any of the excipients. The patient is unable to fill out the study questionnaires or be interviewed in Dutch, or is unable to undergo the tests needed for the study, or is unable to give informed consent for participation in the study. The investigator can exclude patients for this trial which are deemed not suitable for any reason.
Sites / Locations
- Leiden University Medical CenterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Placebo Comparator
Experimental
Placebo Comparator
Pyridostigmine
Placebo (pyridostigmine)
Amifampridine (base) with modified release
Placebo (amifampridine)
The dose of pyridostigmine will be based on the patient's prior experience with pyridostigmine under the assumption that the patient already gained sufficient experience during their disease course to know which dose is effective for them as patients are advised by their treating neurologist to continually adjust their dose based on their symptoms and side effects.
Same as "Experimental", however capsules contain placebo.
Patients will receive amifampridine 2 dd 15 mg and amifampridine 2 dd 30 mg as add-on to the pre-study dose of pyridostigmine.
Same as "Experimental", however capsules contain placebo.