Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (NCT03304054) | Clinical Trial Compass
CompletedPhase 3
Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG
United States93 participantsStarted 2018-04-18
Plain-language summary
Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
. Male or female ≥18 years of age.
. Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at Screening or by previous antibody test, with report available.
. Confirmatory EMG or EMG report.
. Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening.
. MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to non-ocular items.
. Patients receiving steroids or pyridostigmine should not have any modification of drug regimen during the month before Screening.
. Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin \[HCG\] at screening); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.
Exclusion criteria
. Epilepsy and currently on medication.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
Timeframe: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).
. Concomitant use of medicinal products with a known potential to cause QTc prolongation.
. Patients with long QT syndromes.
. History of thymectomy within 12 months before Screening.
. An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.
. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
. Patients receiving immunomodulatory treatment (e.g. plasma exchange \[PE\], therapeutic plasma exchange \[TPE\], intravenous immunoglobulin G \[IVIG\]) should not have any treatment in the previous 4 weeks prior to Randomization or at any time during the study.
. Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to Screening.