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 range18 Years

SexALL

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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.
✕. 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.

What they're measuring

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).

Trial details

NCT IDNCT03304054

SponsorCatalyst Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-01-31

Results submitted2021-04-13

View on ClinicalTrials.gov More Myasthenia Gravis, Generalized trials