Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)

Last updated: April 15, 2024
Sponsor: Catalyst Pharmaceuticals, Inc.
Overall Status: Terminated

Phase

3

Condition

Polymyositis (Inflammatory Muscle Disease)

Neuropathy

Sarcopenia

Treatment

Amifampridine Phosphate

Clinical Study ID

NCT03579966
MSK-003
  • Ages > 18
  • All Genders

Study Summary

Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.

Eligibility Criteria

Inclusion

Inclusion Criteria

  1. Participated in the MSK-002 study

  2. 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.

  3. Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin [HCG] at the end of MSK-002 study); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.

  4. Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.

Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:

  1. Epilepsy and currently on medication.

  2. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.

  3. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.

  4. Intolerable amifampridine-related side effects

  5. Treatment with an investigational drug (other than amifampridine) or device while participating in this study.

  6. Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient.

Study Design

Total Participants: 63
Treatment Group(s): 1
Primary Treatment: Amifampridine Phosphate
Phase: 3
Study Start date:
June 11, 2018
Estimated Completion Date:
August 05, 2022

Study Description

The Primary objective :

To characterize the long-term safety and tolerability of amifampridine phosphate in patients with MG

The Secondary Objective:

To assess the clinical efficacy of amifampridine phosphate over time in patients with MG based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL)

This was a long-term extension study for subjects who participated in Protocol MSK-002 where the efficacy and safety of amifampridine was evaluated in subjects diagnosed with MuSK MG or AChR-MG. The optimal dose and schedule for amifampridine from the end of the Run-in Period from Study MSK-002 was initially used for each patient. The Investigator could adjust the dose of amifampridine during the course of the trial, in order to optimize neuromuscular benefit for the patient. Clinic visits for safety assessment and for evaluation of MG-ADL were made at Months 3, 6, 9, 12, 15, 21, 27, 33 and 39. Additional visits could occur at the discretion of the Investigator.

The Original protocol (12 Nov 2017) included analysis of MG-ADL change over time, however, Statistical Analysis Plan (SAP), dated 21 Feb 2023, removed the analysis of MG-ADL score change over time.

Connect with a study center

  • Department of Neuroimmunology and Neuromuscular Diseases Carlo Besta Neurological Institute Via Celoria

    Milan, 11-201332
    Italy

    Site Not Available

  • UC Irvine

    Irvine, California 92868
    United States

    Site Not Available

  • Rush University

    Chicago, Illinois 60612
    United States

    Site Not Available

  • OSF Healthcare St. Francis Medical Center

    Peoria, Illinois 61637
    United States

    Site Not Available

  • University of Kansas Medical Center

    Fairway, Kansas 66205
    United States

    Site Not Available

  • Wayne State University

    Detroit, Michigan 48201
    United States

    Site Not Available

  • Michigan State University

    East Lansing, Michigan 48824
    United States

    Site Not Available

  • Unviersity of Missouri

    Columbia, Missouri 65212
    United States

    Site Not Available

  • University of Cincinnati

    Cincinnati, Ohio 45219
    United States

    Site Not Available

  • Cleveland Clinic

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • Ohio State University

    Columbus, Ohio 43221
    United States

    Site Not Available

  • Univerity of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Wesley Neurology Clinic

    Memphis, Tennessee 38018
    United States

    Site Not Available

  • Houston Methodist

    Houston, Texas 77030
    United States

    Site Not Available

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