TerminatedPhase 3

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

Stopped: Lack of measure appropriate data (MG-ADL).

United States, Italy63 participantsStarted 2018-06-11

Plain-language summary

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

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Participated in the MSK-002 study
✓. 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.
✓. 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.
✓. 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.

✕. Epilepsy and currently on medication.
✕. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
✕. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
✕. Intolerable amifampridine-related side effects
✕. Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
✕. 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.

Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)

Timeframe: over 39 months

NCT IDNCT03579966

SponsorCatalyst Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-08-05

Results submitted2024-03-12

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Participated in the MSK-002 study
✓. 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.
✓. 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.
✓. 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.

✕. Epilepsy and currently on medication.
✕. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
✕. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
✕. Intolerable amifampridine-related side effects
✕. Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
✕. 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.