Clinical study evaluating the long term safety of amifampridine phosphate in patients with MuSK antibody and AChR antibody positive myasthenia gravis
- Conditions
- MuSK antibody and AChR antibody positive myasthenia gravisMedDRA version: 21.1Level: PTClassification code 10028417Term: Myasthenia gravisSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2017-004777-14-IT
- Lead Sponsor
- CATALYST PHARMACEUTICALS INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 70
- 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 63
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 7
- 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term safety and tolerability of amifampridine in patients with MuSK antibody positive myasthenia gravis and AChR antibody positive myasthenia gravis;Secondary Objective: To assess the effect of amifampridine phosphate on Myasthenia Gravis Activities of Daily Living Score (MG-ADL).<br>;Primary end point(s): The primary endpoint of the study is the safety and the tolerability of amifampridine. Safety will be assessed by the incidence of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs). Vital signs, 12-lead ECGs, clinical laboratory tests, physical examination findings, and concomitant medications will also be evaluated;Timepoint(s) of evaluation of this end point: The primary endpoint will be assessed at months 3, 6, 9, 12, 15 and 21 from the start of treatment with amifampridine
- Secondary Outcome Measures
Name Time Method Secondary end point(s): To assess the clinical efficacy of amifampridine phosphate over time in patients with Myasthenia Gravis MG based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL);Timepoint(s) of evaluation of this end point: Evaluation of MG-ADL will be made at months 3, 6, 9, 12, 15, and 21 from the start of treatment