MuSK antibody positive myasthenia gravis MedDRA version: 21.1 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
CATALYST PHARMACEUTICALS INC.
Eligibility
Sex/Gender
All
Inclusion criteria
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 electromyography (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 and/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 - 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
Exclusion criteria
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 - Treatment with an investigational drug (other than amifampridine), device, or biological agent within 60 days prior to screening or 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 - History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To characterize the overall safety and tolerability of amifampridine phosphate compared with placebo in patients with MuSK antibody positive myasthenia gravis. To assess the clinical efficacy of amifampridine phosphate compared with placebo in patients with MuSK antibody positive myasthenia gravis based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL);Secondary Objective: To assess the clinical efficacy of amifampridine phosphate compared with placebo using the Quantitative Myasthenia Gravis (QMG) score. To assess the safety and efficacy of amifampridine phosphate compared with placebo in a sample of patients with AChR antibody positive myasthenia gravis;Primary end point(s): The primary efficacy endpoint of the study is the change in MG-ADL score from Day 0 (baseline) for MuSK-MG subjects treated with amifampridine and placebo;Timepoint(s) of evaluation of this end point: About 38 days after the start of the IMP intake in the run-in phase | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The change in QMG score from Day 0 (baseline) for MuSK-MG subjects treated with amifampridine and placebo; The proportion of subjects with a change of 2, or more, in MG-ADL score for MuSK-MG subject treated with amifampridine and placebo; The proportion of subjects with a change of 3, or more, in QMG score for MuSK-MG subjects treated with amifampridine and placebo; Safety of IMP 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, and concomitant medications will also be evaluated;Timepoint(s) of evaluation of this end point: About 38 days after the start of the IMP intake in the run-in phase; About 38 days after the start of the IMP intake in the run-in phase; About 38 days after the start of the IMP intake in the run-in phase; The study period during which all non-serious AEs will be reported begins after the first administration of study drug through the termination visit or at the early termination visit. After informed consent but prior to initiation of study treatment, only SAEs associated with any protocol-imposed interventions will be reported. The reporting period for SAEs begins after informed consent is obtained and continues through 4 weeks after the last visit | — |
Countries
Canada, Italy, United States
Contacts
Public ContactGary Ingenito
Catalyst Pharmaceuticals Inc.
Outcome results
None listed