Clinical study evaluating the effect of Amifampridine phosphate in patients with MuSK antibody positive myasthenia gravis, and a sample of AchR antibody positive myasthenia gravis patients
- Conditions
- MuSK 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
- EUCTR2017-004018-25-IT
- Lead Sponsor
- CATALYST PHARMACEUTICALS INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 70
- 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
- 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)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method