ong term safety study of amifampridine phosphate in patients with Congenital Myasthenic Syndromes (CMS) - CMS-002

CATALYST PHARMACEUTICALS INC.0 个研究点目标入组 20 人开始时间: 2021年6月17日最近更新: 2021年9月7日

适应症

试验速览

暂无简介。

•1.Participated in the CMS-001 study or failed to meet screening or randomization criteria of Protocol CMS-001 but considered to have potential clinical benefit from treatment with amifampridine phosphate.
•2.Willing and able to provide written informed consent by patient or guardian after the nature of the study has been explained and before the start of any research-related procedures.
•3.Female patients of childbearing potential defined according to the Clinical Trial Facilitation Group - CTFG guidelines must have a negative urine pregnancy test and must practice an effective, reliable contraceptive regimen according to the CTFG guidelines criteria 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.
•Are the trial subjects under 18? yes
•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 13
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•Individuals who meet any of the exclusion criteria in the protocol CMS-001 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.

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