Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 4:56 PM
Ignite Modification Date:
2025-12-24 @ 4:56 PM
NCT ID:
NCT06630650
Eligibility Criteria:
* INCLUSION CRITERIA:
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
* Stated willingness to comply with all study procedures and availability for the duration of the study
* Male or female, aged \>= 6 months of age
* Clinically stable as evidenced by medical record review and remote screening questionnaire
* Genetically confirmed congenital myasthenic syndrome (pathogenic or likely pathogenic variants identified by CLIA testing in an established CMS-related gene including but not limited to DOK7, COLQ, CHRNE, RAPSN, CHAT, GFPT1, DPAGT1 OR pathogenic/likely pathogenic variant in combination with a variant of uncertain significance (VUS) AND additional clinical supporting evidence of CMS).
* Agreement to adhere to Lifestyle Considerations throughout study duration
* Ability of subject to understand and the willingness to provide informed consent (\>=18 years of age) and assent (\>=7 years of age).
EXCLUSION CRITERIA:
* Received gene transfer therapy
* Pregnant women (prior to enrollment)
* Ongoing medical condition or medication use that is deemed by the Principal Investigator to interfere with the conduct or assessments of the study or safety of the subject.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
6 Months
Maximum Age:
99 Years
Study:
NCT06630650
Study Brief:
Protocol Section:
NCT06630650