STXBP1 and SYNGAP1 Related Disorders Natural History Study (NCT06555965) | Clinical Trial Compass
RecruitingNot Applicable
STXBP1 and SYNGAP1 Related Disorders Natural History Study
United States600 participantsStarted 2023-08-30
Plain-language summary
The purpose of this study is to find out more about STXBP1 and SYNGAP1 related disorders. The information gathered by this study will be used to prepare for clinical treatment trials. The primary objective of the study is to better define and outline the clinical spectrum of STXBP1 and SYNGAP1 through detailed developmental, seizure, and quality of life assessments as an extension of routine clinical care.
Who can participate
SexALL
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Inclusion Criteria:
* Male or female of any age.
* Presence of a STXBP1 or SYNGAP1 gene mutation. The variant in STXBP1 or SYNGAP1 must be classified as causative based on clinical and variant classification criteria. Historical documentation is sufficient to support eligibility for the study. Confirmatory testing will be obtained, if necessary, at baseline and performed by a CLIA certified laboratory.
Exclusion Criteria:
* The presence of a confirmed mutation in a gene other than STXBP1 or SYNGAP1 that is known to contribute to a neurodevelopmental disability. This includes full gene deletions of STXBP1 or SYNGAP1 that include other genes beyond STXBP1 or SYNGAP1.
* The presence of a significant non-STXBP1-RD or non-SYNGAP1-RD related central nervous impairment/behavioral disturbance that would confound the scientific rigor or interpretation of results of the study.
* History of intraventricular hemorrhage, structural brain deficit or congenital heart disease
* The presence of a clinical comorbidity deemed by the investigator to potentially confound the typical presentation of STXBP1-RD or SYNGAP1-RD.
* Pregnant women or females of age of menarche who are found to be pregnant upon urine pregnancy testing.
What they're measuring
1
Changes in percentiles recorded on clinical assessments over time