Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Capsida Biotherapeutics, Inc.
Age
0–5
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Male or female, \> 1 day to ≤ 5 years of age at the time of informed consent.
Diagnosed with seizure disorder
Presence of a STXBP1 gene mutation. Historical documentation is sufficient to support eligibility for the study. Confirmatory testing will be obtained at baseline and performed by a CLIA certified laboratory
Written informed consent provided by a parent or legal guardian
You may not be if
History of prior gene therapy treatment
Current enrollment in an interventional study or has received an investigational drug within 30 days or fewer than 5 half-lives prior to screening visit, whichever is longer
Treatment with any antisense oligonucleotide therapy within 6 months prior to screening and anticipate remaining on treatment throughout the study
The presence of a confirmed mutation in a gene other than STXPB1 that is known to contribute to a neurodevelopmental disability
Presence of a significant non-STXBP1 related central nervous impairment/behavioral disturbance that would confound the scientific rigor or interpretation of results of the study
History of prematurity (defined as gestational age \<35 weeks), interventricular hemorrhage, structural brain deficit or congenital heart disease
Requires mechanical ventilation or non-invasive respiratory support such as continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) at the time of informed consent
Clareo Health | Natural History Study in Pediatric Patients with STXBP1 Encephalopathy with Epilepsy
