An observational study in STXBP1 Encephalopathy with Epilepsy, sponsored by Capsida Biotherapeutics, Inc.. Withdrawn. Open to participants aged 1 Day to 5 Years. Per ClinicalTrials.gov, last updated 2024-10-18.
Sponsored by Capsida Biotherapeutics, Inc. · Observational
This is a prospective, non-interventional, longitudinal study designed to characterize the natural history of STXBP1 related encephalopathy with epilepsy, in participants ≤ \<5 years of age.
1,842 studies on the registry are indexed under Epilepsy; 423 are open to participants now.
Browse Epilepsy studies →Capsida Biotherapeutics, Inc. is the lead sponsor of 3 studies on the registry; none are open to participants now.
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≤5 years of age with STXBP1 related encephalopathy with epilepsy.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Changes in neurodevelopmental parameters of adaptive behavior function over time utilizing the Vineland Adaptive Behavior Scales-II (VABS-II) Age-equivalent Scores
The Vineland Adaptive Behavior Scales, Second Edition (Survey Interview Form) is a measure of adaptive behavior in children, adolescents and adults. It yields an overall standard score (Adaptive Behavior Composite, ABC) and age standard scores in four domains. ABC scores have a mean of 100 and a standard deviation of 15 (range = 20 to 160). Higher scores suggest a higher level of adaptive functioning.
Time frame: 2 Years
Changes in neurodevelopment parameters over time utilizing an age appropriate assessment.
The determination of whether a patient received Bayley Scales of Infant Development-III (BSID-III) is based on an algorithm that includes the patient's calendar age and VABS-II age-equivalent score (See Outcome 1). The BSID-III is a series of measurements to assess the motor (fine and gross), language (receptive and expressive), and cognitive development of infants and toddlers and consists of a series of developmental play tasks. contains two scoring systems of composite scores and percentile ranks. The normal range for composite scores is between 40-160 with mean at 100 and 0-99 for the percentile ranks. Higher scores mean a better outcome.
Time frame: 2 Years
Changes in neurodevelopment parameters over time utilizing an age appropriate assessment.
The determination of whether a patient received Kaufman Assessment Battery for Children-II (KABC-II) is based on an algorithm that includes the patient's calendar age and VABS-II age-equivalent score (See Outcome 1). The KABC-II is an individually administered measure of processing and reasoning abilities. The scoring range is 69 and below through 131 or greater. Higher scores mean a better outcome.
Time frame: 2 Years
Changes in seizure frequency over time
Seizure diary
Time frame: 2 Years
Changes in communication ability over time
Observer-Reported Communication Ability (ORCA) Outcome Measure. The ORCA measure produces a single score that is an estimate of an individual's overall level of communication ability. Higher ORCA scores reflect greater communication ability; the mastery of expressive, receptive, and pragmatic types of communication and higher vocabularies for verbal words and symbols on assistive devices. The ORCA T-score range is from 26.82 to 83.24.
Time frame: 2 Years
Changes in sleep behavior over time
Patient sleep habits will be assessed using Children's Sleep Habits Questionnaires (CSHQ).
Time frame: 2 Years
Changes in electroencephalogram (EEG) recording over time
Standard EEG, lasting approximately 1 hour, to assess the presence of epileptiform activity.
Time frame: 2 Years
Changes in motor function over time utilizing an age appropriate assessment.
The Hammersmith Infant Neurological Examination (HINE) is aimed to be used for infants between 3 and 24 months of age and assess different aspects of neurological function: cranial nerve function, movements, reflexes and protective reactions and behavior, as well as some age-dependent items that reflect the development of gross and fine motor function. Each item is scored 0-3, higher scores mean a better outcome.
Time frame: 2 Years
Changes in motor function over time utilizing an age appropriate assessment.
The Gross Motor Function Measure-88 is aimed to be used for children 5 months or older of age and assess changes in gross motor function over time. Each item is scored 0-3, higher scores mean a better outcome.
Time frame: 2 Years
No study locations are listed for this record.
Plan to share: Undecided
No publications or documents are linked to this record.
Start is estimated for Oct 2024; primary completion for Jan 2026, 15 months later.
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This study is withdrawn, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
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Capsida Biotherapeutics, Inc.