Phase 2
N=84
Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Tuberous Sclerosis Complex
Bottom Line
View on ClinicalTrials.gov: NCT02849457 ↗Enrolled (actual)
84
Serious AEs
29.5%
Results posted
Aug 2024
Primary outcome: Primary: Cognitive Assessment Scores and Developmental Impact — 83.9; 80.9; 97.3; 85.0 score on a scale — p=0.8681
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Early Vigabatrin (Drug); Delayed Vigabatrin (Placebo) (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Martina Bebin
- Primary completion
- Apr 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Cognitive Assessment Scores and Developmental Impact |
83.9; 80.9; 97.3; 85.0 | 0.8681 |
| SECONDARY Number of Subjects That Develop Seizures When Treated With Study Drug During the Randomized Phase of the Study. |
19; 20 | 0.7375 |
| SECONDARY Time to the Subject's First Clinical Seizure From Randomization |
2.77; 9.47 | 0.1174 |
| SECONDARY Count of Participants With Drug Resistant Epilepsy at 24 Months of Age. |
14; 14 | 0.4653 |
| SECONDARY Evaluate Vineland II ABC Scores and Impact of Early Versus Late Treatment |
86.9; 86.0; 97.8; 86.3; 90.6; 84.9 | 0.1697 |
| SECONDARY Evaluate Autism Diagnostic Observation Schedule 2nd Edition (ADOS2) Scores and Impact of Early Versus Late Treatment |
— | — |
| SECONDARY Number of Subjects With Vigabatrin Related Adverse Events and Severe Adverse Events |
6; 2 | — |
| SECONDARY EEG Biomarker for Developing Epilepsy |
19; 20; 0; 4 | — |
Summary
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure
Eligibility Criteria
Inclusion Criteria
- less than or equal to 6 months of age
- No history of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
- Meet genetic or clinical diagnostic criteria for TSC, the latter based on current recommendations for diagnostic evaluation, such as physical exam, neuroimaging, echocardiogram
Exclusion Criteria
- Is greater than 6 months of age
- Has not been diagnosed with TSC
- History of seizures or infantile spasms, or evidence of subclinical electrographic seizures on a previous video EEG
- Has received any anticonvulsant medication including vigabatrin, other anti-seizure therapeutic agent including cannabidiol
- Has received an oral mTOR inhibitor such as everolimus or sirolimus
- Has taken an investigational drug, including but not limited to cannabidiol, as part of a research study 30 days prior to enrollment, or plans on taking an investigational drug at any time during the duration of the study
- Is currently enrolled, or plans on enrolling at any time during the duration of the study, in an experimental behavioral early intervention study
- Has a history of being born prematurely (born less than <30 weeks gestation at the time of delivery)
Data sourced from ClinicalTrials.gov (NCT02849457). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.