Phase 2
N=5
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
Tuberous Sclerosis Complex · Epilepsy
Bottom Line
View on ClinicalTrials.gov: NCT04595513 ↗Enrolled (actual)
5
Serious AEs
0.0%
Results posted
May 2024
Primary outcome: Primary: Safety - Adverse Events — 40 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- TAVT-18 (sirolimus) (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety - Adverse Events |
40 | — |
| PRIMARY Efficacy - Time to Seizure Onset |
102 | — |
| SECONDARY Treatment Discontinuance Due to Adverse Events |
20 | — |
| SECONDARY Treatment Disruption Due to Adverse Events |
19.5 | — |
| SECONDARY Precision Dosing Accuracy |
6.6 | — |
| SECONDARY Age at Seizure Onset |
10.6 | — |
| SECONDARY Seizure Type |
40; 20; 40; 60 | — |
| SECONDARY Seizure Frequency |
0; 0 | — |
| SECONDARY TAND Severity Assessed by the TAND-L Checklist |
2; 2; 1; 3.5 | — |
| SECONDARY Adaptive Behavior Assessed by the the VABS |
91; 88 | — |
| SECONDARY Global Neurodevelopment Assessed by the Bayley Scales of Infant Development |
90; 89; 87; 95; 77; 74 | — |
Summary
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
Eligibility Criteria
Inclusion Criteria
- 0-6 months of age at the time of enrollment (randomization and treatment initiation must occur before 7 months of age and infants born prematurely must have a corrected age of at least 39 weeks, calculated by subtracting the number of weeks born before 40 weeks gestation from the actual chronological age, in weeks)
- Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Exclusion Criteria
- Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG
- Has been treated in the past or is currently being treated at the time of enrollment with conventional anticonvulsant medications (AEDs), systemic (oral) mTOR inhibitors (such as rapamycin, sirolimus, or everolimus), ketogenic-related special diet, or another anti-seizure therapeutic agent, device, or procedure
- Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit
- Has a significant illness or active infection at the time of the baseline screening visit
- Has a history of significant prematurity, defined as gestational age <30 weeks at the time of delivery, or other significant medical complications at birth or during the neonatal period that other than TSC would convey additional risk of seizures or neurodevelopmental delay (i.e. HIE, severe neonatal infection, major surgery, prolonged ventilatory or other life-saving supportive care or procedures)
- Abnormal laboratory values at baseline (i.e., renal function, liver function, or bone marrow production) that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject
- Prior, planned or anticipated neurosurgery within 3 months of the baseline visit
- Has a TSC-associated condition for which mTOR treatment is clinically indicated (i.e. SEGA or AML)
- Subjects who are, in the opinion of the investigator, unable to comply with the requirements of the study
Data sourced from ClinicalTrials.gov (NCT04595513). 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.