Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants

• Status: Recruiting
• Trial end date: 2024-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This trial will use a two stage, phase I/II clinical trial design. The first stage will employ an open-label clinical trial design to verify dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5). Results will then be carried forward to inform appropriate initial dosing, dosing frequency, and dosing adjustments for the second stage, a randomized, double-blind, placebo-controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Treatments: Sirolimus

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