Clinical trial · Interventional
Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants
NCT04595513CI-TRIAL-00077192STOP2completedPhase 1 / Phase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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).
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Epilepsy | — | UNRESOLVED | — |
| Tuberous Sclerosis Complex | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| TAVT-18 (sirolimus) | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Stage 1 Open Label
- description
- Phase I/II, open-label PK and initial safety analysis. TAVT-18 administered orally twice/daily to achieve precision dosing target of 10 ng/ml. Whole blood sirolimus levels are assessed at defined intervals on days 1, 7, and 14. After day 14, participants can elect to continue open-label treatment with TAVT-18 until 12 months of age. Final developmental outcomes are assessed at 24 months of age.
- interventionNames
- Drug: TAVT-18 (sirolimus)
Primary outcomes (2)
- measure
- Safety - Adverse Events
- timeFrame
- 12 months of age
- description
- Percentage of subjects reporting severe (CTCAE v5.0 grade \>= 3) adverse event (AE) or serious adverse event (SAE)
- measure
- Efficacy - Time to Seizure Onset
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Day
- Maximum age
- 6 Months
Show eligibility criteria text
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
References
Publications (1)
- DERIVEDMasuda S, Lemaitre F, Barten MJ, Bergan S, Shipkova M, van Gelder T, Vinks S, Wieland E, Bornemann-Kolatzki K, Brunet M, de Winter B, Dieterlen MT, Elens L, Ito T, Johnson-Davis K, Kunicki PK, Lawson R, Lloberas N, Marquet P, Millan O, Mizuno T, Moes DJAR, Noceti O, Oellerich M, Pattanaik S, Pawinski T, Seger C, van Schaik R, Venkataramanan R, Walson P, Woillard JB, Langman LJ. Everolimus Personalized Therapy: Second Consensus Report by the International Association of Therapeutic Drug Monitoring and Clinical Toxicology. Ther Drug Monit. 2025 Feb 1;47(1):4-31. doi: 10.1097/FTD.0000000000001250. Epub 2024 Sep 25. PMID 39331837