Clinical trial · Interventional
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Randomized, Placebo-controlled, Double-blind and Double-dummy Clinical Trial Comparing the Safety, Tolerability, and Efficacy of Vigabatrin and Rapamycin in a Preventive Treatment of Infants With Tuberous Sclerosis Complex
NCT04987463CI-TRIAL-00073851ViRapunknownPhase 2 / Phase 3ClinicalTrials.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)
The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Tuberous Sclerosis Complex | — | UNRESOLVED | — |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Placebo | Drug | — | UNRESOLVED |
| Rapamycin | Drug | Sirolimus | ALIAS |
| Vigabatrin | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Vigabatrin arm
- description
- Vigabatrin in capsules co-administered with placebo in liquid.
- interventionNames
- Drug: Vigabatrin
- Drug: Placebo
- type
- EXPERIMENTAL
- label
- Rapamycin arm
- description
- Rapamycin in liquid co-administered with placebo in capsules.
- interventionNames
- Drug: Rapamycin
- Drug: Placebo
Primary outcomes (2)
- measure
- Occurrence of clinical seizures in the blinded phase of the study,
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 4 Weeks
- Maximum age
- 16 Weeks
Show eligibility criteria text
Inclusion Criteria: * Male or female aged from 4 up to 16 weeks (44-56 weeks of gestational age) at the day of randomization * Parents/caregivers are willing to and able to give informed consent form for the participation in the study * Parents/caregivers are willing to and able to comply with all study requirements * Definite diagnosis of TSC according to the Consensus criteria (Northrup,2013) * At least 1 focus of cortical dysplasia disclosed on brain MRI Exclusion Criteria: * history of seizures prior to randomization, * history of antiepileptic treatment, * history of treatment with mTOR (mammalian Target of Rapamycin) inhibitor, * gestational age below 44 weeks at the day of randomization, * body weight lower than 3 kg at the day of randomization, * SEGA (Subependymal Giant Cell Astrocytoma) or other TSC-associated lesion requiring urgent surgical intervention * recent surgery within 1 month prior to the randomization * intercurrent infection at the date of randomization * known history of HIV seropositivity * live vaccination within 1 month prior to randomization\* * lack of first TBC and hepatitis B vaccinations * Any significant clinical, laboratory , ECG or other abnormalities, comorbidity or concomitant treatment which, in the opinion of the investigator, may either put a patient at significant risk associated with the participation in the study or may influence the results of the study. * Use of an investigational drug within 1 month prior to randomization.
References
Publications (0)
Data not yet available
No reference posted for this study.