Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
NCT ID: NCT04987463
Last Updated: 2024-02-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
60 participants
INTERVENTIONAL
2021-05-07
2026-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
PREVENTION
DOUBLE
Study Groups
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Vigabatrin arm
Vigabatrin in capsules co-administered with placebo in liquid.
Vigabatrin
Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.
Placebo
Placebo in liquid administered orally, once daily, in the morning. The starting dose of placebo in liquid will be calculated according to the body weight of the patient measured at V1.
Rapamycin arm
Rapamycin in liquid co-administered with placebo in capsules.
Rapamycin
Rapamycin in liquid administered orally, in the morning, every other day or daily depending on the patient's body weight. The starting dose of rapamycin will be calculated according to the body weight of the patient measured at V1.
Placebo
Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.
Interventions
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Vigabatrin
Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.
Rapamycin
Rapamycin in liquid administered orally, in the morning, every other day or daily depending on the patient's body weight. The starting dose of rapamycin will be calculated according to the body weight of the patient measured at V1.
Placebo
Placebo in liquid administered orally, once daily, in the morning. The starting dose of placebo in liquid will be calculated according to the body weight of the patient measured at V1.
Placebo
Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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 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.
4 Weeks
16 Weeks
ALL
No
Sponsors
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Katarzyna Kotulska
OTHER
Responsible Party
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Katarzyna Kotulska
Head of the Department of Neurology and Epileptology at The Children's Memorial Health Institute
Principal Investigators
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Katarzyna Kotulska-Jozwiak
Role: PRINCIPAL_INVESTIGATOR
The Children's Memorial Health Institute
Locations
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Medical University of Warsaw, Department of Pediatric Neurology
Warsaw, , Poland
Children's Memorial Health Institute, Neurology and Epileptology
Warsaw, , Poland
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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ViRap
Identifier Type: -
Identifier Source: org_study_id
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