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โ† Trials
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Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants

RecruitingPhase 2, Phase 3Ages 4 Weeks - 16 WeeksAllKatarzyna Kotulska
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • Vigabatrin
  • Rapamycin
  • Placebo
  • Placebo
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Where

2 sites
Medical University of Warsaw, Department of Pediatric Neurology
Warsaw, Poland
Children's Memorial Health Institute, Neurology and Epileptology
Warsaw, Poland
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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