Tuberous Sclerosis ComplexPhase 2/3ViRapNCT04987463
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
A Phase 2/3 study testing Vigabatrin and Rapamycin in people with Tuberous Sclerosis Complex, run by Katarzyna Kotulska at 2 sites in Poland.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 4 to 16 weeks
- Conditions the study is for
- Tuberous Sclerosis Complex
- What is being tested?
- Vigabatrin and Rapamycin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 2 sitesPoland
- Study dates
- 7 May 2021to Dec 2028
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 16 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Poland2 sites, 2 recruiting
- Not yet recruiting
Medical University of Warsaw, Department of Pediatric Neurology
Warsaw, 02-091
Sergiusz Jozwiak, 48 22 3179681, sergiusz.jozwiak@wum.edu.pl
- Recruiting
Children's Memorial Health Institute, Neurology and Epileptology
Warsaw, 04-730
Katarzyna Kotulska-Jozwiak, +48 22 8157404, k.kotulska@ipczd.pl
About this study
Official title: 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
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).
Read the detailed description
This is a two-arm, randomized, double-blind and double-dummy, placebo controlled study to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with TSC. The study consists of 3 phases for each patient: screening, core blinded phase, and open-label follow-up phase. Patients who meet the eligibility criteria will be randomized to receive vigabatrin or rapamycin. The randomization ratio is 1:1. Randomization will be stratified by the sex and the presence of epileptiform activity on baseline videoEEG (video electroencephalography) recording (yes versus no). Approximately 60 infants are planned to be enrolled in the study.
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- Vigabatrin, Rapamycin, Placebo
- Keywords
- tuberous sclerosis complex, epilepsy, rapamycin, vigabatrin, tumors, prevention
- Medical subject headings
- Tuberous Sclerosis, Epilepsy, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VigabatrinDrug
Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.
RapamycinDrug
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.
PlaceboDrug
Also called Placebo in liquid
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.
PlaceboDrug
Also called Placebo in granules
Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.
| Study group | Role | What they receive |
|---|---|---|
| Vigabatrin arm | Experimental | Vigabatrin in capsules co-administered with placebo in liquid. Receives: Drug: Vigabatrin; Drug: Placebo |
| Rapamycin arm | Experimental | Rapamycin in liquid co-administered with placebo in capsules. Receives: Drug: Rapamycin; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of clinical seizures in the blinded phase of the study,
Measured: 730 days
Summarized volume of TSC-associated tumors ≥ 125% of initial value within the blinded phase of the study
Measured: 730 days
Secondary outcomes (8)
Total volume of TSC-associated tumors within the blinded phase and the whole study
Measured: 730 days
The risk for high risk of autism assessed with psychological test at 6, 12, 18, 24 months
Measured: 6, 12, 18, 24 months
The risk for low developmental quotient (< 70 points in Bayley Scales of Infant Development, measured at the end of the blinded phase and at the end of the entire study) at the end of the study
Measured: 730 days
The risk of drug-resistant epilepsy at any point of the study
Measured: 730 days
Occurrence of adverse events within the blinded phase of the study
Measured: 730 days
Number of adverse events across the whole study
Measured: 730 days
Parameters of physical development (weight gain history) across the whole study
Measured: 730 days
Parameters of physical development (height gain history) across the whole study
Measured: 730 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2021
- Study start7 May 2021
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Katarzyna Kotulska
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Katarzyna Kotulska, Children's Memorial Health Institute, Poland
- Investigators
- Katarzyna Kotulska-Jozwiak, Principal investigator, The Children's Memorial Health Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04987463
- Sponsor's protocol ID
- ViRap
- Acronym
- ViRap
- Status last verified
- Oct 2026
- First submitted
- 28 May 2021
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No