Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	4 Weeks - 16 Weeks
Gender	All

More Inclusion & Exclusion Criteria

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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT04987463
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2/Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Katarzyna Kotulska
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Katarzyna Kotulska-Jozwiak
Principal Investigator Affiliation	The Children's Memorial Health Institute
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	Poland
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Tuberous Sclerosis Complex

Additional Details

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.

Arms & Interventions

Arms

Experimental: Vigabatrin arm

Vigabatrin in capsules co-administered with placebo in liquid.

Experimental: Rapamycin arm

Rapamycin in liquid co-administered with placebo in capsules.

Interventions

Drug: - Vigabatrin

Vigabatrin in capsules administered orally, initially (between V1 and V2) once daily in the evening,and starting from V2 administered two times daily.

Drug: - 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.

Drug: - 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.

Drug: - Placebo

Placebo in granules administered orally, initially once daily in the evening,and after reaching the targeted dose administered two times daily.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Warsaw, Poland

Status

Not yet recruiting

Address

Medical University of Warsaw, Department of Pediatric Neurology

Warsaw, , 02-091

Site Contact

Sergiusz Jozwiak

[email protected]

48 22 3179681

Warsaw, Poland

Status

Recruiting

Address

Children's Memorial Health Institute, Neurology and Epileptology

Warsaw, , 04-730

Site Contact

Katarzyna Kotulska-Jozwiak

[email protected]

+48 22 8157404

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