Comparing Rapamycin and Vigabatrin for Preventing Symptoms of Tuberous Sclerosis 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
This study is testing whether the medications rapamycin or vigabatrin can help prevent symptoms in infants with Tuberous Sclerosis.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 4 Weeks to 16 Weeks |
| Sex | All |
| Sponsor | Children's Memorial Health Institute, Poland Academic / other |
| Locations | 2 sites (Warsaw and 1 other locations) |
| Trial ID | NCT04987463 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the efficacy, tolerability, and safety of two medications, vigabatrin and rapamycin, as preventive treatments for infants diagnosed with Tuberous Sclerosis Complex (TSC). It is designed as a randomized, double-blind, and placebo-controlled study, involving three phases: screening, a core blinded phase, and an open-label follow-up phase. Approximately 60 infants will be enrolled, and participants will be randomly assigned to receive either vigabatrin or rapamycin, with randomization stratified by sex and baseline epileptiform activity. The study aims to determine which medication is more effective in preventing symptoms associated with TSC.
Who should consider this trial
Good fit: Ideal candidates for this study are infants aged 4 to 16 weeks with a definite diagnosis of Tuberous Sclerosis Complex and at least one focus of cortical dysplasia on brain MRI.
Not a fit: Patients with a history of seizures prior to randomization or those requiring urgent surgical intervention for TSC-associated lesions may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new preventive treatment option for infants with Tuberous Sclerosis Complex, potentially reducing the severity of symptoms and improving quality of life.
How similar studies have performed: While there have been studies on treatments for Tuberous Sclerosis Complex, the specific comparison of rapamycin and vigabatrin in this preventive context is relatively novel.
Eligibility criteria
Show full 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.
Where this trial is running
Warsaw and 1 other locations
- Medical University of Warsaw, Department of Pediatric Neurology — Warsaw, Poland (Not_yet_recruiting)
- Children's Memorial Health Institute, Neurology and Epileptology — Warsaw, Poland (Recruiting)
Study contacts
- Principal investigator: Katarzyna Kotulska-Jozwiak — The Children's Memorial Health Institute
- Study coordinator: Katarzyna Kotulska-Jozwiak
- Email: k.kotulska@ipczd.pl
- Phone: +48 22 8157404
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.