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

Launched by KATARZYNA KOTULSKA · Jul 23, 2021

Keywords

ClinConnect Summary

This clinical trial is studying two medications, rapamycin and vigabatrin, to see how well they work in preventing symptoms of Tuberous Sclerosis Complex (TSC) in infants. TSC is a condition that can cause growths in various organs, including the brain. The trial is currently looking for infants between 4 to 16 weeks old who have been diagnosed with TSC and show some specific brain changes. To participate, parents or caregivers must be able to give consent and follow the study's requirements.

If eligible, participants will be randomly assigned to receive either rapamycin or vigabatrin, and the study will monitor how well these medications work and how safe they are for the infants. It's important to know that certain health conditions or treatments may prevent a child from being in the study, such as having a history of seizures or infections. Overall, this research aims to find better ways to manage TSC symptoms in young children, providing hope for improved treatments in the future.

Gender

ALL

Eligibility 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.