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

Korábbi kezelés

• history of seizures prior to randomization,
• history of treatment with mTOR (mammalian Target of Rapamycin) inhibitor,
• SEGA (Subependymal Giant Cell Astrocytoma) or other TSC-associated lesion requiring urgent surgical intervention
• recent surgery within 1 month prior to the randomization
• live vaccination within 1 month prior to randomization*
• Use of an investigational drug within 1 month prior to randomization.

Más betegség

• history of antiepileptic treatment,
• intercurrent infection at the date of randomization
• known history of HIV seropositivity
• lack of first TBC and hepatitis B vaccinations

Laborértékek

• 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.

Életkor

• gestational age below 44 weeks at the day of randomization,

Egyéb

• body weight lower than 3 kg at the day of randomization,

• 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