Open-label study to assess safety and efficacy of rapamycin in drugresistant epilepsy associated with mTOR pathway pathologies
Key facts
- Where
- Warsaw, LengyelországSee the site
- Phase
- 2. fázis
Where in Lengyelország
- Instytut Pomnik Centrum Zdrowia DzieckaWarsaw, LengyelországNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Instytut Pomnik Centrum Zdrowia Dziecka
- Trial code
- EU 2024-515950-25-00
- Record updated
- 2025. január 11.
- Original description
The primary objective of the study is to determine safety and tolerability of rapamycin in a drug resistant epilepsy associated with rare and ultrarare diseases of the central nervous system associated with the activation of the mTOR pathway.Read full original descriptionShow less
Open, randomized, IInd phase clinical study evaluating the safety and efficacy of rapamycin in the treatment of drug-resistant epilepsy in children with rare and ultra rare diseases of the central nervous system associated with the activation of the mTOR pathway : BraimTOR-NEURO
The primary objective of the study is to determine safety and tolerability of rapamycin in a drug resistant epilepsy associated with rare and ultrarare diseases of the central nervous system associated with the activation of the mTOR pathway.
Inclusion: Age at inclusin from 4 months to 18 years; Diagnosis of drug-resistant epilepsy as defined by ILAE (Kwan 2010), with focal onset (ICD-10: G40.2), with confirmed coexistence or suspicion of mTORopathy in MRI, including: FCD, hemimegalencephaly, LEATs; active epilepsy at study entry, at least 8 seizures in the prior 4 weeks prior to study entry; Informed consent to the participation in the study by the patient's legal caregivers and the patient himself, if he is 13 years of age or older; Negative result of the pregnancy test performed within 2 weeks prior to the start of the study in patients of reproductive age; Written consent to the use of effective contraception in sexually active patients or an obligation to abstaining from sexual activity during and for 3 months after its treatment.
Exclusion: Radiation therapy of the CNS <28 days; Systemic treatment with an mTOR inhibitor from other indications in the 3 months preceding study enrollment; Live vaccine intake <6 weeks prior to study inclusion; Receipt of inactivated vaccine or mRNA vaccine <4 weeks prior to study inclusion; Simultaneous administration of drugs that are strong inhibitors of CYP3A or PgP; Major surgery, open biopsy <28 days prior to treatment initiation; Minor surgical procedures <2 days before the start of treatment, e.g. implantation of a vascular port; Severe coexisting diseases, e.g. renal failure, immunodeficiency; Clinically significant disease of the cardiovascular system, e.g. arrhythmia; Active infection requiring general treatment; Contraindications to use, including the inability to take or known hypersensitivity to the drug used in the study; Radiological features of active CNS bleeding except for asymptomatic resolving changes after biopsy or point bleeding in the tumor; Earlier diagnosis of neoplasm <5 years requiring antitumor treatment; Non-healing wound, unhealed fracture; Pregnancy, breastfeeding; Active participation in another research program or completed <30 days; Use of marijuana or its derivatives; Tuberous sclerosis.