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Open-label study to assess safety and efficacy of rapamycin in drugresistant epilepsy associated with mTOR pathway pathologies
EU CTIS ID: 2024-515950-25-00
What this study is testing
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.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- Severe coexisting diseases, e.g. renal failure, immunodeficiency
- Non-healing wound, unhealed fracture
- Radiation therapy of the CNS <28 days
- Pregnancy, breastfeeding
- Active participation in another research program or completed <30 days
- Systemic treatment with an mTOR inhibitor from other indications in the 3 months preceding study enrollment
See the full eligibility criteria
- 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.
- Severe coexisting diseases, e.g. renal failure, immunodeficiency
- Non-healing wound, unhealed fracture
- Radiation therapy of the CNS <28 days
- Pregnancy, breastfeeding
- Active participation in another research program or completed <30 days
- Systemic treatment with an mTOR inhibitor from other indications in the 3 months preceding study enrollment
- Use of marijuana or its derivatives
- Live vaccine intake <6 weeks prior to study inclusion
- Receipt of inactivated vaccine or mRNA vaccine <4 weeks prior to study inclusion
- Tuberous sclerosis
- Clinically significant disease of the cardiovascular system, e.g. arrhythmia
- Earlier diagnosis of neoplasm <5 years requiring antitumor treatment
- Active infection requiring general treatment
- Contraindications to use, including the inability to take or known hypersensitivity to the drug used in the study
- Simultaneous administration of drugs that are strong inhibitors of CYP3A or PgP
- Radiological features of active CNS bleeding except for asymptomatic resolving changes after biopsy or point bleeding in the tumor
- 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.