2024-515950-25-00CompletedPhase 2
Open-label study to assess safety and efficacy of rapamycin in drugresistant epilepsy associated with mTOR pathway pathologies
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
Source: EU CTIS (euclinicaltrials.eu) · Last updated 11 Jan 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 50 (actual)
- First submitted
- 16 Oct 2024
- Start date
- 27 Jun 2023
- Lead sponsor
- Instytut Pomnik Centrum Zdrowia Dziecka
- Diseases
- Epilepsy
- EU CT number
- 2024-515950-25-00
- EudraCT number
- 2021-006702-78
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 50
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study design.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- 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
Age
- Earlier diagnosis of neoplasm <5 years requiring antitumor treatment
Other
- 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
Who the study is looking for
- 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.
Age in the registry: - to 17 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Instytut Pomnik Centrum Zdrowia Dziecka | Warsaw | Poland | Completed |
This page is information, not medical advice. Discuss any trial with your doctor.