Rapamune 1 mg/mL oral solution clinical trials
5 indexed studies · 0 currently recruiting
- Ongoing, recruitingTherapeutic exploratory (Phase II)
An open randomized phase II clinical trial evaluating the safety and efficacy of rapamycin in the treatment of gliomas high-grade malignant gliomas in children as part of the establishment management of rare and ultra rare diseases of the central nervous system associated with mTOR pathway activation: BraimTOR- ONKO
This study is testing a medicine called Rapamune for children with a serious type of brain tumour. Doctors want to see how safe the medicine is and if it helps children live longer or if their tumour gets smaller. It's a key step in finding better treatments for rare conditions.
For: high grade gliomaPoland - AuthorisedPhase I and Phase II (Integrated)- First administration to humans
A Phase I/II, open-label, dose escalation study to evaluate the safety of 2 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (limb-girdle muscular dystrophy LGMDR5, formerly LGMD2C).
This study is testing a new gene therapy for a rare muscle condition called limb-girdle muscular dystrophy type R5 (LGMD-R5). It aims to see if the treatment is safe, what dose works best, and if it helps improve muscle function.
For: limb-girdle muscular dystrophy type R5 (previously named LGMD2C)France · Italy - Ongoing, recruitingTherapeutic exploratory (Phase II)
TOPical sirolimus in linGUal microkystic lymphatic malformation
This study is testing a skin cream with sirolimus for a rare condition called lingual microcystic lymphatic malformation (LMLM), which causes cysts on the tongue. Doctors want to see if the cream can reduce symptoms like swelling, bleeding, and pain to improve patients' quality of life.
For: Lingual microcystic lymphatic malformations (LMLMs) are rare congenital vascular malformations · presenting as clusters of cysts filled with lymph fluid or blood. They are responsible for a heavy burden even with small well-limited lesions because of oozing · bleedingFrance - AuthorisedTherapeutic exploratory (Phase II)
RAPAMALYMPH - Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
This study looks at how a medicine called Rapamycin treats a rare growth of blood and fluid vessels, called a lymphatic malformation, in the head and neck area that is difficult to treat. We want to see if the medicine can shrink the size of these growths.
For: Poor prognosis cervico-facial lymphatic malformations (Lymphangima)France - Ongoing, recruitingTherapeutic exploratory (Phase II)
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
This study is looking at a medicine called rapamycin for children whose epilepsy is difficult to treat. We want to see how safe the medicine is and if it can reduce seizures in children with rare brain conditions linked to a specific pathway called mTOR.
For: epilepsy · mTORopathies · focal cortical dysplasiaPoland