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- Essai clinique NCT07689942
Expanded Access to 0.5 mg eRapa for Familial Adenomatous Polyposis (eRapa-FAP)
Expanded Access Program for 0.5 mg eRapa in Familial Adenomatous Polyposis
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
amilial adenomatous polyposis (FAP) is an inherited cancer predisposition syndrome characterized by the development of numerous colorectal adenomas and an increased risk of colorectal and other gastrointestinal malignancies. Although prophylactic surgery substantially reduces the risk of colorectal cancer, patients frequently continue to develop adenomas in the remaining gastrointestinal tract, and effective medical therapies to reduce polyp burden are limited.
Rapamycin inhibits the mammalian target of rapamycin (mTOR) signaling pathway, which regulates cell growth, proliferation, and survival. Dysregulation of mTOR signaling has been implicated in intestinal tumorigenesis associated with FAP, providing a biological rationale for mTOR inhibition as a therapeutic approach.
This expanded access program is intended to provide treatment with 0.5 mg eRapa (encapsulated rapamycin) for eligible patients with FAP when participation in a clinical trial is not feasible or available and no comparable or satisfactory therapeutic alternatives exist. Treatment decisions, dosing, duration of therapy, and clinical monitoring will be determined by the treating physician in accordance with the program inclusion and exclusion policy. Patients will undergo appropriate assessments to monitor safety, tolerability, and clinical status throughout treatment.
Type d'étude
Type d'accès étendu
- Patients individuels
Contacts et emplacements
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
- Enfant
- Adulte
- Adulte plus âgé
Accepte les volontaires sains
La description
Inclusion Criteria:
- Patient is post pubertal and reached full adult height
- Patient has FAP confirmed by APC genotype mutation tesing or a history of FAP in one of the parents. Genetic mosaics or Attenuated (as well as classical) FAP may participate
- Patient has significant colorectal and/or duodenal FAP disease burden.
Exclusion Criteria:
- Patient has existing carcinoma or high-grade dysplasia in the GI tract and/or requires imminent definitive surgical intervention (either partial or complete colectomy or duodenectomy).
- Patient with an acquired or congenital immunodeficiency, active and clinically significant tuberculosis, bacterial, fungal, or viral infections, including HIV.
- Patient has clinically significant elevations of hepatic enzymes or bilirubin, or other evidence of active hepatitis.
- Patient has clinically significant impairment of renal function.
- Patient has a history or evidence of clinically significant hyperlipoproteinemia or hypertriglyceridemia.
- Patient has active or recurrent bouts of pancreatitis, or clinically significant elevations of lipase or amylase.
- Patient is taking medications that are considered strong inducers or inhibitors of cytochrome P450 (CYP) 3A4/5 or strong inducers or inhibitors of P-glycoprotein 1 (P-gp1) that cannot be discontinued at least 1 week prior to first dose of treatment intervention and for the duration of the treatment.
- Patients with known hypersensitivity to rapamycin (sirolimus) or any of the excipients in eRapa.
- Sexually active patients who are unwilling to use a highly effective contraceptive method and to refrain from donating gametes (sperm or oocytes) throughout the time they are receiving eRAPA and for 12 weeks beyond that time, and to refrain from breast-feeding their children while on treatment.
- Patients who are pregnant or who are trying to become pregnant while taking eRapa.
- Patients unwilling or unable to undergo continued endoscopic surveillance in accordance with standards of care while taking eRapa.
- Patient has Mutations in the MUTYH gene.
Plan d'étude
Comment l'étude est-elle conçue ?
Collaborateurs et enquêteurs
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Publications et liens utiles
Liens utiles
Dates d'enregistrement des études
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
- Tumeurs par site
- Tumeurs
- Maladies génétiques, innées
- Maladies intestinales
- Tumeurs par type histologique
- Tumeurs gastro-intestinales
- Tumeurs du système digestif
- Maladies du système digestif
- Maladies gastro-intestinales
- Tumeurs colorectales
- Tumeurs intestinales
- Tumeurs, glandulaires et épithéliales
- Maladies du côlon
- Adénome
- Syndromes néoplasiques, héréditaires
- Polypes adénomateux
- Polypose intestinale
- Maladies et anomalies congénitales, héréditaires et néonatales
- Polypose Adénomateuse Coli
- Produits chimiques organiques
- Macrolides
- Lactones
- Sirolimus
Autres numéros d'identification d'étude
- MTX230-003
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