Expanded Access to 0.5 mg eRapa for Familial Adenomatous Polyposis
brief summary
This expanded access program provides 0.5 mg eRapa (encapsulated rapamycin) to patients with familial adenomatous polyposis (FAP) who have no satisfactory alternative treatment options and are not eligible to participate in a clinical trial. The objective is to provide access to eRapa based on the treating physician's assessment that the potential benefits outweigh the potential risks, with appropriate clinical monitoring for safety and tolerability.
detailed description
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.
official title
Expanded Access Program for 0.5 mg eRapa in Familial Adenomatous Polyposis