Access to 0.5 mg eRapa for Familial Adenomatous Polyposis Patients

Sponsor: Biodexa Pharmaceuticals

Expanded Access
Sponsor score: 0

ClinicalTrials ID:NCT07689942

Provider View

This program offers access to eRapa, a form of rapamycin, for patients with familial adenomatous polyposis (FAP) who have no other treatment options and cannot join a clinical trial. The goal is to provide eRapa if the doctor believes the benefits outweigh the risks, with careful monitoring for safety.

Patient Parameters

Program Overview

This program is designed to provide eRapa, a medication that targets a specific pathway involved in cell growth, to patients with familial adenomatous polyposis (FAP). FAP is a genetic condition that leads to the development of many polyps in the colon and an increased risk of colorectal cancer. While surgery can reduce cancer risk, polyps often continue to form. eRapa may help by inhibiting a pathway linked to tumor growth. This program is for patients who cannot join a clinical trial and have no other treatment options. Doctors will decide on treatment details and monitor patients' health throughout the process.

Description

Familial adenomatous polyposis (FAP) is a genetic condition that causes numerous polyps in the colon and increases the risk of colorectal cancer. Surgery can help reduce this risk, but polyps may still develop in other parts of the digestive system. eRapa, a form of rapamycin, works by blocking a pathway that affects cell growth and survival, which is often overactive in FAP-related tumors. This program provides eRapa to patients with FAP who cannot participate in clinical trials and have no other treatment options. Doctors will determine the treatment plan and monitor patients for safety and effectiveness.

Eligibility Criteria

Inclusion Criteria

  • Must have completed puberty and reached full adult height.
  • Must have FAP confirmed by genetic testing or a family history of FAP.
  • Must have a significant number of polyps in the colon or duodenum.

Exclusion Criteria

  • Cannot have existing cancer or severe pre-cancerous changes in the digestive tract that require immediate surgery.
  • Cannot have immune system deficiencies or active infections like tuberculosis or HIV.
  • Cannot have significant liver or kidney problems.
  • Cannot have high levels of fats in the blood or a history of pancreatitis.
  • Cannot be taking certain medications that affect how eRapa works.
  • Cannot be allergic to rapamycin or its ingredients.
  • Must agree to use effective birth control and not donate sperm or eggs during treatment and for 12 weeks after.
  • Cannot be pregnant or trying to become pregnant.
  • Must agree to regular endoscopic check-ups while on eRapa.
  • Cannot have specific genetic mutations in the MUTYH gene.

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