Expanded Access to 0.5 mg eRapa for Familial Adenomatous Polyposis (eRapa-FAP)

June 30, 2026 updated by: Biodexa Pharmaceuticals

Expanded Access Program for 0.5 mg eRapa in Familial Adenomatous Polyposis

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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

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.

Study Type

Expanded Access

Expanded Access Type

  • Individual Patients

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  1. Patient is post pubertal and reached full adult height
  2. 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
  3. Patient has significant colorectal and/or duodenal FAP disease burden.

Exclusion Criteria:

  1. 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).
  2. Patient with an acquired or congenital immunodeficiency, active and clinically significant tuberculosis, bacterial, fungal, or viral infections, including HIV.
  3. Patient has clinically significant elevations of hepatic enzymes or bilirubin, or other evidence of active hepatitis.
  4. Patient has clinically significant impairment of renal function.
  5. Patient has a history or evidence of clinically significant hyperlipoproteinemia or hypertriglyceridemia.
  6. Patient has active or recurrent bouts of pancreatitis, or clinically significant elevations of lipase or amylase.
  7. 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.
  8. Patients with known hypersensitivity to rapamycin (sirolimus) or any of the excipients in eRapa.
  9. 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.
  10. Patients who are pregnant or who are trying to become pregnant while taking eRapa.
  11. Patients unwilling or unable to undergo continued endoscopic surveillance in accordance with standards of care while taking eRapa.
  12. Patient has Mutations in the MUTYH gene.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Biodexa Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

June 30, 2026

First Submitted That Met QC Criteria

June 30, 2026

First Posted (Actual)

July 8, 2026

Study Record Updates

Last Update Posted (Actual)

July 8, 2026

Last Update Submitted That Met QC Criteria

June 30, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MTX230-003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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