Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

NCT06950385 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: MTX230-301
• Study Type: interventional
• Target: 168
• Locations: 6 countries
• Sites: 26

Brief Summary

The main goal of this clinical trial is to learn if the drug eRapa works to slow down the progression of disease in patients diagnosed with Familial Adenomatous Polyposis (FAP). Researchers will compare eRapa to Placebo. The questions to be answered by this trial are:

• Does taking eRapa help to slow down the progression of the disease in patients with FAP?
• Is eRapa a safe treatment for patients diagnosed with FAP?
• What is the effect of eRapa on the number of polyps found in GI tract of patients diagnosed with FAP?
• How does treatment with eRapa affect a patient's quality of life? Participants will:
• Take eRapa or placebo once per day every other week until disease progresses (gets worse), stops taking part in the trial or dies.
• Visit the clinic once every 3 months for check ups and tests.
• Have an endoscopy at the start of the trial and then every 6 months to check on whether the disease is getting better or worse.

Conditions

Familial Adenomatous Polyposis (FAP)

Keywords

FAP; Polyposis; Polyps; ileal pouch

Outcome Measures

Primary Outcomes (1)

• Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo.

  * Death from any cause * Cancer/high-grade dysplasia * Major FAP-related surgery (e.g., colectomy, proctectomy, total proctocolectomy with ileal pouch anal anastomosis \[IPAA\], pouch resection, ileostomy, duodenectomy, or surgical ampullectomy) * Advancement of Spigelman stage (not related solely to increase in polyp number) * Meets criteria for surgery (consistent with United States \[US\] and European Union \[EU\] practice guidelines) (Yang, Gurudu et al. 2020, Zaffaroni, Mannucci et al.2024) * Retained rectum/sigmoid or pouch (≥10 polyps ≥3 mm in size at baseline) * Duodenum (Stage 3/4 and at least 1 polyp ≥10 mm removed in last 18 months)

  3 years

Secondary Outcomes (6)

• The safety and tolerability of eRapa in patients with FAP

  3 years

• The effect of eRapa treatment on GI polyposis in patients with FAP

  3 years

• The effect of eRapa treatment on Spigelman stage score in patients with FAP

  3 years

• The effect of eRapa treatment on quality-of-life measures, assessed by the 5 level EuroQoL-5 Dimension (EQ-5D-5L)

  3 years

• Determine the immunomodulating effect of eRapa treatment in patients with FAP

  3 years

Study Arms (2)

eRapa

EXPERIMENTAL

0.5 mg eRapa once a day (QD) every other week

Drug: eRapa (encapsulated rapamycin)

Placebo

PLACEBO COMPARATOR

Placebo once a day (QD) every other week

Drug: Placebo

Interventions

eRapa (encapsulated rapamycin) DRUG

0.5 mg capsules for oral use; white opaque capsule filled with off-white powder; Trial intervention will be provided in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.

eRapa

Placebo DRUG

Capsules in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must be ≥18 years of age inclusive.
• Participant must have documented FAP, confirmed by adenomatous polyposis coli genotype mutation testing.
• Participant must have at least 1 of the following high-risk features: \>100 polyps but ≤500 polyps in the colon, or ≥10 polyps in the retained rectum/sigmoid or ileal pouch (≥3 mm in size), or Spigelman stage 3 or 4 with at least 1 polyp ≥10 mm to be removed at baseline or on endoscopy performed within 18 months of screening.
• Contraceptive use by participants or participant partners until at at least 12 weeks after stopping study treatment.
• Agree not to donate gametes for the purpose of reproduction until at at least 12 weeks after stopping study treatment.
• Willing to undergo endoscopic evaluation.

You may not qualify if:

• Participant has unresected or incompletely resected high-grade dysplasia or cancer within the duodenum, colon, rectum, or ileal pouch at screening endoscopy.
• Participant has any polyps ≥8 mm in the duodenum, colon, rectum, or ileal pouch remaining after screening endoscopy (polyps ≥8 mm are to be resected during screening endoscopy).
• Participant has had surgery within 6 weeks of the trial.
• Participant has active malignancy or history of malignancy diagnosed within 24 months of first dose of trial intervention.
• Participant has a history of, or currently has, an acquired or primary (congenital) immunodeficiency.
• Participant has active and clinically significant tuberculosis (positive Quantiferon Gold test), bacterial, fungal, or viral infection, including human immunodeficiency virus (HIV).
• Participant has any medical or social condition that, in the opinion of the Investigator, might increase participant risk if enrolled, prevent participant compliance to trial procedures, or present an unacceptable confound to safety or clinical trial data.

Sponsors & Collaborators

• Rapamycin Holdings Inc.: lead
• Biodexa Pharmaceuticals: collaborator

Study Sites (26)

City of Hope

Arcadia, California, 91007, United States

NOT YET RECRUITING

Yale Cancer Center

New Haven, Connecticut, 06520, United States

RECRUITING

Georgetown University

Washington D.C., District of Columbia, 20057, United States

RECRUITING

Digestive & Liver Center of Florida

Orlando, Florida, 32825, United States

RECRUITING

Cleveland Clinic Florida

Weston, Florida, 33331, United States

RECRUITING

University of Chicago

Chicago, Illinois, 60615, United States

NOT YET RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21205, United States

RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

NOT YET RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

Department of Surgery, Section of Colon Rectal and Surgery

St Louis, Missouri, 63110, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Ohio State University

Columbus, Ohio, 43210, United States

RECRUITING

Geisinger Medical Center

Danville, Pennsylvania, 17822, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Benaroya Research Institute at Virginia Mason

Seattle, Washington, 98101, United States

RECRUITING

University of Washington - Fred Hutchinson

Seattle, Washington, 98195, United States

RECRUITING

Copenhagen University Hospital

Copenhagen, Denmark

RECRUITING

Universitätsklinikum Bonn

Bonn, Germany

RECRUITING

Amsterdam UMC

Amsterdam, Netherlands

RECRUITING

Radboud University Medical Center

Nijmegen, Netherlands

RECRUITING

Hospital Oncologico - Puerto Rico Medical Center

Rio Piedras, 00935, Puerto Rico

RECRUITING

Hospital Clínic de Barcelona

Barcelona, Spain

RECRUITING

Hospital Comarcal de Inca

Inca, Spain

NOT YET RECRUITING

Hospital La Fe de Valencia

Valencia, Spain

RECRUITING

MeSH Terms

Conditions

Adenomatous Polyposis Coli; Polyps

Condition Hierarchy (Ancestors)

Adenomatous Polyps; Adenoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Colorectal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplastic Syndromes, Hereditary; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Intestinal Diseases; Intestinal Polyposis; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Gary Shangold, MD

  Biodexa

  STUDY DIRECTOR

Central Study Contacts

Vice President Clinical Operations

CONTACT

XXX-XXX-XXXX; jim.kostka@biodexapharma.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2025
• First Posted: April 30, 2025
• Study Start: July 18, 2025
• Primary Completion (Estimated): July 1, 2030
• Study Completion (Estimated): January 1, 2031
• Last Updated: March 17, 2026

Record last verified: 2026-03

Locations

US (18); NL (2); ES (3); DE (1); PR (1); DK (1)