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Familial Adenomatous Polyposis (FAP)Phase 3SERENTANCT06950385

Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

A Phase 3 study testing eRapa (encapsulated rapamycin) in people with Familial Adenomatous Polyposis (FAP), run by Rapamycin Holdings Inc. at 29 sites in 7 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Familial Adenomatous Polyposis (FAP)
What is being tested?
eRapa (encapsulated rapamycin)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
168 participants
Where
29 sitesin 7 countries
Study dates
18 Jul 2025to Jan 2031
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

29 sites in 7 countries. Contact the study team or a site near you to ask about taking part.

Vice President Clinical Operations contact

XXX-XXX-XXXX

jim.kostka@biodexapharma.com

29 sites in 7 countries

United States18 sites, 18 recruiting
  • University of Michigan

    Ann Arbor, Michigan, 48109

    Erika Koeppe, 734-998-1274, eskoeppe@med.umich.edu

    Recruiting
  • City of Hope

    Arcadia, California, 91007

    Gregory Idos, 626-218-2570, gidos@coh.org

    Andrew Fernandez, andfernandez@coh.org

    Recruiting
  • Johns Hopkins University

    Baltimore, Maryland, 21205

    Lisa Datta, 410-614-1982, erapastudy@live.johnshopkins.edu

    Recruiting
  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

    Samantha Kuney, 617-582-9095, ColonScreening@dfci.harvard.edu

    Recruiting
  • University of Chicago

    Chicago, Illinois, 60615

    Sonia Kupfer, 773-702-6140, cancerclinicaltrials@bsd.uchicago.edu

    Kristi Kearney, kristi.kearney@uchicagomedicine.org

    Recruiting
  • Cleveland Clinic

    Cleveland, Ohio, 44195

    Lindsey Reardon, (216) 444-7493, Reardol3@ccf.org

    Recruiting
  • Ohio State University

    Columbus, Ohio, 43210

    Kebire Gofar, 614-600-2113, kebire.gofar@osumc.edu

    Recruiting
  • Geisinger Medical Center

    Danville, Pennsylvania, 17822

    Kay Reiner, 570-214-5421, kmreiner1@geisinger.edu

    Recruiting
  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

    Annie Lincoln, 919-923-4430, aglincoln@mdanderson.org

    Recruiting
  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

    Llana Abella, 913-584-0533, labella@kumc.edu

    Recruiting
  • Yale Cancer Center

    New Haven, Connecticut, 06520

    Edgar Benitez, 2033936591, edgar.benitez@yale.edu

    Recruiting
  • Digestive & Liver Center of Florida

    Orlando, Florida, 32825

    Alisa Del Vecchio, 407-490-4526, alisa.delvecchio@dlcfl.com

    Recruiting
  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213

    Sara Booz, 412-864-7516, boozs@upmc.edu

    Recruiting
  • Benaroya Research Institute at Virginia Mason

    Seattle, Washington, 98101

    Alicia Reyes, (206)-341-1450, mariaalicia.reyes@commonspirit.org

    Recruiting
  • University of Washington - Fred Hutchinson

    Seattle, Washington, 98195

    Brian Forester, (206) 685-1179, foerstb@uw.edu

    Recruiting
  • Department of Surgery, Section of Colon Rectal and Surgery

    St Louis, Missouri, 63110

    Asima Badic, 314- 362-2646, asimabadic@wustl.edu

    Recruiting
  • Georgetown University

    Washington D.C., District of Columbia, 20057

    Priyanth Kanth, 202-444-1431, priyanka.kanth@medstar.net

    Recruiting
  • Cleveland Clinic Florida

    Weston, Florida, 33331

    Michael Nicolas, 954-659-6213, nicolam4@ccf.org

    Recruiting

Other trials in United States

Spain4 sites, 4 recruiting
  • Hospital Clínic de Barcelona

    Barcelona

    Frencesc Balaguer Prunes, fprunes@clinic.cat

    Recruiting
  • Hospital Comarcal de Inca

    Inca

    Jose Reyes Moreno, jose.reyes@hcin.es

    Recruiting
  • Hospital General Universitario Gregorio Marañón

    Madrid

    Luis Miguel Jiménez-Gomez, M.D, +34 915868522, luismiguel.jimenez@salud.madrid.org

    Recruiting
  • Hospital La Fe de Valencia

    Valencia

    Marco Bustamante, bustamante_mar@gva.es

    Recruiting

Other trials in Spain

Italy2 sites, 2 recruiting
  • IRCCS Ospedale San Raffaele

    Milan

    Giulia Martina Cavestro, M.D, XXX-XXX-XXXX, cavestro.giuliamartina@hsr.it

    Recruiting
  • Istituto Nazionale dei Tumori

    Milan

    Marco Vitellaro, M.D, + 39 02 23902540, marco.vitellaro@istitutotumori.mi.it

    Recruiting

Other trials in Italy

Netherlands2 sites, 2 recruiting
  • Amsterdam UMC

    Amsterdam

    Evelien Dekker, e.dekker@amsterdamumc.nl

    Recruiting
  • Radboud University Medical Center

    Nijmegen

    Tanya Bisseling, tanya.bisseling@radboudumc.nl

    Recruiting

Other trials in Netherlands

Denmark1 site, 1 recruiting
  • Copenhagen University Hospital

    Copenhagen

    John Karstensen, john.gasdal.karstensen@regionh.dk

    Recruiting

Other trials in Denmark

Germany1 site, 1 recruiting
  • Universitätsklinikum Bonn

    Bonn

    Robert Hüneburg, robert.hueneburg@ukbonn.de

    Recruiting

Other trials in Germany

Puerto Rico1 site, 1 recruiting
  • Hospital Oncologico - Puerto Rico Medical Center

    Rio Piedras, 00935

    Ramon Vega, 787-407-3333, ramon.vega@panoncologytrials.com

    Recruiting

Other trials in Puerto Rico

About this study

Official title: A Phase 3, Multi-Site, Prospective, Randomized, Double-Blind, Placebo-Controlled Trial of eRapa to Improve Clinical Outcomes in Patients With Familial Adenomatous Polyposis

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?
Read more

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.
Read the detailed description

This is a Phase 3, multi-site, prospective, randomized, double-blind, placebo-controlled trial of eRapa administered to patients with FAP who are at high risk of disease progression. 168 patients with FAP will be enrolled in the trial and randomized 2:1 to receive 0.5 mg eRapa or matching placebo orally, once a day (QD) every other week. There is no minimum treatment duration as this is an event-driven trial; however, the intervention period will continue until disease progression, participant withdrawal from treatment, or until the overall trial endpoint is reached. Participant eligibility is restricted to patients under active surveillance for genetic or clinically diagnosed FAP and who have an intact colon; who are postcolectomy/subtotal colectomy and have documented residual polyps in the rectum/sigmoid or who are post-proctocolectomy with ileal-pouch anal anastomosis and documented polyps in the pouch. Eligible participants will undergo a baseline endoscopy and subsequent endoscopic procedures performed every 6 months to monitor for disease progression.

Randomized patients will be stratified based on the following disease characteristics:

  • Intact colon versus post-surgical resection with retained rectum/sigmoid or pouch, and
  • Duodenal polyposis (current Spigelman stage score ≤2 versus Spigelman stage score ≥3) For the purposes of this trial, high-risk for disease progression is defined as meeting one of the following:
  • Patients who have intact colons and have >100 polyps but ≤500 polyps
  • Patients who have retained rectum/sigmoid or ileal-pouch-anal anastomosis and have ≥10 polyps that are ≥3 mm in diameter, or
  • Patients who have a history of duodenal polyposis Spigelman stage score of 3 or 4 with at least 1 duodenal polyp that has been removed within 18 months of screening.

Trial assessments should be conducted as per the Schedule of Activities with a visit occurring about once every 3 months.

Assessment of Spigelman stage will not require a biopsy unless the lesion has an abnormal appearance and/or is ≥10 mm.

Being tested
eRapa (encapsulated rapamycin), Placebo
Keywords
FAP, Polyposis, Polyps, ileal pouch
Medical subject headings
Adenomatous Polyposis Coli, Polyps

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

  • PlaceboDrug

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

Study groups in NCT06950385
Study groupRoleWhat they receive
eRapaExperimental0.5 mg eRapa once a day (QD) every other week
Receives: Drug: eRapa (encapsulated rapamycin)
PlaceboPlacebo comparatorPlacebo once a day (QD) every other week
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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)

    Measured: 3 years

Secondary outcomes (6)
  1. The safety and tolerability of eRapa in patients with FAP

    -Frequency of all grades (as per the Common Terminology Criteria for Adverse Events [CTCAE] version [v] 5.0) treatment-emergent adverse events (TEAEs) in participants receiving eRapa;

    • Frequency of Grade 3 and higher (as per the CTCAE v5.0) TEAEs in participants receiving eRapa
    • Percentage of participants discontinuing eRapa treatment due to adverse drug reactions (ADRs)

    Measured: 3 years

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

    • Percent change from baseline in the PB (i.e., the sum of the diameters of all polyps >3 mm) at 6, 12, 18, 24, 30, and 36 months as observed by surveillance endoscopy Note: The total PB will be based on polyps observed in the upper GI tract (duodenum) and the lower GI tract (colon, retained rectum/sigmoid or pouch)
    • Percent change from baseline in the PB in the upper GI tract (duodenum) at 6, 12, 18, 24, 30, and 36 months as observed by upper endoscopy
    • Percent change from baseline in the PB in the lower GI tract (colon, retained rectum/ sigmoid or pouch) at 6, 12, 18, 24, 30, and 36 months as observed by lower endoscopy

    Measured: 3 years

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

    Change from baseline in Spigelman stage score at 6, 12, 18, 24, 30, and 36 months

    Measured: 3 years

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

    -Change from baseline in EQ-5D-5L score at 6, 12, 18, 24, 30, and 36 months;

    Measured: 3 years

  5. Determine the immunomodulating effect of eRapa treatment in patients with FAP

    • Change from baseline in T cell phenotypes and function at 12, 24, and 36 months

    Measured: 3 years

  6. The effect of eRapa treatment on quality-of-life measures, assessed by EORTC-30

    -Change from baseline in EORTC-30 score at 6, 12, 18, 24, 30, and 36 months;

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Apr 2025
  2. Study start18 Jul 2025
  3. Primary completionJul 2030 (estimated)
  4. Study completionJan 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
168 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06950385
Sponsor's protocol ID
MTX230-301
Acronym
SERENTA
Status last verified
Jun 2026
First submitted
10 Apr 2025
Last updated
2 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing eRapa (encapsulated rapamycin) in people with Familial Adenomatous Polyposis (FAP), run by Rapamycin Holdings Inc. at 29 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 2 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in United States, Spain, Italy, Netherlands, Denmark and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Vice President Clinical Operations (XXX-XXX-XXXX, jim.kostka@biodexapharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo. (time frame: 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jun 2026.

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