eRapa for Adenomatous Polyposis

(SERENTA Trial)

Recruiting at 6 trial locations

Overseen By Amy Ellenberger

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Rapamycin Holdings Inc.

Disqualifiers: Cancer, Immunodeficiency, Infections, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: Up to 3 years

168 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if the drug eRapa can slow the progression of Familial Adenomatous Polyposis (FAP), a condition that causes many polyps (small growths) to form in the colon and can lead to cancer. Participants will take either eRapa or a placebo to assess eRapa's effectiveness and safety. The trial will evaluate how eRapa affects the number of polyps and the quality of life for those with FAP. It is suitable for individuals diagnosed with FAP who have a certain number of polyps and are willing to undergo regular endoscopies. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Do I need to stop my current medications for the eRapa trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that eRapa is likely to be safe for humans?

Research has shown that eRapa has been tested for safety in people with Familial Adenomatous Polyposis (FAP). In one study, 75% of patients who took eRapa did not experience a worsening of their condition after 12 months, suggesting that eRapa might be safe to use.

eRapa has also received Fast Track Designation from the FDA, indicating a need for new treatments and that early studies showed promising results. Although specific side effects were not mentioned, reaching this stage in research usually means the treatment is considered safe enough for further testing.

This trial is in a later phase, which generally indicates some evidence of eRapa's safety. However, like any treatment, there could be risks, and ongoing research will provide more information about its safety. Participants should consult their healthcare providers about any concerns before joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for FAP?

Unlike the standard treatments for adenomatous polyposis, which typically involve surgical removal of polyps or medications that reduce polyp growth, eRapa offers a unique approach. eRapa is a low-dose formulation of rapamycin, which works by targeting the mTOR pathway—a key player in cell growth and proliferation. Researchers are excited about eRapa because it has the potential to reduce polyp formation with fewer side effects due to its intermittent dosing schedule. This could offer a less invasive and more tolerable alternative for patients managing this condition.

What evidence suggests that eRapa might be an effective treatment for Familial Adenomatous Polyposis?

Research has shown that eRapa, which participants in this trial may receive, may help manage Familial Adenomatous Polyposis (FAP). In one study, 89% of patients taking eRapa did not experience a worsening of their condition, and there was an average 29% decrease in the number of polyps after 12 months. Polyps are small growths that can become cancerous if not treated. Another study found that eRapa helped reduce the size of polyps for many patients. These results suggest that eRapa might slow the disease and lower the number of polyps in people with FAP. ^{1 2 3 6 7}

Who Is on the Research Team?

Vance Sohn, MD

Principal Investigator

LumaBridge

Are You a Good Fit for This Trial?

This trial is for individuals with Familial Adenomatous Polyposis (FAP), confirmed by genetic testing. Participants must have a specific number of polyps in their colon or rectum, or advanced polyposis, and be willing to undergo regular endoscopic evaluations. They should agree not to donate gametes during and up to 12 weeks post-treatment, and use contraception.

Inclusion Criteria

I will use birth control during and for 12 weeks after the study.

I agree not to donate eggs or sperm for 12 weeks after my treatment ends.

I have FAP confirmed by a specific genetic test.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive eRapa or placebo once per day every other week until disease progression, withdrawal, or death

Up to 3 years

Clinic visits every 3 months, endoscopy every 6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• eRapa

Trial Overview The trial is examining if eRapa can slow down FAP progression compared to a placebo. Patients will take the drug or placebo every other week until disease progression, withdrawal from the study, or death. They'll visit the clinic quarterly for check-ups and have biannual endoscopies.

How Is the Trial Designed?

Group I: eRapa Experimental Treatment 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

Placebo once a day (QD) every other week

Who Is Running the Clinical Trial?

Rapamycin Holdings Inc.

Lead Sponsor

Trials

1

Recruited

30+

Biodexa Pharmaceuticals

Industry Sponsor

Trials

2

Recruited

30+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06950385

Study Details | NCT06950385 | Phase 3 Trial of eRapa in ... 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 ...

2. biodexapharma.com biodexapharma.com/biodexa-receives-us-fda-fast-track-designation-for-erapa-in-familial-adenomatous-polyposis/

Biodexa Receives US FDA Fast Track Designation for ... Patients in cohort 2 experienced an 89% non-progression rate and 29% median reduction in polyp burden at 12 months compared with baseline. The ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12127233/

Familial adenomatous polyposis: non-surgical management of ... Twelve month Phase 2 Clinical Trial Results of eRapa™ in Familial Adenomatous Polyposis (FAP) to be Presented at Prestigious Biennial ...

4. ddw.digitellinc.com ddw.digitellinc.com/p/s/phase-iia-trial-of-encapsulated-rapamycin-erapa-in-patients-with-familial-adenomatous-polyposis-to-reduce-intestinal-polyp-burden-6-month-interim-results-7265

PHASE IIA TRIAL OF ENCAPSULATED RAPAMYCIN (ERAPA ... Polyp outcomes were classified as progressive disease (PD: >20% increase), stable disease (SD: ± 20% change) or partial response (PR: >20% reduction in polyp ...

5. mayo.edu mayo.edu/research/clinical-trials/diseases-conditions/familial-adenomatous-polyposis

Familial Adenomatous Polyposis Clinical Trials The purposes of this study are to analyze the frequency and severity of adverse events associated with low-dose encapsulated rapamycin in Familial Adenomatous ...

6. facingourrisk.org facingourrisk.org/hereditary-cancer-genes-and-risk/APC-risks-prevention-treatment

APC Gene Mutations: Risk, Management and Treatment The purpose of this study is to find out if a medication called eRapa can help slow down the formation of polyps in people with Familial ...

7. gastroenterologyadvisor.com gastroenterologyadvisor.com/news/encapsulated-rapamycin-fast-tracked-for-familial-adenomatous-polyposis/

Encapsulated Rapamycin Fast Tracked for Familial ... Findings showed 75% (21/28) of patients treated with eRapa were considered non-progressors at 12 months, with a median decrease in overall polyp ...