Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
ConditionFamilial Adenomatous Polyposis (FAP)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorRapamycin Holdings Inc.
About this trial
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.
Eligibility criteria
Qualifiers
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- eRapa (encapsulated rapamycin)
- Placebo
Treatment groups
168 Participants
are divided into 2 treatment groupsSponsors and collaborators
Rapamycin Holdings Inc.
Lead sponsor
Biodexa Pharmaceuticals
Collaborator
Source ClinicalTrials.gov