Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Sur… (NCT04230499) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance
United States30 participantsStarted 2021-01-18
Plain-language summary
Patients with Familial Adenomatous Polyposis (FAP) who are undergoing endoscopic surveillance will be given Encapsulated Rapamycin (eRapa) at one of three escalating doses/schedules for 12 months with the aim of reducing polyp burden.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Sign and date an informed consent form.
. Stated willingness to comply with all study procedures and availability for the duration of the study.
. Male or female, age at least 18 years at the time of consent.
. Phenotypic familial adenomatous polyposis (FAP) with disease involvement of the colorectum by either genetic or clinical diagnosis: Adenomatous polyposis coli (APC) germline mutation with or without family history, or with greater than a cumulative lifetime history of (\>) 100 adenomas in large intestine and a family history of FAP, or FAP phenotype post colectomy for polyposis with a family history of FAP. Minimum number of polyps required for enrollment is 10.
. Abilitiy to safely undergo endoscopy.
. Ability to take oral medication and be willing to adhere to the eRapa regimen.
. For females of reproductive potential: use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 12 weeks after the end of eRapa administration.
. A woman must agree not to breast feed or donate eggs (ova, oocytes) during the study and for a period of 12 weeks after the last administration of study drug.
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Frequency and severity of adverse events associated with low dose eRapa in FAP patients
Timeframe: All adverse events with start dates occurring any time after informed consent is obtained until 7 days (for non-serious adverse events) or 30 days (for serious adverse events) after the last day of study participation will be recorded.
2
Determine the Recommended Phase 2 Dose (RP2D)
Timeframe: After informed consent is obtained up to 30 days after the last day of study participation.
3
Efficacy of eRapa in delaying polyp progression in patients with FAP as measured by change in polyp burden over time.
Timeframe: Time for each patient is baseline to 6 months.
. Risk-reduction surgery (colectomy or partial colectomy) within the 12 months prior to screening.
. Use of non-steroidal anti-inflammatory drugs other than aspirin during the study. The use of 81 milligrams (mg) of aspirin a day or 650 mg of aspirin per week is allowed.
. Treatment with other FAP-directed drug therapy (including NSAID \[Non-steroidal anti-inflammatory drug\] drugs), unless completes a 4 week washout period prior to enrollment.
. Duodenum or colon/ rectum with high grade dysplasia or cancer on biopsy at screening.
. Duodenal or colorectal polyp \> 1 centimeter (cm) not excised at the screening evaluation.
. Pregnancy or breast feeding.
. Unable to provide consent or anticipated inability to attend appropriate follow-up visits.
. Serum creatinine or measured/ calculated creatinine clearance (or glomerular filtration rate \[GFR\]) \> 1.5 x ULN OR \< 30mL/min for participants with creatine levels \> 1.5 x institutional ULN. Bilirubin ≥ 1.5 x ULN unless conjugated bilirubin ≤ ULN; alkaline phosphatase \> 5 x ULN; ALT/AST \> 2 x ULN.