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 range18 Years
SexALL
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
✕. 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.
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.
✕. 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.