Familial adenomatous polyposis (FAP)
Conditions
Brief summary
Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo. Disease progression is defined as a FAP-specific, clinically significant event. FAP-specific progression events will be defined as follows: 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)
Detailed description
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), 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, Change from baseline in Spigelman stage score at 6, 12, 18, 24, 30, and 36 months, Change from baseline in EQ-5D5L score at 6, 12, 18, 24, 30, and 36 months, Change from baseline in EORTC QLQ-30 score at 6, 12, 18, 24, 30, and 36 months
Interventions
DRUGWhite opaque capsule filled with a white to off- white powder
DRUGeRapa
Sponsors
Biodexa Limited, Rapamycin Holdings Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Progression-free survival (PFS) in high-risk patients with FAP treated with eRapa versus placebo. Disease progression is defined as a FAP-specific, clinically significant event. FAP-specific progression events will be defined as follows: 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) | — |
Secondary
| Measure | Time frame |
|---|---|
| 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), 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 end | — |
Countries
Denmark, Germany, Netherlands, Spain
Outcome results
None listed