Sirolimus and Familial Adenomatous Polyposis (FAP)

• Status: Completed
• Trial end date: 2018-12-10
• Target enrollment: 0
• Participant gender: All

Summary

The aim of the study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• ≥ 18 years

• A genetically confirmed APC mutation

• Classical FAP phenotype (100-1000 colorectal adenomatous polyps)

• Subtotal colectomy with ileorectal anastomosis (IRA) or total colectomy with ileo-anal pouch anastomosis (IPAA)

• Severe rectal or pouch polyposis, defined as having >25 polyps amenable to complete removal (InSiGHT 2011 Staging System score of 3)

• Fertile patients must use effective contraception during study treatment and until 12 weeks after study treatment

Exclusion Criteria:

• Inability to give informed consent

• Participation in another interventional clinical trial

• Subjects who are pregnant or breast-feeding, proved with a negative pregnancy test if female of child-bearing potential

• Prior pelvic irradiation

• Invasive malignancy in the past 5 years

• Subjects who are HIV positive

• Subjects with severe systemic infections, current or within 2 weeks prior to study start

• Subjects with known severe restrictive or obstructive pulmonary disorders

• Known sucrase insufficiency, isomaltase insufficiency, fructose intolerance, glucose malabsorption, galactose malabsorption, galactose intolerance or Lapp-lactase deficiency

• History of pulmonary embolism or deep venous thrombosis

• Major surgery less than or equal to 2 weeks prior to enrollment or any planned surgery within treatment period

• Active post-operative complication, e.g. infection, delayed wound healing

• History of hypersensitivity to sirolimus or to drugs of similar chemical classes

• Regular NSAID use (defined as more than twice a week for 4 consecutive weeks) within 3 months prior to baseline

• Use of other FAP directed drug therapies (accepted if discontinued 3 months prior to start of the study)

• Subjects requiring systemic anticoagulation

• Co-medication that could interact with sirolimus

• Abnormal laboratory results (assessed within 14 days prior to start of study drug)