This trial is active, not recruiting.

Condition adenomatous polyposis coli, familial
Treatments ursodeoxycholic acid, placebo
Phase phase 2/phase 3
Sponsor Assistance Publique - Hôpitaux de Paris
Collaborator Axcan Pharma
Start date October 2004
End date June 2009
Trial size 90 participants
Trial identifier NCT00134758, AOM 03041, P030419


Malignant transformation of adenomas of the duodenum is now the leading cause of death in familial adenomatous polyposis (FAP) patients who had a restorative proctocolectomy. Ursodeoxycholic acid (UDCA) modifies the biliary acid profile and could reduce the severity of duodenal adenomas and prevent such transformation.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Ursodeoxycholic acid during 2 years : between 40 and 50 kg : 500 mg/day between 51 and 75 kg : 750 mg/day between 76 and 100 kg : 1000 mg/day
ursodeoxycholic acid Delursan
During 2 years : between 40 and 50 kg : 500 mg/day between 51 and 75 kg : 750 mg/day between 76 and 100 kg : 1000 mg/day
(Placebo Comparator)
placebo Placebo
During 2 years : between 40 and 50 kg : 2 tabs/day between 51 and 75 kg : 3 tabs/day between 76 and 100 kg : 4 tabs/day

Primary Outcomes

SPIGELMAN severity score of duodenal lesion after 2 years of follow-up
time frame: Baseline, 1 and 2 years

Secondary Outcomes

Cellular proliferation (Ki 67 and PCNA)
time frame: At the baseline, 1 and 2 years
Biliary acid profile
time frame: At the baseline, 1 and 2 years
Compliance to the treatment
time frame: Every 6 months during 2 years

Eligibility Criteria

Male or female participants from 18 years up to 65 years old.

Inclusion Criteria: - Male or female patients between 18 and 65 years of age - Weight less than or equal to 100 kg - Restorative proctocolectomy - Activated protein C (APC) mutation identified or more than 100 polyps on the colectomy specimen - SPIGELMAN score of duodenal adenoma greater than or equal to 1 - Efficient contraceptive treatment for pre-menopausal women - Cooperative patient - Signed consent - Social security insurance Exclusion Criteria: - SPIGELMAN score of duodenal adenoma equal to 4 with severe dysplasia - Hepatic disease - Intermesenteric desmoid tumour - Any severe disease - Daily use during the last 3 months of: - aspirin; - non-steroid anti-inflammatory drugs; - tamoxifen; - cholestyramine. - Pregnancy - Breast-feeding

Additional Information

Official title Efficiency of Ursodesoxycholic Acid in the Treatment of Duodenal Adenomas in Familial Adenomatous Polyposis Patients. URSOPAF
Principal investigator Yann RA Parc, M.D., Ph.D.
Description We designed a randomized double blinded study to evaluate the efficiency of UDCA in the treatment of duodenal adenomas. One hundred patients are planned to be included. Fifty will receive UDCA and fifty a placebo. Three duodenoscopies are planned: one before inclusion, one at the end of the first year of follow-up and one after two years of follow-up at the end of the protocol. These duodenoscopies are associated to endoscopies of the ileal reservoir performed at the time of restorative proctocolectomy and are recorded numerically. Severity of the duodenal adenomas are evaluated according to the SPIGELMAN score. Patients are seen every 6 months. Before each endoscopy, blood samples are collected for biliary acid profile analysis. Moreover, during endoscopies, duodenal fluid and ileal fluid are collected for biliary acid profile analysis, also. At the end of the follow-up of the last patients included (nov 2008), biliary acid profile analysis will be performed and statistical analysis of the results will be performed.
Trial information was received from ClinicalTrials.gov and was last updated in July 2009.
Information provided to ClinicalTrials.gov by Assistance Publique - Hôpitaux de Paris.