Ursodeoxycholic Acid in the Treatment of Duodenal Adenomas in Familial Adenomatous Polyposis (FAP) Patients | oneFAPvoice

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Ursodeoxycholic Acid in the Treatment of Duodenal Adenomas in Familial Adenomatous Polyposis (FAP) Patients

study id #: NCT00134758

condition: Adenomatous Polyposis Coli, Familial

status: Status unknown

purpose:

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.

intervention:
Drug: Ursodeoxycholic acid
Drug: Placebo

start date: October 2004

estimated completion: June 2009

last updated: July 29, 2009

phase of development: Phase 2, Phase 3

size / enrollment: 90

study design:
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment

study 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.

primary outcomes:

  • SPIGELMAN severity score of duodenal lesion after 2 years of follow-up [ Time Frame:Baseline ] [ Designated as safety issue: No ]

secondary outcomes:

  • Cellular proliferation (Ki 67 and PCNA) [ Time Frame: At the baseline, 1 and 2 years ] [ Designated as safety issue: No ]
  • Biliary acid profile [ Time Frame: At the baseline, 1 and 2 years ] [ Designated as safety issue: No ]
  • Compliance to the treatment [ Time Frame: Every 6 months during 2 years ] [ Designated as safety issue: No ]

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:
1. aspirin;
2. non-steroid anti-inflammatory drugs;
3. tamoxifen;
4. cholestyramine.
- Pregnancy
- Breast-feeding

sponsor: Assistance Publique - Hôpitaux de Paris

investigators: Yann RA Parc, MD, PhD

locations: France