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Adenomatous Polyposis Coli, FamilialPhase 2/3NCT00134758

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

A Phase 2/3 study testing Ursodeoxycholic acid in people with Adenomatous Polyposis Coli, Familial, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Adenomatous Polyposis Coli, Familial
What is being tested?
Ursodeoxycholic acid
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
90 participants
Where
1 siteFrance
Study dates
Oct 2004to Oct 2009
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 9Show fewer
  • Cooperative patient
  • Signed consent
  • Social security insurance

Who cannot join

  • 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;
Show all 11Show fewer
  • non-steroid anti-inflammatory drugs;
  • tamoxifen;
  • cholestyramine.
  • Pregnancy
  • Breast-feeding

Trial locations and contacts

1 site in 1 country.

France1 site

  • Saint-Antoine Hospital

    Paris, 75012

Other trials in France

About this study

Official title: Efficiency of Ursodesoxycholic Acid in the Treatment of Duodenal Adenomas in Familial Adenomatous Polyposis Patients. URSOPAF

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.

Read the detailed 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.

Being tested
Ursodeoxycholic acid, Placebo
Keywords
Treatment of adenomas of the duodenum in FAP patients., Adenoma
Medical subject headings
Adenomatous Polyposis Coli, Adenoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ursodeoxycholic acidDrug

    Also called 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

  • PlaceboDrug

    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
Study groups in NCT00134758
Study groupRoleWhat they receive
1ExperimentalUrsodeoxycholic 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
Receives: Drug: Ursodeoxycholic acid
2Placebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. SPIGELMAN severity score of duodenal lesion after 2 years of follow-up

    Measured: Baseline, 1 and 2 years

Secondary outcomes (3)

  1. Cellular proliferation (Ki 67 and PCNA)

    Measured: At the baseline, 1 and 2 years

  2. Biliary acid profile

    Measured: At the baseline, 1 and 2 years

  3. Compliance to the treatment

    Measured: Every 6 months during 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Aug 2005 (estimated)
  2. Study startOct 2004
  3. Primary completionJun 2009
  4. Study completionOct 2009 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
90 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00134758
Sponsor's protocol ID
P030419
Other IDs
AOM 03041
Status last verified
Jul 2009
First submitted
23 Aug 2005
Last updated
29 Jul 2009
Results posted
No
Data monitoring committee
Yes

Publications

  • Spigelman AD, Williams CB, Talbot IC, Domizio P, Phillips RK. Upper gastrointestinal cancer in patients with familial adenomatous polyposis. Lancet. 1989 Sep 30;2(8666):783-5. doi: 10.1016/s0140-6736(89)90840-4.

    PubMed 2571019

  • Nugent KP, Farmer KC, Spigelman AD, Williams CB, Phillips RK. Randomized controlled trial of the effect of sulindac on duodenal and rectal polyposis and cell proliferation in patients with familial adenomatous polyposis. Br J Surg. 1993 Dec;80(12):1618-9. doi: 10.1002/bjs.1800801244.

    PubMed 8298943

  • Jarvinen HJ, Nyberg M, Peltokallio P. Biliary involvement in familial adenomatosis coli. Dis Colon Rectum. 1983 Aug;26(8):525-8. doi: 10.1007/BF02563746.

    PubMed 6872780

  • Spigelman AD, Owen RW, Hill MJ, Phillips RK. Biliary bile acid profiles in familial adenomatous polyposis. Br J Surg. 1991 Mar;78(3):321-5. doi: 10.1002/bjs.1800780318.

    PubMed 1850640

  • Mower HF, Ray RM, Shoff R, Stemmermann GN, Nomura A, Glober GA, Kamiyama S, Shimada A, Yamakawa H. Fecal bile acids in two Japanese populations with different colon cancer risks. Cancer Res. 1979 Feb;39(2 Pt 1):328-31.

    PubMed 761204

  • Hill MJ, Drasar BS, Williams RE, Meade TW, Cox AG, Simpson JE, Morson BC. Faecal bile-acids and clostridia in patients with cancer of the large bowel. Lancet. 1975 Mar 8;1(7906):535-9. doi: 10.1016/s0140-6736(75)91556-1.

    PubMed 47015

  • Tanida N, Hikasa Y, Shimoyama T, Setchell KD. Comparison of faecal bile acid profiles between patients with adenomatous polyps of the large bowel and healthy subjects in Japan. Gut. 1984 Aug;25(8):824-32. doi: 10.1136/gut.25.8.824.

    PubMed 6745720

  • van der Werf SD, Nagengast FM, van Berge Henegouwen GP, Huijbregts AW, van Tongeren JH. Colonic absorption of secondary bile-acids in patients with adenomatous polyps and in matched controls. Lancet. 1982 Apr 3;1(8275):759-62. doi: 10.1016/s0140-6736(82)91810-4. No abstract available.

    PubMed 6121223

  • Wilpart M, Mainguet P, Maskens A, Roberfroid M. Structure-activity relationship amongst biliary acids showing comutagenic activity towards 1,2-dimethylhydrazine. Carcinogenesis. 1983 Oct;4(10):1239-41. doi: 10.1093/carcin/4.10.1239.

    PubMed 6616753

  • Earnest DL, Holubec H, Wali RK, Jolley CS, Bissonette M, Bhattacharyya AK, Roy H, Khare S, Brasitus TA. Chemoprevention of azoxymethane-induced colonic carcinogenesis by supplemental dietary ursodeoxycholic acid. Cancer Res. 1994 Oct 1;54(19):5071-4.

    PubMed 7923119

  • Serfaty L, De Leusse A, Rosmorduc O, Desaint B, Flejou JF, Chazouilleres O, Poupon RE, Poupon R. Ursodeoxycholic acid therapy and the risk of colorectal adenoma in patients with primary biliary cirrhosis: an observational study. Hepatology. 2003 Jul;38(1):203-9. doi: 10.1053/jhep.2003.50311.

    PubMed 12830003

  • Parc Y, Piquard A, Dozois RR, Parc R, Tiret E. Long-term outcome of familial adenomatous polyposis patients after restorative coloproctectomy. Ann Surg. 2004 Mar;239(3):378-82. doi: 10.1097/01.sla.0000114216.90947.f6.

    PubMed 15075655

  • Parc Y, Desaint B, Flejou JF, Lefevre JH, Serfaty L, Vienne A, Kotti S, Simon T, Tiret E. The effect of ursodesoxycholic acid on duodenal adenomas in familial adenomatous polyposis: a prospective randomized placebo-control trial. Colorectal Dis. 2012 Jul;14(7):854-60. doi: 10.1111/j.1463-1318.2011.02816.x.

    PubMed 21899713

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Ursodeoxycholic acid in people with Adenomatous Polyposis Coli, Familial, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 29 Jul 2009. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

SPIGELMAN severity score of duodenal lesion after 2 years of follow-up (time frame: Baseline, 1 and 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00134758, which also lists 13 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2009.

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