Condition category
Digestive System
Date applied
03/07/2008
Date assigned
13/08/2008
Last edited
22/03/2016
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Erin Castelloe

ORCID ID

Contact details

Clinical Consultant - Pharmacovigilance
4370 La Jolla Village Drive
Suite 1050
San Diego
92122
United States of America
+1 (0)858 354 6441
ecastelloe@interceptpharma.com

Additional identifiers

EudraCT number

ClinicalTrials.gov number

NCT00550862

Protocol/serial number

747-202

Study information

Scientific title

A study of INT-747 (6-ethyl chenodeoxycholic acid [6-ECDCA]) in combination with ursodeoxycholic acid (UDCA [URSO®]) in patients with primary biliary cirrhosis (PBC)

Acronym

Study hypothesis

The primary hypothesis is that INT-747 will cause a reduction in alkaline phosphatase (AP) levels in primary biliary cirrhosis (PBC) patients, over a 12 week treatment period, as compared to placebo.

Ethics approval

Ethics approval received from:
1. USA: Institutional Review Board, Beth Israel Medical Centre on the 2nd October 2007 (ref: 133-07)
2. Canada: University of Toronto, University Health Network Research Ethics Board on the 10th June 2008 (ref: 07-0624-A)

Ethics approval received from (as of 09/12/2009):
3. Austria: Ethikkommission der Medizinischen Universität Graz on the 1st October 2008 (ref: 19-316 ex 07/09)
4. France: CPP Ile de France VI on the 27th October 2008 (ref: 85-08)
5. Germany: Ethik-Kommission der Medizinischen Hochschule Hannover on the 17th December 2008 (ref: 5162M)
6. Spain: Comitè Ètic Investigació Clínica on the 19th September 2008 (ref: 747-202)

Ethics approval pending from:
7. UK: Multicentre Research Ethics Committee (MREC)
8. The Netherlands
9. Italy

All other centres within recruiting countries will seek ethics approval before recruiting participants.

Study design

Treatment, randomised, double blind (subject, investigator), placebo controlled, parallel assignment, safety/efficacy study

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the sponsor details below to request a patient information sheet

Condition

Primary biliary cirrhosis

Intervention

1. Experimental treatment: INT-747 10 mg orally po once daily (QD)
2. Experimental treatment: INT-747 25 mg po QD
3. Experimental treatment INT-747 50 mg po QD
4. Matched placebo comparator: placebo po QD

Screening can last up to 4 weeks. Treatment is 12 weeks. Follow up after treatment is 2 weeks. Ursodeoxycholic acid (UDCA) treatment is prescribed by each patient's physician; the UDCA dose and timing of its administration each day is determined by each patient's physician (not by the protocol).

Intervention type

Drug

Phase

Not Applicable

Drug names

INT-747 (6-ethyl chenodeoxycholic acid [6-ECDCA]), ursodeoxycholic acid (UDCA [URSO®])

Primary outcome measures

To assess the effects of INT-747 on:
1. Alkaline phosphatase (AP) levels
2. Safety

Time frame: 12 weeks

Secondary outcome measures

1. To assess the effects of INT-747 on:
1.1. Hepatocellular injury and liver function
1.2. Disease-specific and general health symptoms
1.3. Biomarkers of hepatic inflammation and fibrosis
2. Plasma trough concentrations of INT-747 and its major, known metabolites

Time frame: 12 weeks

Overall trial start date

01/11/2007

Overall trial end date

02/12/2010

Reason abandoned

Eligibility

Participant inclusion criteria

1. Male or female age 18 to 70 years
2. Stable dose of ursodeoxycholic acid (UDCA [URSO®]) for at least six months prior to screening
3. Female patients must be post-menopausal, surgically sterile, or prepared to use two methods of contraception with all sexual partners during the study and for 14 days after the end of dosing
4. Male patients must be prepared to use two methods of contraception with all sexual partners during the study and for 14 days after the end of the dosing
5. Proven or likely PBC, as demonstrated by the patient presenting with at least two of the following three diagnostic factors:
5.1. History of increased AP levels for at least 6 months prior to Day 0
5.2. Positive antimitochondrial antibody (AMA) titre (greater than 1:40 titre on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay [ELISA]) or PBC-specific antinuclear antibodies (antinuclear dot and nuclear rim positive)
5.3. Liver biopsy consistent with PBC
6. Screening AP value between 1.5 and 10 x upper limit of normal (ULN)

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

140

Participant exclusion criteria

1. Administration of the following drugs at any time during the three months prior to screening for the study:
1.1. Colchicine
1.2. Methotrexate
1.3. Azathioprine
1.4. Systemic corticosteroids
2. Screening conjugated (direct) bilirubin greater than 2 x ULN
3. Screening alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 5 x ULN
4. Screening serum creatinine greater than 133 µmol/L (1.5 mg/dL)
5. History or presence of hepatic decompensation (e.g., variceal bleeds, encephalopathy, or poorly controlled ascites)
6. History or presence of other concomitant liver diseases including hepatitis due to hepatitis B or C virus (HBV, HCV) infection, primary sclerosing cholangitis (PSC), alcoholic liver disease, definite autoimmune liver disease or biopsy proven nonalcoholic steatohepatitis (NASH)
7. Pregnancy

Recruitment start date

01/11/2007

Recruitment end date

02/12/2010

Locations

Countries of recruitment

Austria, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States of America

Trial participating centre

Intercept Pharmaceuticals
San Diego
92122
United States of America

Sponsor information

Organisation

Intercept Pharmaceuticals (USA)

Sponsor details

4370 La Jolla Village Drive
Suite 1050
San Diego
92122
United States of America
+1 (0)858 652 6800
csciacca@interceptpharma.com

Sponsor type

Industry

Website

http://www.interceptpharma.com

Funders

Funder type

Industry

Funder name

Genextra S.p.A. (Italy)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Funder name

Visium (USA)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Funder name

JAFCO Life Science Investment (Japan)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

See https://clinicaltrials.gov/ct2/show/results/NCT00550862

Publication summary

Publication citations

Additional files

Editorial Notes

22/03/2016: added link to results - basic reporting. On 15/01/2013 the overall trial end date was changed from 01/12/2008 to 02/12/2010.