Study of INT-747 in combination with ursodeoxycholic acid (UDCA [URSO®]) in patients with primary biliary cirrhosis (PBC)
| ISRCTN | ISRCTN67465025 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN67465025 |
| ClinicalTrials.gov number | NCT00550862 |
| Secondary identifying numbers | 747-202 |
- Submission date
- 03/07/2008
- Registration date
- 13/08/2008
- Last edited
- 27/04/2018
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Digestive System
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Erin Castelloe
Scientific
Scientific
Clinical Consultant - Pharmacovigilance
4370 La Jolla Village Drive
Suite 1050
San Diego
92122
United States of America
| Phone | +1 (0)858 354 6441 |
|---|---|
| ecastelloe@interceptpharma.com |
Study information
| 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 |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the sponsor details to request a patient information sheet |
| 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) |
| Study objectives | 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(s) | 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. |
| Health condition(s) or problem(s) studied | 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 |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | INT-747 (6-ethyl chenodeoxycholic acid [6-ECDCA]), ursodeoxycholic acid (UDCA [URSO®]) |
| Primary outcome measure | 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 study start date | 01/11/2007 |
| Completion date | 02/12/2010 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 140 |
| Key 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) |
| Key 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 |
| Date of first enrolment | 01/11/2007 |
| Date of final enrolment | 02/12/2010 |
Locations
Countries of recruitment
- Austria
- Canada
- France
- Germany
- Italy
- Netherlands
- Spain
- United Kingdom
- United States of America
Study participating centre
Intercept Pharmaceuticals
San Diego
92122
United States of America
92122
United States of America
Sponsor information
Intercept Pharmaceuticals (USA)
Industry
Industry
4370 La Jolla Village Drive
Suite 1050
San Diego
92122
United States of America
| Phone | +1 (0)858 652 6800 |
|---|---|
| csciacca@interceptpharma.com | |
| Website | http://www.interceptpharma.com |
"ROR" |
https://ror.org/01sx6jc36 |
Funders
Funder type
Industry
Genextra S.p.A. (Italy)
No information available
Visium (USA)
No information available
JAFCO Life Science Investment (Japan)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Basic results | No | No | |||
| Results article | results | 01/04/2015 | Yes | No |
Editorial Notes
27/04/2018: Publication reference added.
22/03/2016: Added link to results - basic reporting.
15/01/2013: The overall trial end date was changed from 01/12/2008 to 02/12/2010.
