Study of INT-747 as monotherapy in patients with primary biliary cirrhosis (PBC)
| ISRCTN | ISRCTN68931598 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN68931598 |
| ClinicalTrials.gov number | NCT00570765 |
| Secondary identifying numbers | 747-201 |
- Submission date
- 03/07/2008
- Registration date
- 13/08/2008
- Last edited
- 16/04/2019
- 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]) monotherapy in patients with primary biliary cirrhosis (PBC) |
| Study objectives | The primary hypothesis is that INT-747 will cause a reduction in alkaline phosphatase 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: Virginia Commonwealth University, McGuire Institutional Review Board, McGuire VA Medical Centre, 02/10/2007, ref: 01379 2. UK: Multicentre Research Ethics Committee (MREC) 3. Austria: Ethikkommission der Medizinischen Universität Graz, 27/10/2008, ref: 20-003 ex 08/09 4. France: CPP Ile de France VI, 09/07/2008, ref: 47-08 5. Germany: Ethik-Kommission der Medizinischen Hochschule Hannover, 22/04/2009, ref: 5164M 6. Spain: Comitè Ètic Investigació Clínica, 30/06/2008, ref: 747-201 Ethics approval pending from: 7. The Netherlands 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 50 mg po QD 3. Matched placebo comparator: placebo po QD Screening period can be up to 4 weeks. Treatment is 12 weeks. Follow up after treatment is 2 weeks. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | INT-747 (6-ethyl chenodeoxycholic acid [6-ECDCA]) |
| 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 | 31/12/2014 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 120 |
| Total final enrolment | 59 |
| Key inclusion criteria | 1. Male or female age 18 to 70 years 2. Female patients must be postmenopausal, 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 3. 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 4. Proven or likely PBC, as demonstrated by the patient presenting with at least two of the following three diagnostic factors: 4.1. History of increased AP levels for at least 6 months prior to Day 0 4.2. 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) 4.3. Liver biopsy consistent with PBC 5. Screening alkaline phosphatase (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. Ursodeoxycholic acid (UDCA, URSO®) 1.2. Colchicine 1.3. Methotrexate 1.4. Azathioprine 1.5. Systemic corticosteroids 2. Screening conjugated (direct) bilirubin greater than 2 x ULN 3. Screening alanine aminotrasferase (ALT) or aspartate aminotrasnferase (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 | 31/12/2014 |
Locations
Countries of recruitment
- Austria
- Canada
- France
- Germany
- 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 | |||
| Abstract results | conference abstract | 01/09/2011 | No | No |
Editorial Notes
16/04/2019: The following changes were made to the trial record:
1. Added clinicaltrials.gov link to basic results (scientific).
2. Publication reference added.
3. The total final enrolment was added.
16/11/2016: No publications found, verifying study status with principal investigator.
15/01/2013: The following changes were made to the record:
1. Canada was added to the countries of recruitment.
2. The overall trial end date was changed from 01/12/2008 to 31/12/2014.
3. Recruitment is closed in the UK, Spain, France, Germany, Canada and Austria.
UK: Closed to recruitment but study ongoing
Spain: Closed to recruitment but study ongoing (until 23/01/2013)
France: Completed/closed
Germany: Completed/closed
Austria: Completed/closed
United States of America: Closed to recruitment but study ongoing
Canada: Closed to Recruitment but study ongoing
