Primary Sclerosing CholangitisPhase 2/3NCT00059202
Trial of High-dose Urso in Primary Sclerosing Cholangitis
A Phase 2/3 study testing Ursodeoxycholic Acid in people with Primary Sclerosing Cholangitis, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 7 sites in United States.
Trial summary
This is a multicenter, randomized, controlled trial of high dose ursodiol versus placebo for patients with primary sclerosing cholangitis (PSC). The average duration of follow-up will be approximately five years with important clinical endpoints such as death, eligibility for liver transplantation, changes in… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- Ursodeoxycholic Acid
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 7 sitesUnited States
- Study dates
- Jul 2002to Jun 2008
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
- Chronic cholestatic disease of at least six months' duration.
- Serum alkaline phosphatase at least 1 ½ times the upper limits of normal.
- Retrograde, operative, percutaneous, or magnetic resonance cholangiography demonstrating intrahepatic and/or extrahepatic biliary duct obstruction, beading, or narrowing consistent with PSC within one year of the study entry.
- Liver biopsy in the previous one year which is available for review and compatible with the diagnosis of PSC. Compatible biopsy features include fibrous cholangitis, ductopenia with periportal inflammation and biliary fibrosis.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Mayo Clinic
Jacksonville, Florida
University of Nebraska Medical Center
Omaha, Nebraska
Medical College of Virginia
Richmond, Virginia
Mayo Clinic
Rochester, Minnesota, 55905
Mayo Clinic
Scottsdale, Arizona
University of Washington
Seattle, Washington
St. Louis University
St Louis, Missouri
About this study
Official title: Multicentered Randomized Trial of High-dose Urso in Primary Sclerosing Cholangitis
This is a multicenter, randomized, controlled trial of high dose ursodiol versus placebo for patients with primary sclerosing cholangitis (PSC). The average duration of follow-up will be approximately five years with important clinical endpoints such as death, eligibility for liver transplantation, changes in histology and cholangiogram as well as liver biochemistries and quality of life data collected.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Ursodeoxycholic Acid, Placebo
- Keywords
- Urso, PSC
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ursodeoxycholic AcidDrug
Also called UDCA
Ursodeoxycholic Acid 28-30 mg/kg/day in divided doses
PlaceboDrug
Placebo for Urso
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo tablet that is identical (size, color, etc) to experimental ursodeoxycholic acid tablet. Receives: Drug: Placebo |
| Ursodeoxycholic acid | Experimental | Ursodeoxycholic acid 28-30 mg/kg/day Receives: Drug: Ursodeoxycholic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Failure
Treatment failure was defined as death, transplantation, meeting minimal listing criteria, development of varices, cholangiocarcinoma, or progression to cirrhosis
Measured: 5 years
Secondary outcomes (15)
Number of Deaths
Death at any time up to 5 years
Measured: 5 years
Number of Participants Who Have a Liver Transplantation
Liver transplantation at any time up to 5 years
Measured: 5 years
Number of Participants Meeting Minimal Listing Criteria for Liver Transplantation
Meeting minimal listing criteria for liver transplantation at any time up to 5 years. Minimal listing criteria are defined as a MELD score of 14 or higher. The Model For End-Stage Liver Disease (MELD) is a numeric score with a range of 6 to 40, where a higher score means worse liver disease.
Measured: 5 years
Number of Participants Who Developed Cholangiocarcinoma
Development of cholangiocarcinoma at any time up to 5 years
Measured: 5 years
Number of Participants Who Developed Varices
Development of esophogeal and/or gastric varices
Measured: 5 years
Number of Participants Who Developed Cirrhosis
Development of cirrhosis based on liver biopsy
Measured: 5 years
Alkaline Phosphatase at 12 Months
Alkaline phosphatase divided by the upper limit of normal
Measured: 12 months
Alkaline Phosphatase at 24 Months
Alkaline phosphatase divided by the upper limit of normal
Measured: 24 months
Alkaline Phosphatase at 36 Months
Alkaline phosphatase divided by the upper limit of normal
Measured: 36 months
Aspartate Aminotransferase at 12 Months
Aspartate aminotransferase at divided by the upper limit of normal
Measured: 12 months
Aspartate Aminotransferase at 24 Months
Aspartate aminotransferase at divided by the upper limit of normal
Measured: 24 months
Aspartate Aminotransferase at 36 Months
Aspartate aminotransferase at divided by the upper limit of normal
Measured: 36 months
Bilirubin at 12 Months
Bilirubin divided by the upper limit of normal
Measured: 12 months
Bilirubin at 24 Months
Bilirubin divided by the upper limit of normal
Measured: 24 months
Bilirubin at 36 Months
Bilirubin divided by the upper limit of normal
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2003 (estimated)
- Study startJul 2002
- Primary completionJun 2008
- Study completionJun 2008
Why it stopped: DSMB reviewed interim analysis and terminated the study due to futility.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
- More trials run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00059202
- Sponsor's protocol ID
- URSO (completed)
- Other IDs
- R01DK056924
- Status last verified
- Apr 2021
- First submitted
- 21 Apr 2003
- Last updated
- 3 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Lindor KD, Kowdley KV, Luketic VA, Harrison ME, McCashland T, Befeler AS, Harnois D, Jorgensen R, Petz J, Keach J, Mooney J, Sargeant C, Braaten J, Bernard T, King D, Miceli E, Schmoll J, Hoskin T, Thapa P, Enders F. High-dose ursodeoxycholic acid for the treatment of primary sclerosing cholangitis. Hepatology. 2009 Sep;50(3):808-14. doi: 10.1002/hep.23082.
PubMed 19585548
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentered Randomized Trial of High-dose Urso in Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT00059202
- Acronym / sponsor ID
- URSO (completed)
- Conditions
- Primary Sclerosing Cholangitis
- Keywords
- Urso, PSC
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: DSMB reviewed interim analysis and terminated the study due to futility.
- Enrolment
- 150 participants (actual)
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Study start
- Jul 2002
- Primary completion
- Jun 2008 (actual)
- Study completion
- Jun 2008 (actual)
- First posted
- 22 Apr 2003 (estimated)
- Results
- Posted 8 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, randomized, controlled trial of high dose ursodiol versus placebo for patients with primary sclerosing cholangitis (PSC). The average duration of follow-up will be approximately five years with important clinical endpoints such as death, eligibility for liver transplantation, changes in histology and cholangiogram as well as liver biochemistries and quality of life data collected.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo tablet that is identical (size, color, etc) to experimental ursodeoxycholic acid tablet. |
| Ursodeoxycholic acid | Experimental | Drug: Ursodeoxycholic AcidUrsodeoxycholic acid 28-30 mg/kg/day |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ursodeoxycholic AcidAlso: UDCAUrsodeoxycholic Acid 28-30 mg/kg/day in divided doses | Drug | Ursodeoxycholic acid |
| PlaceboPlacebo for Urso | Drug | Placebo |
Primary outcome
Number of Participants With Treatment Failure
Time frame: 5 years
Treatment failure was defined as death, transplantation, meeting minimal listing criteria, development of varices, cholangiocarcinoma, or progression to cirrhosis
15 secondary outcomes
Number of Deaths
Time frame: 5 years
Number of Participants Who Have a Liver Transplantation
Time frame: 5 years
Number of Participants Meeting Minimal Listing Criteria for Liver Transplantation
Time frame: 5 years
Number of Participants Who Developed Cholangiocarcinoma
Time frame: 5 years
Number of Participants Who Developed Varices
Time frame: 5 years
Number of Participants Who Developed Cirrhosis
Time frame: 5 years
Alkaline Phosphatase at 12 Months
Time frame: 12 months
Alkaline Phosphatase at 24 Months
Time frame: 24 months
Alkaline Phosphatase at 36 Months
Time frame: 36 months
Aspartate Aminotransferase at 12 Months
Time frame: 12 months
Aspartate Aminotransferase at 24 Months
Time frame: 24 months
Aspartate Aminotransferase at 36 Months
Time frame: 36 months
Bilirubin at 12 Months
Time frame: 12 months
Bilirubin at 24 Months
Time frame: 24 months
Bilirubin at 36 Months
Time frame: 36 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 19585548Lindor KD, Kowdley KV, Luketic VA, Harrison ME, McCashland T, Befeler AS, Harnois D, Jorgensen R, Petz J, Keach J, Mooney J, Sargeant C, Braaten J, Bernard T, King D, Miceli E, Schmoll J, Hoskin T, Thapa P, Enders F. High-dose ursodeoxycholic acid for the treatment of primary sclerosing cholangitis. Hepatology. 2009 Sep;50(3):808-14. doi: 10.1002/hep.23082.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Ursodeoxycholic Acid in people with Primary Sclerosing Cholangitis, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: stopped early, as of the sponsor's last update on 3 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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?
Yes. Results are posted in the registry (first posted 8 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Treatment Failure (time frame: 5 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 NCT00059202, which also lists 1 linked publication.
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 3 May 2021.