Cholangitis, SclerosingPhase 1NCT00004842
Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis
A Phase 1 study testing budesonide in people with Cholangitis, Sclerosing and Liver Cirrhosis, Biliary, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site in United States.
Trial summary
OBJECTIVES: I. Assess the safety and effectiveness of budesonide in patients with primary sclerosing cholangitis or primary biliary cirrhosis experiencing a suboptimal response to ursodeoxycholic acid. II. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Cholangitis, Sclerosing, Liver Cirrhosis, Biliary
- What is being tested?
- budesonide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- May 1997to Apr 1999
- Phase
- Phase 1
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Pathologically confirmed primary sclerosing cholangitis (PSC) meeting the following criteria:
- Chronic cholestatic disease for at least 6 months
- Liver biopsy within the past 6 months compatible with the diagnosis of PSC Intra and/or extrahepatic biliary duct obstruction, beading, or narrowing
OR
Pathologically confirmed primary biliary cirrhosis (PBC) that is experiencing suboptimal response to ursodeoxycholic acid and meeting the following criteria:
- Chronic cholestatic liver disease for at least 6 months
- Positive antimitochondrial antibody
- No biliary obstruction by ultrasound, CT, or cholangiography
- Prior liver biopsy compatible with diagnosis of PBC
- Received ursodeoxycholic acid for at least 6 months
--Prior/Concurrent Therapy--
Biologic therapy:
- At least 3 months since prior D-penicillamine
- No planned transplantation for at least 1 year
Chemotherapy:
- At least 3 months (6 months for PBC) since prior cyclosporin, colchicine, azathioprine, or methotrexate
- At least 6 months since prior chlorambucil (PBC only)
Endocrine therapy: At least 3 months (6 months for PBC) since prior corticosteroids
Surgery: No prior intraductal stones or operations on the biliary tree except cholecystectomy (PSC only)
Other:
- At least 3 months since prior pentoxifylline, ursodeoxycholic acid, or nicotine (PSC only)
- At least 6 months since prior chenodeoxycholic acid (PBC only)
--Patient Characteristics--
Life expectancy: At least 3 years
Hematopoietic: Not specified
Hepatic:
- Alkaline phosphatase at least 2 times upper limits of normal
- No chronic hepatitis B infection
- No hepatitis C infection
- No autoimmune hepatitis
- Bilirubin no greater than 4 mg/dL (PBC only)
Cardiovascular: No severe cardiopulmonary disease
Other:
- No concurrent advanced malignancy
- At least 3 months since prior inflammatory bowel disease requiring specific treatment except maintenance therapy (PSC only)
- No anticipated need for transplantation within 1 year
- Not pregnant
- No liver disease of other etiology such as:
Chronic alcoholic liver disease
Hemochromatosis
Wilson's disease
Congenital biliary disease
Cholangiocarcinoma
No recurrent ascending cholangitis requiring hospitalization more than 2 times per year (PSC only)
At least 1 year since prior active peptic ulcer
No recurrent variceal bleeds
No spontaneous encephalopathy
No diuretic-resistant ascites
Trial locations and contacts
1 site in 1 country.
About this study
OBJECTIVES: I. Assess the safety and effectiveness of budesonide in patients with primary sclerosing cholangitis or primary biliary cirrhosis experiencing a suboptimal response to ursodeoxycholic acid.
II. Estimate the efficacy of this therapy in these patient groups as a means of evaluating the feasibility of a long-term randomized trial.
Read the detailed description
PROTOCOL OUTLINE:
Patients receive budesonide by mouth 3 times daily for a minimum of 6 months. If liver biochemistries become normal, dosage is reduced to once daily. Treatment discontinues after 1 year.
- Conditions
- Cholangitis, Sclerosing, Liver Cirrhosis, Biliary
- Being tested
- budesonide
- Keywords
- cirrhosis, gastrointestinal disorders, primary biliary cirrhosis, primary sclerosing cholangitis, rare disease
- Medical subject headings
- Cholangitis, Sclerosing, Liver Cirrhosis, Biliary, Fibrosis, Gastrointestinal Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
budesonideDrug
Oral budesonide, 9 mg daily was administered for 1 year
| Study group | Role | What they receive |
|---|---|---|
| Budesonide | Experimental | Oral budesonide Receives: Drug: budesonide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in serum alkaline phosphatase
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startMay 1997
- Primary completionApr 1999
- Study completionApr 1999
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cholangitis, SclerosingStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Mayo Clinic
- Responsible party
- Sponsor
- Investigators
- Keith D. Lindor, Study chair, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004842
- Sponsor's protocol ID
- 199/13922
- Other IDs
- MAYOC-41296, R03DK052344
- Status last verified
- May 2017
- First submitted
- 24 Feb 2000
- Last updated
- 31 May 2017
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Angulo P, Jorgensen RA, Keach JC, Dickson ER, Smith C, Lindor KD. Oral budesonide in the treatment of patients with primary biliary cirrhosis with a suboptimal response to ursodeoxycholic acid. Hepatology. 2000 Feb;31(2):318-23. doi: 10.1002/hep.510310209.
PubMed 10655252
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT00004842
- Acronym / sponsor ID
- 199/13922
- Conditions
- Cholangitis, Sclerosing, Liver Cirrhosis, Biliary
- Keywords
- cirrhosis, gastrointestinal disorders, primary biliary cirrhosis, primary sclerosing cholangitis, rare disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)US National Institutes of Health
- Collaborators
- Mayo Clinic
- Study start
- May 1997 (actual)
- Primary completion
- Apr 1999 (actual)
- Study completion
- Apr 1999 (actual)
- First posted
- 25 Feb 2000 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES: I. Assess the safety and effectiveness of budesonide in patients with primary sclerosing cholangitis or primary biliary cirrhosis experiencing a suboptimal response to ursodeoxycholic acid.
II. Estimate the efficacy of this therapy in these patient groups as a means of evaluating the feasibility of a long-term randomized trial.
Detailed description
PROTOCOL OUTLINE:
Patients receive budesonide by mouth 3 times daily for a minimum of 6 months. If liver biochemistries become normal, dosage is reduced to once daily. Treatment discontinues after 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Budesonide | Experimental | Drug: budesonideOral budesonide |
Who may qualify
- Ages
- 18 to 70 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 |
|---|---|---|
| budesonideOral budesonide, 9 mg daily was administered for 1 year | Drug | Budesonide |
Primary outcome
Change in serum alkaline phosphatase
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2017.
- Results publication: PubMed 10655252Angulo P, Jorgensen RA, Keach JC, Dickson ER, Smith C, Lindor KD. Oral budesonide in the treatment of patients with primary biliary cirrhosis with a suboptimal response to ursodeoxycholic acid. Hepatology. 2000 Feb;31(2):318-23. doi: 10.1002/hep.510310209.
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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.
Keith D. Lindor study chair
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing budesonide in people with Cholangitis, Sclerosing and Liver Cirrhosis, Biliary, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 1 site 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: finished, as of the sponsor's last update on 31 May 2017. 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 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change in serum alkaline phosphatase (time frame: 1 year). 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 NCT00004842, 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 31 May 2017.