Study Comparing Tetrathiomolybdate vs Standard Treatment in Primary Biliary Cirrhosis
Primary Purpose
Primary Biliary Cirrhosis
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Tetrathiomolybdate
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Biliary Cirrhosis focused on measuring Copper, ceruloplasmin, tetrathiomolybdate
Eligibility Criteria
Inclusion Criteria:
- Generally medically healthy
- Age 18 and older
- Documented primary biliary cirrhosis
- Alkaline phosphatase > 137
Exclusion Criteria:
- Severe liver decompensation
- Requirement for renal dialysis
- Pregnancy or nursing
- Meld score > 15 (13-15 will require a physician's clinical judgment)
- Uncontrolled congestive heart failure
- Severe diabetic neuropathy
- Severe pulmonary disease
- Advanced cancer
- Requirement for steroid therapy
- Uncontrolled ascites, variceal hemorrhage or spontaneous bacterial peritonitis
- Pregnant or nursing
Sites / Locations
- University of Michigan
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
1
2
Arm Description
Tetrathiomolybdate with ursodiol
Placebo with ursodiol
Outcomes
Primary Outcome Measures
Improvement in drug treated group vs placebo group in two liver function tests and one serum cytokine measurement
Secondary Outcome Measures
Improvement in drug treated group vs placebo group in serum CRP or interleukin-1-beta-levels
Full Information
NCT ID
NCT00805805
First Posted
December 8, 2008
Last Updated
May 19, 2017
Sponsor
George Brewer
Collaborators
FDA Office of Orphan Products Development
1. Study Identification
Unique Protocol Identification Number
NCT00805805
Brief Title
Study Comparing Tetrathiomolybdate vs Standard Treatment in Primary Biliary Cirrhosis
Official Title
Phase III Trial of Tetrathiomolybdate (TM) in Primary Biliary Cirrhosis
Study Type
Interventional
2. Study Status
Record Verification Date
May 2017
Overall Recruitment Status
Completed
Study Start Date
April 2006 (undefined)
Primary Completion Date
December 2008 (Actual)
Study Completion Date
December 2008 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
George Brewer
Collaborators
FDA Office of Orphan Products Development
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The University of Michigan is conducting a study investigating a potential new treatment aimed at slowing/halting progression of primary biliary cirrhosis. This will be a 2 arm double blind study in which half of the patients will be randomly selected to receive a placebo (capsule with no active ingredient) and half will receive the new treatment drug, tetrathiomolybdate. Neither the patient nor the treating physician will know which arm the patient is in. The length of the study for each patient is 24 months of drug therapy. Lab draws will be necessary weekly for the first 6 weeks of the study, followed by every other week for 3 weeks, and then monthly for the remainder of the 2 year period. In addition, intermittent history and physicals and urine samples will also be necessary. There is no cost to you for any experimental treatment. All patients in both arms will continue on ursodiol and receive standard of care treatment
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Biliary Cirrhosis
Keywords
Copper, ceruloplasmin, tetrathiomolybdate
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
29 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Arm Description
Tetrathiomolybdate with ursodiol
Arm Title
2
Arm Type
Placebo Comparator
Arm Description
Placebo with ursodiol
Intervention Type
Drug
Intervention Name(s)
Tetrathiomolybdate
Intervention Description
120 mg/day, divided as 20 mg three times/day with meals and 60 mg away from food at bedtime, for one week to test gastric tolerance. Increased to 180 mg/day, divided as 40 mg three times/day with meals and 60 mg away from food at bedtime. Serum ceruloplasmin levels measured weekly will be used as a surrogate measure of copper status, with a target of 10-15 mg/dl (normal 20-40). When target Cp levels are reached, usually in 4-8 weeks, a maintenance dose of usually 40-80 mg of TM/day, divided half with a major meal, and half away from food at bedtime will be established (vary from 10 mg to 120 mg/day).
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Arm 2 will basically mirror Arm 1 with the patients receiving 120 mg/day, divided as 20 mg three times/day with meals and 60 mg away from food at bedtime the first week. Increased to 180 mg/day, divided as 40 mg three times/day with meals and 60 mg away from food at bedtime. With the dosage being reduced at about the same frequency as the patients receiving TM
Primary Outcome Measure Information:
Title
Improvement in drug treated group vs placebo group in two liver function tests and one serum cytokine measurement
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Improvement in drug treated group vs placebo group in serum CRP or interleukin-1-beta-levels
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Generally medically healthy
Age 18 and older
Documented primary biliary cirrhosis
Alkaline phosphatase > 137
Exclusion Criteria:
Severe liver decompensation
Requirement for renal dialysis
Pregnancy or nursing
Meld score > 15 (13-15 will require a physician's clinical judgment)
Uncontrolled congestive heart failure
Severe diabetic neuropathy
Severe pulmonary disease
Advanced cancer
Requirement for steroid therapy
Uncontrolled ascites, variceal hemorrhage or spontaneous bacterial peritonitis
Pregnant or nursing
Facility Information:
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
12. IPD Sharing Statement
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Study Comparing Tetrathiomolybdate vs Standard Treatment in Primary Biliary Cirrhosis
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