Study Comparing Tetrathiomolybdate vs Standard Treatment in Primary Biliary Cirrhosis

NCT ID: NCT00805805

Last Updated: 2017-05-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 29 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-04-30
• Study Completion Date: 2008-12-31

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

Conditions

Primary Biliary Cirrhosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

1

Tetrathiomolybdate with ursodiol

Group Type: EXPERIMENTAL

Tetrathiomolybdate

Intervention Type: DRUG

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).

2

Placebo with ursodiol

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

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

Interventions

Tetrathiomolybdate

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: DRUG

Placebo

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

Intervention Type: OTHER

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

FDA Office of Orphan Products Development

FED

Sponsor Role: collaborator

George Brewer

OTHER

Sponsor Role: lead

Responsible Party

George Brewer

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

University of Michigan

Ann Arbor, Michigan, United States

Countries

United States

Other Identifiers

PBC

Identifier Type: -

Identifier Source: org_study_id