Betaine in Patients With Nonalcoholic Steatohepatitis

This study has been completed.
Sponsor:
Collaborators:
University of Florida
Orphan Medical
Information provided by:
Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00586911
First received: December 21, 2007
Last updated: NA
Last verified: July 2007
History: No changes posted
  Purpose

To assess the safety and efficacy of betaine in patients with NASH on markers of disease severity such as liver histology, liver biochemistries, and health related quality of life.


Condition Intervention Phase
Nonalcoholic Steatohepatitis
Drug: Cystadane
Drug: Identical Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Placebo-Controlled Trial of Betaine in Patients With Nonalcoholic Steatohepatitis (NASH)

Resource links provided by NLM:


Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • changes in degree of steatosis, necroinflammatory activity and fibrosis in liver biopsy. [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Secondary end-points will be changes in liver test results and health related quality of life. [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]

Enrollment: 26
Study Start Date: January 2003
Study Completion Date: June 2006
Primary Completion Date: June 2006 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Cystadane
    Betaine 20 mg a day or identical placebo for 1 year.
    Drug: Identical Placebo
    Placebo
  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Adult males or females, age 18 - 70 (inclusive) with NASH.
  • Abnormal aminotransferase levels (1.5x normal) on at least two occasions at less three months apart.
  • Histologic evidence of steatohepatitis on liver biopsy performed within six months of entry.
  • Absence of sustained alcohol ingestion (less than 20 g/d in women or 30 g/d in men).
  • Compensated liver disease and blood cell counts within the following limits: Hb > 12 gr/dl, platelets > 120,000/mm3, and WBC > 3,000/mm3
  • TSH (thyroid-stimulating hormone) within normal limits of testing laboratory.
  • Appropriate exclusion of other liver disease such as viral, autoimmune and metabolic/hereditary liver disease.
  • If a history of diabetes, a hemoglobin A1C < 10.0%.
  • Alpha-fetoprotein in normal range (obtained within the previous year), or if greater than normal, the patient requires a negative ultrasound for hepatocellular carcinoma within prior 3 months.
  • Sexually active female patients of childbearing potential must practice adequate contraception during the treatment period and for 6 months after discontinuation of therapy. A pregnancy test obtained at entry prior to the initiation of treatment must by negative. Female patients must not be breast-feeding.
  • Sexually active male patients must practice acceptable methods of contraception during the treatment period and for 6 months after discontinuation of therapy.
  • Written informed consent for participation in this study.

Exclusion Criteria:

  • Treatment with any experimental drug for NASH, betaine, ursodeoxycholic acid (URSO), methionine, rosiglitazone, metformin, pioglitazone, or vitamin E within 3 months of enrollment or at time of pre-entry liver biopsy. (Patients with diabetes and on stable medical management for six months prior to entry and an anticipated stable program throughout the study will be eligible. Medications that may be used include insulin, biguanides, thiazolidnediones, metformin, and sulfonylureas. Patients with hyperlipidemia on a medical program for control of lipids who have had a change in drug treatment in the preceding six months or with anticipated changes in the year of the study will also be eligible.)
  • Any cause for the liver disease based on patient history and biopsy (where applicable) other than NASH
  • Evidence of decompensated liver disease such as history or presence of ascites, bleeding varices, spontaneous encephalopathy.
  • Any known pre-existing medical condition that could interfere with the patient's participation in and completion of the study such as significant cardiovascular dysfunction or chronic obstructive pulmonary disease requiring specific therapy
  • Pregnancy or breastfeeding.
  • Unwillingness of patient and/or partner to use contraception during treatment.
  • Previous malignant disease (other than non-melanoma skin cancer) in the previous two years.
  • Substance abuse, such as alcohol, I.V. drugs and inhaled drugs.
  • Any other conditions that in the opinion of the investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in and completing the protocol.
  • Lactose intolerant patient since placebo preparation contains lactose.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00586911

Locations
United States, Florida
University of Florida at Gainesville
Gainesville, Florida, United States, 32610
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
University of Florida
Orphan Medical
Investigators
Principal Investigator: Keith D Lindor, MD Mayo Clinic
  More Information

No publications provided

Responsible Party: Dr. Keith D. Lindor, MD / PI, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00586911     History of Changes
Other Study ID Numbers: 458-00
Study First Received: December 21, 2007
Last Updated: December 21, 2007
Health Authority: United States: Food and Drug Administration

Keywords provided by Mayo Clinic:
Nonalcoholic Steatohepatitis
NASH
Betaine

Additional relevant MeSH terms:
Fatty Liver
Liver Diseases
Digestive System Diseases
Betaine
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions
Lipotropic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents

ClinicalTrials.gov processed this record on September 16, 2014