Betaine in Patients With Nonalcoholic Steatohepatitis

NCT00586911 | Completed Phase 2

• 1 other identifier: 458-00
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Nonalcoholic Steatohepatitis

Keywords

Nonalcoholic Steatohepatitis; NASH; Betaine

Outcome Measures

Primary Outcomes (1)

• changes in degree of steatosis, necroinflammatory activity and fibrosis in liver biopsy.

  1 year

Secondary Outcomes (1)

• Secondary end-points will be changes in liver test results and health related quality of life.

  1 year

Study Arms (2)

Cystadane

ACTIVE COMPARATOR

Drug: Cystadane

Identical Placebo

PLACEBO COMPARATOR

Drug: Identical Placebo

Interventions

Cystadane DRUG

Betaine 20 mg a day or identical placebo for 1 year.

Cystadane

Identical Placebo DRUG

Placebo

Identical Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Mayo Clinic: lead
• University of Florida: collaborator
• Orphan Medical: collaborator

Study Sites (2)

University of Florida at Gainesville

Gainesville, Florida, 32610, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease

Interventions

Betaine

Condition Hierarchy (Ancestors)

Fatty Liver; Liver Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Trimethyl Ammonium Compounds; Quaternary Ammonium Compounds; Amines; Organic Chemicals; Onium Compounds

Study Officials

• Keith D Lindor, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: December 21, 2007
• First Posted: January 7, 2008
• Study Start: January 1, 2003
• Primary Completion: June 1, 2006
• Study Completion: June 1, 2006
• Last Updated: April 8, 2015

Record last verified: 2015-04

Locations

US (2)