Nonalcoholic SteatohepatitisPhase 2NCT00586911
Betaine in Patients With Nonalcoholic Steatohepatitis
A Phase 2 study testing Cystadane in people with Nonalcoholic Steatohepatitis, run by Mayo Clinic at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Nonalcoholic Steatohepatitis
- What is being tested?
- Cystadane
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2003to Jun 2006
- Phase
- Phase 2
- 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
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Florida at Gainesville
Gainesville, Florida, 32610
Mayo Clinic
Rochester, Minnesota, 55905
About this study
Official title: A Randomized, Placebo-Controlled Trial of Betaine in Patients With Nonalcoholic Steatohepatitis (NASH)
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
- Being tested
- Cystadane, Identical Placebo
- Keywords
- Nonalcoholic Steatohepatitis, NASH, Betaine
- Medical subject headings
- Non-alcoholic Fatty Liver Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CystadaneDrug
Betaine 20 mg a day or identical placebo for 1 year.
Identical PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Cystadane | Active comparator | Not described Receives: Drug: Cystadane |
| Identical Placebo | Placebo comparator | Not described Receives: Drug: Identical Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
changes in degree of steatosis, necroinflammatory activity and fibrosis in liver biopsy.
Measured: 1 year
Secondary outcomes (1)
Secondary end-points will be changes in liver test results and health related quality of life.
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 posted7 Jan 2008 (estimated)
- Study startJan 2003
- Primary completionJun 2006
- Study completionJun 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nonalcoholic SteatohepatitisStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- University of Florida, Orphan Medical
- Investigators
- Keith D Lindor, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00586911
- Sponsor's protocol ID
- 458-00
- Status last verified
- Apr 2015
- First submitted
- 21 Dec 2007
- Last updated
- 8 Apr 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-Controlled Trial of Betaine in Patients With Nonalcoholic Steatohepatitis (NASH)
- ClinicalTrials.gov ID
- NCT00586911
- Acronym / sponsor ID
- 458-00
- Conditions
- Nonalcoholic Steatohepatitis
- Keywords
- Nonalcoholic Steatohepatitis, NASH, Betaine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- University of Florida, Orphan Medical
- Study start
- Jan 2003
- Primary completion
- Jun 2006 (actual)
- Study completion
- Jun 2006 (actual)
- First posted
- 7 Jan 2008 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cystadane | Active comparator | Drug: Cystadane |
| Identical Placebo | Placebo comparator | Drug: Identical Placebo |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
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 |
|---|---|---|
| CystadaneBetaine 20 mg a day or identical placebo for 1 year. | Drug | Cystadane |
| Identical PlaceboPlacebo | Drug | Identical Placebo |
Primary outcome
changes in degree of steatosis, necroinflammatory activity and fibrosis in liver biopsy.
Time frame: 1 year
1 secondary outcome
Secondary end-points will be changes in liver test results and health related quality of life.
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 8 Apr 2015.
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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, MD principal investigator
Mayo Clinic
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cystadane in people with Nonalcoholic Steatohepatitis, run by Mayo Clinic at 2 sites 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?
The sponsor lists 12 inclusion and 10 exclusion criteria. 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 8 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
changes in degree of steatosis, necroinflammatory activity and fibrosis in liver biopsy. (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Apr 2015.