What side effects are associated with this type of intervention?

Common treatments for liver cirrhosis, such as antiviral therapies (entecavir, tenofovir), diuretics, beta-blockers, and medications like lactulose, have various side effects. Entecavir can cause lactic acidosis and acute kidney injury, while beta-blockers may lead to renal dysfunction and increased risk of hepatorenal syndrome. Danazol, an anabolic steroid studied for cytopenias in cirrhosis, may cause liver toxicity, masculinization, and thromboembolic events. These side effects highlight the need for careful management and monitoring in cirrhosis patients.

What prior FDA approvals exist?

FDA-approved treatments for liver cirrhosis primarily focus on managing complications and underlying causes rather than directly targeting telomere length. Commonly approved drugs include antiviral agents like entecavir and tenofovir for hepatitis B-related cirrhosis, and direct-acting antivirals (DAAs) for hepatitis C. Danazol, an anabolic steroid, is being studied for its potential to increase telomere length and reduce telomere attrition in cirrhosis patients, but it is not yet FDA-approved for this specific use. Current treatments aim to improve liver function and manage symptoms rather than directly modifying telomere dynamics.

What other types of research exist?

Promising alternatives to Danazol for liver cirrhosis patients include nucleos(t)ide analogs such as lamivudine, adefovir, and entecavir, which have shown benefits in improving prognosis and reducing recurrence in HBV-related HCC. Additionally, Sho-saiko-to, a traditional Chinese herbal mixture, has demonstrated potential in reducing hepatic fibrosis and improving survival in cirrhotic patients. These treatments offer novel approaches to managing liver cirrhosis by targeting viral suppression and liver health.

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Androgen

Danazol for Low Blood Counts in Liver Cirrhosis

Phase 2

Recruiting

Led By Casey O'Connell, MD

Research Sponsored by University of Southern California

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 18 years or older and able to provide informed consent

- Of note, patient's found to have telomere mutations know to confer a gain of function will be excluded

Must not have

Cirrhosis secondary to chronic hepatitis B or any history of hepatitis B

Liver decompensation event within the last 6 months (i.e. variceal bleed, ascites requiring paracentesis, hepatic encephalopathy)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

All Individual Drugs Already Approved

Approved for 5 Other Conditions

No Placebo-Only Group

Summary

This trial is testing danazol, a medication taken by mouth, to see if it can help people with liver disease and low blood cell counts. Danazol may work by changing hormone levels to increase blood cell production. It has been used to treat certain bleeding conditions, showing long-term benefits.

Who is the study for?

Adults over 18 with compensated liver cirrhosis (Child-Pugh class A/B) and low blood counts, who can consent to treatment. They must not be pregnant or breastfeeding, agree to use effective contraception if of childbearing potential, and have no history of certain conditions like heart failure or uncontrolled diseases. Excludes those with hepatitis B, recent liver decompensation events, HIV infection, high bilirubin levels, or on specific medications.

What is being tested?

The trial is testing the safety and effectiveness of a drug called Danazol at a dose of 600 mg daily for two years in patients with liver cirrhosis experiencing low blood cell counts. It aims to enroll 10 patients including those without telomere mutations.

What are the potential side effects?

Danazol may cause muscle cramps, changes in menstrual flow or sexual function, flushing or sweating; it could also affect the liver enzymes and lipid profiles. Rarely it might lead to more serious issues like blood clots or liver problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 or older and can give my consent.

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I do not have telomere mutations that increase their function.

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I can take care of myself and am up and about more than half of the day.

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My blood test shows I have very short telomeres for my age.

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I have a genetic mutation in one of the specified genes linked to my condition.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have cirrhosis due to chronic hepatitis B or have had hepatitis B.

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I have not had a serious liver event in the last 6 months.

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I am not taking any medications that weaken my immune system.

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I am currently using hormone stimulants or blockers.

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I do not have severe heart, liver, kidney problems, or mental health issues that would make taking danazol unsafe.

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I am currently breastfeeding.

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I cannot take danazol due to health reasons.

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I am currently undergoing treatment for cancer.

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I do not have mutations in my genes that cause them to act abnormally.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Hematologic Response

Occurrence of grade 3+ adverse events

Secondary study objectives

Change in blood cell counts

Change in liver fibrosis

Change in liver function - Albumin

+4 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Danazol in Treatment of CytopeniasExperimental Treatment1 Intervention

AGENT: Danazol 600mg, Oral, Daily for 24 months

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for liver cirrhosis work through various mechanisms to manage symptoms and improve patient outcomes. Albumin therapy helps maintain plasma oncotic pressure and reduces complications like ascites. SGLT2 inhibitors, primarily used for diabetes, may reduce fluid retention by promoting glucose and sodium excretion. Antiviral therapies such as entecavir and tenofovir suppress viral replication in hepatitis B, reducing liver inflammation and fibrosis. Danazol, under investigation, may increase telomere length and reduce telomere attrition, potentially improving cell longevity and liver function. These treatments are crucial as they address the underlying causes and complications of cirrhosis, improving quality of life and survival rates for patients.

Treatment for hepatorenal syndrome in people with decompensated liver cirrhosis: a network meta-analysis.Pharmacotherapies that specifically target ammonia for the prevention and treatment of hepatic encephalopathy in adults with cirrhosis.