Danazol for Cytopenias in Cirrhosis
This study is looking at whether a medication called Danazol can help patients with cirrhosis (scarring of the liver) who also have cytopenias (low blood cell counts, like white blood cells or platelets). Many people with cirrhosis experience these low blood cell counts, which can lead to problems like infections or bleeding. The study aims to treat 10 patients with Danazol pills for 24 months. Researchers will be watching to see if their blood counts improve and if there are any serious side effects. You may be able to join if you are 18 or older, have compensated Child-Pugh class A cirrhosis, and have certain low blood cell counts.
- Study design
- This is a pilot study, meaning it's a small study to see if Danazol is safe and effective. It plans to enroll 10 participants.
- What's involved
- You would take Danazol pills daily for 24 months. The study will measure your blood counts and check for side effects.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will track your hematologic response and adverse events for up to 2 years.
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NCT04873102
Danazol for Treatment of Cytopenias in Patients With Cirrhosis
Recruiting
PHASE2Ages 18+InterventionalTreatmentUniversity of Southern CaliforniaInvestigator-initiated
~10 participants
Updated 2025-07-11 on ClinicalTrials.gov
What's tested:Danazol Pill
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Hematologic Response
Measured over 2 years
+1 more outcome measured
Conditions
Where it's being run
1 sites across 1 statesCalifornia1
Study leadership
- Casey O'Connell, MD · PRINCIPAL_INVESTIGATOR · Keck Hospital of USC
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age 18 years or older and able to provide informed consent
ECOG 0-2
Compensated Child-Pugh class A of any etiology with the exception of chronic hepatitis B with one or more of the following cytopenias
Compensated Child-Pugh class B cirrhosis of any etiology with the exception of chronic hepatitis B with one or more of the following cytopenias:
Enrolled patients must have one or more of the following:
Presence of a genetic variant (defined as a known mutation, variant likely to be pathogenic or variant of undetermined significance with likely deleterious effect on transcription or translation) in at least one of the following genes: TERT, TERC, RTEL1, DKC, NOP10, NHP2, TINF2, WRAP53
Shortened telomere length in peripheral blood mononuclear cells (defined as age-adjusted telomere length at or below the 5th percentile)
Of note, patient's found to have telomere mutations know to confer a gain of function will be excluded
For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of \< 1% per year during the treatment period
A woman is considered to be of childbearing potential if she is postmenarcheal, has not reached a postmenopausal state (\>= 12 continuous months of amenorrhea with no identified cause other than menopause), and has not undergone surgical sterilization (removal of ovaries and/or uterus)
Examples of contraceptive methods with a failure rate of \< 1% per year include bilateral tubal ligation, male sterilization, proper use of hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices
The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient; periodic abstinence (e.g., calendar, ovulation, symptothermal, or post ovulation methods) and withdrawal are not acceptable methods of contraception
Women of childbearing potential (WOCBP) must have a negative serum test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin \[HCG\]) within 72 hours prior to the start of treatment
Exclusion
Cirrhosis secondary to chronic hepatitis B or any history of hepatitis B
Patients with telomere related mutations know to confer gain of function will be excluded
Patients known to be infected with HIV
History of any hormone sensitive malignancy, including breast cancer, prostate cancer, hepatocellular carcinoma or liver adenoma as well as any patient considered high risk for developing malignancy (i.e. history of familial cancers including a first degree relative)
Patients who are actively receiving anti-cancer therapy
Liver decompensation event within the last 6 months (i.e. variceal bleed, ascites requiring paracentesis, hepatic encephalopathy)
Active thrombosis or history of unprovoked thromboembolic disease, including cardiovascular events. If a patient has received and completed adequate anticoagulation for a provoked thrombosis, they can be included in the study.
Pregnant or planning to become pregnant
Females patients who are breast feeding
Any contraindication to danazol use
Uncontrolled co-morbid condition which would make the administration of danazol unsafe, including decompensated heart failure or known EF less than 40%, unstable angina pectoris, uncontrolled cardiac arrhythmia, decompensated liver failure, renal failure defined as creatinine greater than \>1.6 or psychiatric illness that would limit compliance with study requirements
Alanine aminotransferase and/or aspartate aminotransferase \>3x upper limit of normal
Alkaline phosphatase \>2.5 x upper limit of normal
Total bilirubin or direct bilirubin \>2.5 x upper limit of normal
Patients with known alcohol or drug abuse within the last year
Concomitant use of hormone stimulants or hormone blocking agents.
Concomitant use of other bone marrow stimulating agents that may affect white blood cell and platelet counts (i.e. G-CSF, romiplostim, eltrombopag, corticosteroids). Short term use of growth factors per standard of care in preparation for procedure or for other medical indications is acceptable. Patients taking corticosteroids above 5 mg of prednisone or the equivalent who are on a stable dose for at least 8 weeks prior to enrollment can be included.
Concomitant treatment with systemic immunosuppressive medications (including but not limited to cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor \[anti-TNF\] agents)
What this trial measures
- Hematologic Response2 years
Defined as normalization of WBC to ≥ 4000/µL or doubling of WBC from baseline, AND/OR normalization of platelet count to ≥150,000/µL or doubling of platelet count from baseline, from study entry to three months.
- Occurrence of grade 3+ adverse eventsUp to 2 years
Adverse events will be measured using CTCAE v5 .