Actively Recruiting
A Phase II Pilot Study of Danazol for Treating Cytopenias in Patients With Cirrhosis
Led by University of Southern California · Updated on 2025-07-11
10
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effects of danazol in treating blood cell deficiencies called cytopenias in people with compensated Child-Pugh Class A or B liver cirrhosis. This phase II pilot study will include patients with or without specific telomere mutations or shortened telomeres. The study aims to enroll 10 patients and explore how danazol might help improve blood cell counts and overall liver health in this group. Participants will take danazol pills orally at a dose of 600 mg daily for 24 months. The study has a single treatment group where all patients receive danazol. The treatment period lasts two years, during which researchers will carefully monitor patients for any side effects or adverse events related to the medication. Throughout the study, participants will undergo regular evaluations including blood tests to track blood cell counts and telomere length, assessments of liver fibrosis and liver function, and monitoring for clinical events like liver decompensation or the need for transplant. Researchers will measure both hematologic response and the occurrence of significant adverse events over two years. The total participant involvement spans the full 24 months of treatment and monitoring.
CONDITIONS
Brief Title
Danazol for Treatment of Cytopenias in Patients With Cirrhosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older and able to provide informed consent
- ECOG performance status 0-2
- Compensated Child-Pugh class A cirrhosis (any cause except chronic hepatitis B) with defined leukopenia and thrombocytopenia measured twice at least 3 months apart within 6 months of enrollment
- Compensated Child-Pugh class B cirrhosis (any cause except chronic hepatitis B) with defined leukopenia or thrombocytopenia measured twice at least 3 months apart within 6 months of enrollment
- Presence of a genetic variant in specified telomere-related genes or shortened telomere length at or below the 5th percentile
- Women of childbearing potential must agree to use highly effective contraception or remain abstinent during treatment
- Negative pregnancy test within 72 hours prior to treatment start
You will not qualify if you...
- Cirrhosis due to chronic hepatitis B or any history of hepatitis B
- Known telomere mutations that confer gain of function
- Known HIV infection
- History of hormone-sensitive cancers or high familial cancer risk
- Currently receiving cancer therapy
- Liver decompensation event in the last 6 months
- Active thrombosis or unprovoked thromboembolic disease (unless adequately treated)
- Pregnant or planning pregnancy
- Breastfeeding females
- Contraindications to danazol use
- Uncontrolled serious medical conditions making danazol unsafe
- Elevated liver enzymes above specified limits
- Recent alcohol or drug abuse within one year
- Use of hormone stimulants or blockers
- Use of other bone marrow stimulating agents except short-term growth factors per standard care
- Use of systemic immunosuppressive medications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 months
Participants receive Danazol 600mg orally daily to treat cytopenias associated with cirrhosis.
Regular visits for monitoring throughout treatment
Trial Site Locations
Total: 1 location
1
Keck Hosital of USC
Los Angeles, California, United States, 90033
Actively Recruiting
Research Team
I
Ibrahim Syed, MBBS
C
Caitlin O'Neill, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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