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New treatment option for Cirrhosis, Liver
Official title Danazol for Treatment of Cytopenias in Patients With Cirrhosis
ClinicalTrials.gov ID: NCT04873102
What this study is testing
What is Danazol Pill?
Danazol Pill is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for cirrhosis, liver.
Also referred to as Danocrine.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a phase II pilot study designed to assess the safety and efficacy of danazol for treatment of cytopenias in patients with CPC A/B cirrhosis. Subjects with or without telomere mutations and/or shortened telomeres will be treated with danazol 600 mg per day by mouth for a duration of 24 months.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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
- Leukopenia defined as white blood cell count \<2000/mm3 or absolute neutrophil count \<1000/mm3 along with thrombocytopenia \<150,000/mm3 measured on...
- Thrombocytopenia defined as platelet count \<50,000/mm3 along with white blood cell count \<4000/mm3 measured on two separate occasions at least 3...
You likely can't join if
- 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...
- 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)
See the full eligibility criteria
- 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
- Leukopenia defined as white blood cell count \<2000/mm3 or absolute neutrophil count \<1000/mm3 along with thrombocytopenia \<150,000/mm3 measured on two separate occasions at least 3 months apart within 6 months of...
- Thrombocytopenia defined as platelet count \<50,000/mm3 along with white blood cell count \<4000/mm3 measured on two separate occasions at least 3 months apart within 6 months of enrollment
- Compensated Child-Pugh class B cirrhosis of any etiology with the exception of chronic hepatitis B with one or more of the following cytopenias: 1\. Leukopenia defined as white blood cell count ≤ 3500/mm3 measured on...
- 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...
- 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...
- 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...
- 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...
- 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
- 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...
- 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...
- 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...
- 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...
- Concomitant treatment with systemic immunosuppressive medications (including but not limited to cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor [anti-TNF] agents)
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
Compensation & support
A stipend or compensation may be offered.
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.