A Phase II Single Center Single Arm Pilot Study Administering Danazol for Treatment of Cytopenias in Patients With Cirrhosis

Who is this study for? Patients with Cytopenia, Liver Cirrhosis
What treatments are being studied? Danazol Pill
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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. The goal will be to treat a total of 10 patients.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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 enrollment

‣ 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 two separate occasions at least 3 months apart within 6 months of enrollment 3. Thrombocytopenia defined as platelet count ≤ 100,000/mm3 measured on two separate occasions at least 3 months apart within 6 months of enrollment

• 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

Locations
United States
California
Keck Hosital of USC
RECRUITING
Los Angeles
Contact Information
Primary
Ibrahim Syed, MBBS
isyed@med.usc.edu
323-865-3000
Backup
Caitlin O'Neill, MD
caitlin.oneill@med.uc.edu
323-865-3000
Time Frame
Start Date: 2024-08-01
Estimated Completion Date: 2027-08-01
Participants
Target number of participants: 10
Treatments
Experimental: Danazol in Treatment of Cytopenias
AGENT: Danazol 600mg, Oral, Daily for 24 months
Sponsors
Leads: University of Southern California

This content was sourced from clinicaltrials.gov

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