Low-Dose Danazol for the Treatment of Telomere-Related Diseases
Researchers at the National Institutes of Health are studying if the medication danazol can be used to treat people with short telomere disease who also have bone marrow failure, liver, or lung disease.
In recent studies, danazol, at high doses, showed a positive influence on telomere length. Additional research is needed to learn more about the role of different doses of danazol on telomere disease as well as its effects on secondary diseases.
Eligible participants:
- Are 3 and older (minors must have parent/guardian consent to participate);
- Are diagnosed with a short telomere disease;
- Also have abnormalities affecting the bone marrow such as aplastic anemia or low blood counts OR lung disease OR liver disease
Study procedures:
- Before starting danazol, you will have tests and evaluations including measurements of your telomere length.
- Depending on your secondary disease you may also have a bone marrow biopsy, lung function tests, or evaluation of your liver.
- You will then begin taking danazol either 100 mg twice a day, or 200 mg twice a day for up to a year.
- While taking danazol you will have outpatient visits to the NIH Clinical Center after six months, a year, and six months after stopping danazol.
- Your participation in this study will last about two years.
Study-related tests, procedures, and medications are at no-cost. Travel within the United States may be reimbursed by the study team.
Location: The NIH Clinical Center, America's research hospital, is conveniently located on the Metro Red Line (Medical Center stop) in Bethesda, MD.
For more information:
NIH Clinical Center Office of Patient Recruitment
1-800-411-1222
TTY users dial 7-1-1
Se habla español
Or go online:
https://go.usa.gov/xQudv
Refer to study #18-H-0004
Department of Health and Human Services
National Institutes of Health Clinical Center
National Heart, Lung, and Blood Institute