Low-Dose Danazol for the Treatment of Telomere Related Diseases

Updated on 22 December 2020
liver disease
pulmonary fibrosis
lung disease
hepatic fibrosis


DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers want to see if low doses of the hormone drug danazol can help.



Researchers at the National Institutes of Health (NIH) 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 telomore length. Additional research is needed to learn more about the role 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 6 months, a year, and 6 months after stopping danazol.
  • Your participation in this study will last about 2 years.

Study-related tests, procedures, and medications are provided at no cost. Travel to and from the NIH, within the United States may be reimbursed.

Location: The NIH Clinical Center, America’s Research Hospital is located on the Metro red line (Medical Center stop) in Bethesda, Maryland.

Condition Aplastic Anemia, Aplastic Anemia, Anemia; Non-Small-Cell Lung Cancer, Pulmonary Fibrosis, Pulmonary Fibrosis, Telomeres Hepatic Fibrosis Liver Disease Lung Disease
Clinical Study IdentifierTX217972
Last Modified on22 December 2020


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Inclusion Criteria

People ages 3 and older with a telomere disease who have either very short telomeres and a specific gene change
They must also show signs of aplastic anemia, lung, or liver disease
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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