Low Dose Danazol for the Treatment of Telomere Related Diseases | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

Low Dose Danazol for the Treatment of Telomere Related Diseases

Trial Sponsor

NIH - National Heart, Lung, and Blood Institute

Contact Info

Ivana Darden

Start Date: March 1, 2018 End Date: April 30, 2026

ClinicalTrials.gov Identifier: NCT03312400

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.


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 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 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.

Bone Marrow Disease(s): 
Phase 2
Age Group: 
3 years and older
Accepts Healthy Volunteers: 
Details on clinicaltrials.gov: 
Inclusion Criteria: 

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.
Exclusion Criteria: 

Personal history of bone marrow transplant.

Find Out More:


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