Hormone drug may slow Aging-Related diseases in rare condition
NCT ID NCT03312400
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested whether low doses of the hormone drug danazol can slow the shortening of telomeres—the protective caps on DNA—in people with telomere diseases. These conditions can cause bone marrow failure, lung scarring, and liver damage. Eighteen participants took danazol for one year, and researchers measured changes in telomere length, lung function, and liver health. The goal was to see if the drug is safe and can reduce the rate of telomere loss.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Danazol (a synthetic hormone)
- What this could lead to
- If successful, this could provide a treatment option to slow disease progression in people with telomere-related conditions.
- What could go wrong
- This is a small, early-phase trial with only 18 participants, so results may not apply broadly. Danazol can cause hormonal side effects, and the long-term benefits are uncertain.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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18 people
The number who actually took part.
- Started
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Feb 2018
- Finished
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Oct 2025
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 to 99 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: 1. Age-adjusted telomere length less than or equal to the first percentile by flow-FISH method. In patients with a known pathogenic or likely pathogenic mutation in a telomere maintenance gene, age adjusted telomere length less than or equal to the 10th percentile is sufficient. 2. A mutation in telomere maintenance genes (TERT, TERC, DKC1, TINF2, NHP2, NOP10, WRAP53, TERF2, PARN, RTEL1, ACD, CTC1, USB1) as tested in a CLIA (or international equivalent) certified laboratory 3. Age greater than or equal to 3 years 4. Weight greater than or equal to 12 Kg AND 5. At least one of the following criteria: 1. Anemia with a hemoglobin less than or equal to 10 g/dL without red blood cell transfusion 2. Thrombocytopenia with a platelet count less than or equal to 50,000/microliter without transfusion 3. Neutropenia with an absolute neutrophil count less than or equal to 1,000/ microliter OR Pulmonary fibrosis diagnosed by either a lung biopsy or computed tomography scan of the chest according to guidelines from the American Thoracic Society and European Respiratory Society. OR 6. Hepatic fibrosis diagnosed by Transient Elastography by Fibroscan value greater than 10 kpa or US evidence of cirrhotic liver or splenomegaly, or transjugular liver biopsy demonstrating fibrosis. EXCLUSION CRITERIA: 1. Patients on androgen hormones to include testosterone or high dose estrogen (estradiol 0.5 mg/day or greater) for the12 months prior to enrollment 2. Patients with active thrombosis or thromboembolic disease and history of such events, undiagnosed abnormal genital bleeding, porphyria, androgendependent tumor, or prostatic hypertrophy 3. Patients with pulmonary fibrosis who are receiving anti-fibrotic drug treatment, such as pirfenidone or nintedanib unless stable on anti-fibrotic drug for at least 6 months prior to starting on danazol as demonstrated by PFTs. 4. Patients with active hepatitis B or C 5. Patients who have received a bone marrow transplant 6. Patient with other hereditary bone marrow failure syndromes such as Fanconi anemia or Diamond Blackfan anemia 7. Patients with infections not adequately responding to appropriate therapy 8. Current pregnancy, or unwillingness to take oral contraceptives or use the barrier methods of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of the study 9. Lactating women, due to the potentially harmful effects on the nursing child 10. Patients with cancer who are actively receiving systemic chemotherapeutic treatment or who take drugs with hematological effects 11. Patients with decompensated liver disease to include persistent ascites, encephalopathy, variceal hemorrhage, or MELD score of 10 or greater 12. Inability to understand the investigational nature of the study or to give informed consent or without a legally authorized representative or surrogate that can provide informed consent 13. Inability to swallow a capsule
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.