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NCT01441037 · ClinicalTrials.gov registry record · Phase 1
Danazol for Genetic Bone Marrow and Lung Disorders
A Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 27
- Enrollment target
NCT01441037: Completed Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT01441037 is a Phase 1 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 27 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (55% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01441037, a Phase 1 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 27 participants
- Enrollment target
Study Summary
Background: \- Some people have bone marrow and lung disorders that are caused by genetic problems. These problems often involve damage to the ends of the chromosomes that pass down genes. One of these disorders is aplastic anemia. This is a disorder in which the bone marrow does not make enough blood cells. Currently, doctors use a male hormone-based drug called Danazol to improve bone marrow function and treat aplastic anemia. More information is needed on whether Danazol can help repair the damaged chromosomes that cause aplastic anemia and similar disorders that cause low blood cell counts or lung problems. Objectives: \- To study the safety and effectiveness of Danazol for bone marrow and lung disorders caused by damaged genes. Eligibility: \- Individuals at least 2 years of age who have low blood cell counts or lung fibrosis caused by damaged genes. Design: * Participants will be screened with a physical exam and medical history. Then they will have blood and urine tests, imaging studies, and a lung function test. They will also take a 6-minute walking test and have a bone marrow biopsy. * Participants will receive Danazol to take twice a day for the duration of the study. * Participants will have regular study visits at 6, 12, and 24 months, with blood tests, imaging studies, a lung function test, and a 6-minute walking test. A bone marrow sample will be collected at the 12-month visit. * Participants will remain on the study for up to 2 years. Researchers will follow up with them for 2 years after the end of the study.
Primary Outcome
The primary efficacy end point was a 20% reduction in the annual rate of telomere attrition measured at 24 months. The biologic response at 24 months, was defined as a reduction in the telomere length attrition rate to 96 bp per year or less. The normal rate of telomere loss of approximately 60 bp per year. Telomere length was determined with a semiautomated, Clinical Laboratory Improvement Amendments (CLIA)-approved real-time quantitative PCR (qPCR) assay performed in triplicate and validated f
Interventions
- DRUG Danazol
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 27 participants |
| Start Date | 2011-07-19 |
| Est. Completion | 2016-11-14 |
| Phase | Phase 1 |
What the finished NCT01441037 record still lists
NCT01441037 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 27 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (55% lower).
The record links to 0 conditions, and to 1 intervention - of which Danazol is the first listed.
NCT01441037 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01441037 about?
NCT01441037 is a clinical study titled "Danazol for Genetic Bone Marrow and Lung Disorders". Background: \- Some people have bone marrow and lung disorders that are caused by genetic problems. These problems often involve damage to the ends of the chromosomes that pass down genes. One of these disorders is aplastic anemia. This is a disorder in which the bone marrow does not make enough bl...
What is the current status of trial NCT01441037?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 27 participants. The study started on 2011-07-19. Estimated completion is 2016-11-14.
What interventions are being tested in trial NCT01441037?
The interventions under investigation include: Danazol (DRUG).
Who is sponsoring clinical trial NCT01441037?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01441037's enrollment target sits among peer trials
27 1463rd of 2000 higher than 519 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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