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NCT03312400 · ClinicalTrials.gov registry record · Phase 2
Low-Dose Danazol for the Treatment of Telomere Related Diseases
A Phase 2 study of Telomere Disease, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Recruiting
- Registry status
- Phase 2
- Development phase
- 40
- Enrollment target
- 1
- Study location
NCT03312400: Recruiting Phase 2 study of Telomere Disease, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT03312400 is a Phase 2 study of Telomere Disease that is actively recruiting participants, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 40 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (70% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03312400, a Phase 2 study of Telomere Disease, is actively recruiting participants, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 40 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: 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. Objective: To study the safety and effect of low dose danazol. Eligibility: 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. Design: Participants will be screened in another protocol. Participants will have: * Medical history * Physical exam * Blood tests * Lung exam. They will breathe into an instrument that records the amount and rate of air breathed in and out over a period of time. 6-minute walking test. * Abdominal ultrasound and liver scan. These tests use sound waves to measure the fibrosis in the liver. Some participants will have: * Pregnancy test * Small sample of the liver removed * Bone marrow biopsy. The bone will be numbed and a small needle will take a sample of the marrow. All participants will have hormone levels checked. All child participants will see a pediatric endocrinologist. Children may need to have a hand x-ray. We will monitor patients for 6 months before starting danazol. Participants will take danazol by mouth twice a day for 1 year. Participants must return to the clinic at 6 months and 12 months while on danazol and 6 months after stopping it. They will have blood and urine tests, a lung exam, abdominal ultrasound, and liver scan.
Primary Outcome
Reduction of telomere attrition rate (decreased rate of telomere attrition by 50%, as compared to the baseline rate)
Conditions Studied
Interventions
- DRUG Danazol
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2018-02-08 |
| Est. Completion | 2027-10-29 |
| Phase | Phase 2 |
What NCT03312400 shows while recruiting
NCT03312400 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 40 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (70% lower).
The record links to 1 condition, with Telomere Disease appearing as the primary indexed condition, and to 1 intervention - of which Danazol is the first listed.
NCT03312400 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT03312400 about?
NCT03312400 is a clinical study titled "Low-Dose Danazol for the Treatment of Telomere Related Diseases". Background: 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 d...
What is the current status of trial NCT03312400?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 40 participants. The study started on 2018-02-08. Estimated completion is 2027-10-29.
What conditions does trial NCT03312400 study?
This clinical trial studies the following conditions: Telomere Disease.
What interventions are being tested in trial NCT03312400?
The interventions under investigation include: Danazol (DRUG).
Who is sponsoring clinical trial NCT03312400?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03312400 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03312400's enrollment target sits among peer trials
40 1362nd of 2000 higher than 543 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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