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NCT01001598 · ClinicalTrials.gov registry record · Phase 1
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
A Phase 1 study, sponsored by Boston Children's Hospital.
- Terminated
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT01001598: Clinical Trial Phase 1 study, sponsored by Boston Children's Hospital.
NCT01001598 is a Phase 1 study that was terminated before completion, run by Boston Children's Hospital. The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01001598, a Phase 1 study, was terminated before completion, sponsored by Boston Children's Hospital.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects, indicating a need for a different medication. Danazol is a less potent androgen,and may therefore have fewer masculinizing side effects. Danazol is currently approved by the Food and Drug Administration (FDA) for the treatment of other diseases, but it has never been studied in patients with FA and DC. The main purpose of this study is to see if danazol is a safe treatment for FA and DC. Specifically,we would like to determine: * the best dose of danazol; * how fast hemoglobin (a protein that carries oxygen in the blood) levels rise in FA and DC patients receiving danazol therapy; and * the genetic pattern (known as expression profile) of certain cells in response to danazol, which can predict how well people respond to the medication. Subjects who enroll in the study will be treated with danazol for up to 24 weeks (about 6 months), and will have up to 11 study visits, including followup visits at 38 weeks (9 months) and 52 weeks (one year).
Primary Outcome
All toxicities were collected and adjudicated to definitely-related, possibly-related, or unrelated to the treatment.
Interventions
- DRUG danazol
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2009-11 |
| Est. Completion | 2014-05 |
| Phase | Phase 1 |
Why NCT01001598 stopped before completion
NCT01001598 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which danazol is the first listed.
NCT01001598 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01001598 about?
NCT01001598 is a clinical study titled "Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita". Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects, indicating a need for a different medication. Danazol is a less potent androgen...
What is the current status of trial NCT01001598?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2009-11. Estimated completion is 2014-05.
What interventions are being tested in trial NCT01001598?
The interventions under investigation include: danazol (DRUG).
Who is sponsoring clinical trial NCT01001598?
This trial is sponsored by Boston Children's Hospital, which has 462 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01001598's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 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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