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Danazol for Genetic Bone Marrow and Lung Disorders

NCT01441037 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2018-08-15

Study results available
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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.

Conditions

  • Aplastic Anemia

Interventions

DRUG

Danazol

Danazol, 800 mg daily by mouth for 2 years

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

  • Neal S Young, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-07-19
Primary Completion
2016-11-14
Completion
2016-11-14
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01441037 on ClinicalTrials.gov