Status:
COMPLETED
Danazol for Genetic Bone Marrow and Lung Disorders
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Aplastic Anemia
Eligibility:
All Genders
2+ years
Phase:
PHASE1
PHASE2
Brief 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 the...
Detailed Description
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow. Telomeres were reported to be short in up to one-third of ...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Short age-adjusted telomere length in the first percentile and/or a mutation in telomerase genes
- One or more of the following cytopenia(s).
- Anemia
- Symptomatic anemia with a hemoglobin \< 9.5 g/dL or red cell transfusion requirements \> 2 units/month for at least 2 months
- Reticulocyte count \< 60,000 /microL
- Thrombocytopenia
- Platelet count \< 30,000 /microL or \< 50,000 /microL associated with bleeding
- Decreased megakaryocytic precursors in the bone marrow
- Neutropenia
- Absolute neutrophil count \< 1,000 /microL
- OR
- 3\. Idiopathic pulmonary fibrosis diagnosed by either a lung biopsy of high resolution computed tomography scan of the chest according to guidelines from the American Thoracic Society and European Respiratory Society
- 4\. Age greater than or equal to 2 years
- 5\. Weight \> 12 kg
- EXCLUSION CRITERIA:
- Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient s ability to tolerate protocol therapy, or that death within 30 days is likely
- Potential subjects with cancer who are on active chemotherapeutic treatment
- Current pregnancy, or unwillingness to avoid pregnancy if of childbearing potential
- Not able to understand the investigational nature of the study or give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent.
Exclusion
Key Trial Info
Start Date :
July 19 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 14 2016
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT01441037
Start Date
July 19 2011
End Date
November 14 2016
Last Update
August 15 2018
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892