Danazol for Genetic Bone Marrow and Lung Disorders

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01441037
• Date of registration: 24/09/2011
• Prospective Registration: No
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Danazol for Genetic Bone Marrow and Lung Disorders
• Scientific title: Male Hormones for Telomere Related Diseases
• Date of first enrolment: July 19, 2011
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01441037
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

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

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

1. Short age-adjusted telomere length in the first percentile and/or a mutation in
telomerase genes

2. One or more of the following cytopenia(s).

- Anemia

1. Symptomatic anemia with a hemoglobin < 9.5 g/dL or red cell transfusion
requirements > 2 units/month for at least 2 months

2. Reticulocyte count < 60,000 /microL

- Thrombocytopenia

1. Platelet count < 30,000 /microL or < 50,000 /microL associated with bleeding

2. Decreased megakaryocytic precursors in the bone marrow

- Neutropenia

1. 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:

1. 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

2. Potential subjects with cancer who are on active chemotherapeutic treatment

3. Current pregnancy, or unwillingness to avoid pregnancy if of childbearing
potential

4. 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.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: Danazol

Primary Outcome(s)

Number of Patients Having Attenuation of Accelerated Telomere Attrition [Time Frame: 24 months]

Secondary ID(s)

110209

11-H-0209

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/07/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01441037