Phase 2 N=27 Treatment

Danazol for Genetic Bone Marrow and Lung Disorders

Aplastic Anemia

Bottom Line

View on ClinicalTrials.gov: NCT01441037 ↗

Enrolled (actual)

Serious AEs

11.1%

Results posted

Jul 2018

Primary outcome: Primary: Number of Patients Having Attenuation of Accelerated Telomere Attrition — 12 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Danazol (Drug)
Age: Pediatric, Adult, Older Adult · 2+ yrs
Sex: All
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Primary completion: Nov 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Patients Having Attenuation of Accelerated Telomere Attrition	12	—

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.

Eligibility Criteria

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 2 units/month for at least 2 months
Reticulocyte count 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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01441037). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.