Clinical Trials and Studies
Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.
Low Dose Danazol for the Treatment of Telomere Related Diseases
Status(es): Recruiting
Study Date(s): Thursday, March 1, 2018 to Thursday, April 30, 2026
Disease(s): Aplastic Anemia
Age Group: 3 years and older
DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers at the National Institutes of Health (NIH) are studying if the medication danazol can be used to treat people with short telomere disease who also have bone marrow failure, liver, or lung disease.In recent studies, danazol, at high doses, showed a positive influence on telomere length. Additional research is needed to learn more...
A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, ATG) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients with Refractory or Recurrent Severe Aplastic Anemia
Status(es): Recruiting
Study Date(s): Thursday, January 4, 2024 to Thursday, March 26, 2026
Disease(s): Aplastic Anemia
Age Group: 40 years to 75 years
The current standard treatment for aplastic anemia (AA) is immunosuppressive therapy (IST), with only temporary symptom relief and high rates of paroxysmal nocturnal hemoglobinuria, clonal hematopoiesis, and myelodysplastic syndrome. When a matched donor is available, allogeneic hematopoietic cell transplant (allo-HCT) is the preferred treatment for severe aplastic anemia (SAA). But the survival rate for patients over 40 years of age is ~ 50%, mainly due to graft-versus-host disease (GvHD). There is an urgent need to find novel therapies for SAA patients who are refractory to IST and lack a...