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.
Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US
Status(es): Enrolling
Study Date(s): Friday, May 31, 2024 to Wednesday, April 30, 2025
Disease(s): Paroxysmal Nocturnal Hemoglobinuria (PNH)
Age Group: 18 years and older
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.
The study will be prospective and observational, conducted over an initial period...
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...
The National Myelodysplastic Syndromes Natural History Study
Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Monday, March 31, 2025
Disease(s): Myelodysplastic Syndromes (MDS)
Age Group: 18 years and older
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Participants will be followed long term. Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.
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...