Clinical Trials and Research Studies
Clinical trials and research studies, such as non-therapeutic studies and market research studies, help identify new ways to prevent, detect or treat disease and understand patient needs. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, non-therapeutic study, or market research study.
Non-therapeutic Study PLEDGE – Patients’ Long-term Experience on VOYDEYA (Danicopan): Gathering Evidence in the Real-World
Status(es): Recruiting
Study Date(s): Monday, May 12, 2025 to Sunday, August 30, 2026
Disease(s): Paroxysmal Nocturnal Hemoglobinuria (PNH)
Age Group: 18 years and older
The IRB-approved observational Phase IV PLEDGE study is set-up to collect real-world data on adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) with symptomatic extravascular hemolysis who recently started VOYDEYATM (danicopan) as add-on treatment to ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab).
The Study will collect & analyze patient-reported outcome (PRO) assessments related to fatigue and quality of life and short custom surveys using a digital patient platform and electronic medical records from adult patients with PNH and extravascular hemolysis. Study...
Market Research Study Understanding the Paroxysmal Nocturnal Hemoglobinuria Journey
Status(es): Recruiting
Study Date(s): Tuesday, September 2, 2025 to Wednesday, September 2, 2026
Disease(s): Paroxysmal Nocturnal Hemoglobinuria (PNH)
Age Group: 21 years and older
The purpose of this study is to better understand the treatment journey and experiences of people living with Paroxysmal Nocturnal Hemoglobinuria (PNH), specifically those currently in a “watch and wait” period after being diagnosed.
Clinical Trial CureAA: Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Status(es): Recruiting
Study Date(s): Sunday, December 1, 2024 to Thursday, February 1, 2029
Disease(s): Aplastic Anemia
Age Group: 3 years to 75 years
This is a Phase II study of hematopoietic stem cell transplantation for previously untreated patients with severe aplastic anemia. This study is comprised of two cohorts - patients receiving haploidentical donor transplant and patients receiving unrelated donor transplant. The goal is to enroll 60 participants total, 30 in each cohort.
Clinical Trial 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...
Clinical Trial 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...