Clinical Trial Clinical Trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease. Trials are in four phases. Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment. If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the trial sponsor.
This is a Phase II study of hematopoietic stem cell transplantation for previously untreated patients with severe aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… . 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.
Patients with a diagnosis of Severe Aplastic Anemia Aplastic Anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… who do not have a fully HLA HLA: See human leukocyte antigen. -matched related sibling donor who have not received any prior treatment. Patients need to have adequate organ function to be able to tolerate a reduced-intensity hematopoietic cell transplant.
Patients with Inherited or Congenital Bone Marrow Failure Bone Marrow Failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… Syndromes are not eligible for this study. Patients cannot have received previous treatment for Aplastic Anemia Aplastic Anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… and cannot have inadequate organ function, uncontrolled infection, or have received a prior solid organ transplant. Patients who are pregnant are not eligible for hematopoietic cell transplantation.