HEMATOLOGIC DISEASES

This study is testing a one-time gene therapy called CTX001 in children with severe transfusion-dependent beta-thalassemia. Doctors use CRISPR gene-editing technology to modify a patient's own blood stem cells, then infuse them back, aiming to eliminate the need for lifelong bloo…

This was an early-stage study to test the safety and find the right dose of a new oral drug called HMPL-523 for adults with immune thrombocytopenia (ITP), a condition where the immune system attacks platelets, causing a risk of bleeding. The study was designed to enroll adults wh…

This study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…

This study is testing if adding a drug called vorinostat to standard medications can better prevent graft-versus-host disease (GVHD) in children and young adults getting a bone marrow transplant for blood cancers. GVHD is a serious complication where the donor's immune cells atta…

This study is testing a new support program designed to help adults who have had a stem cell transplant for blood cancer with sexual problems that are causing distress. About 120 participants will be randomly assigned to receive either this specialized program or standard educati…

This study aims to collect medical information from up to 10,000 patients and stem cell donors to help researchers learn more about a wide range of diseases and train future doctors. Participants' existing medical records, test results, and treatment data will be gathered over tw…

This study aims to understand how muscle strength and body composition affect recovery and long-term health in people with blood cancers who receive a stem cell transplant. Researchers will observe 144 patients to see if their muscle health at the time of transplant can predict c…

This study is tracking the long-term health of over 32,000 people who helped clean up the 2010 Deepwater Horizon oil spill or lived nearby. Researchers are comparing the health of workers who had direct exposure to oil and chemicals with those who did not, to understand if the sp…

This study is testing a new digital platform designed to improve how patient information is shared when someone is transferred between different hospitals. The goal is to make sure doctors at the receiving hospital have all the necessary details to provide safe, continuous care. …

This study is testing whether frozen testicular tissue can be transplanted back into men to restore sperm production. Five healthy adult men who already have children will have small tissue samples frozen during routine surgery, then transplanted back under their skin months late…

This study aims to collect tissue and blood samples from people who have received or donated bone marrow transplants for blood cancers like leukemia and lymphoma. Researchers will analyze these samples to better understand why complications happen and how to improve transplant su…