View clinical trials related to Anemia, Aplastic.
Filter by:Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking. Researchers want to better understand long-term outcomes in people with these diseases. Objective: To follow people diagnosed with acquired or inherited bone marrow failure disease and study the long-term effects of the disease and its treatments on organ function. Eligibility: People aged 2 years and older who have been diagnosed with acquired or inherited bone marrow failure or Telomere Biology Disorder. First degree family members may also be able to take part in the study. Design: Participants will be screened with a medical history, physical exam, and blood tests. They may have a bone marrow biopsy and aspiration. For this, a large needle will be inserted in the hip through a small cut. Marrow will be drawn from the bone. A small piece of bone may be removed. Participants may also be screened with some of the following: Cheek swab or hair follicle sample Skin biopsy Urine or saliva sample Evaluation by disease specialists (e.g., lung, liver, heart) Imaging scan of the chest Liver ultrasounds Six-Minute Walk Test Lung function test Participants will be put into groups based on their disease. They will have visits every 1 to 3 years. At visits, they may repeat some screening tests. They may fill out yearly surveys about their medicines, transfusions, pregnancy, bleeding, and so on. They may have other specialized procedures, such as imaging scans and ultrasounds. Participation will last for up to 20 years.
This is a multicenter, placebo-control, phase 3 study of hetrombopag in patients with treatment-naive severe aplastic anemia. All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study. Subjects will receive the same study drug (hetrombopag or placebo) as in study HR-TPO-SAA-III, with the same doses and administration schedule or with modifications based on the actual conditions. The primary objective of this extension study is to give the subjects participating in the HR-TPO-SAA-III study the continued access to the study drug (hetrombopag or placebo) after the completion of the HR-TPO-SAA-III study.
This is a prospective hybrid implementation-effectiveness study of a model of care for patients with bone marrow failure syndromes and inherited predisposition to haematological malignancy that includes comprehensive diagnostic genomic evaluation, multidisciplinary case review, provision of clinical care including from clinical haematologists, medical geneticists and genetic counsellors.
The study is aimed to find correlation between the assessment of cellularity according to trepanobiopsy data and the results of measuring MRI parameters.
Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China. Method The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the production of blood cells. The main purpose of this two-part study (Part A and Part B) is to test how safe and tolerable REGN7257 is in patients with SAA in which other Immunosuppressive therapies (ISTs) have not worked well. The study is looking at several other research questions to better understand the following properties of REGN7257: - Side effects that may be experienced by participants taking REGN7257 - How REGN7257 works in the body - How much REGN7257 is present in blood after dosing - If REGN7257 works to raise levels of certain blood counts after treatment - How quickly REGN7257 works to raise levels of certain blood counts - In patients for whom REGN7257 works to raise levels of certain blood counts after treatment, how many continue to show such a response throughout the study - If REGN7257 works to lower the number of platelet and red blood cell transfusions needed - How REGN7257 changes immune cell counts and composition - How the body reacts to REGN7257 and if it produces proteins that bind to REGN7257 (this would be called the formation of anti-drug antibodies [ADA])
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells. Standard treatment for SSA is a combination of 3 drugs (Cyclosporine [CsA], Eltrombopag [EPAG], and horse anti-thymocyte globulin [h-ATG]). Researchers want to see if starting people at a lower dose of CsA with EPAG before giving them h-ATG is helpful. Objective: To learn if early initiation of oral therapy with CsA and EPAG is safe and effective in people who have SAA and have not been treated with a course of immunosuppressive therapy and EPAG. Eligibility: People ages 3 and older with SAA Design: Participants will be screened with: medical history physical exam electrocardiogram blood tests family history bone marrow biopsy current medicines. Participants may be screened remotely via telephone conference. Participants will take a lower oral dose of CsA and EPAG. They will take CsA twice a day for 6 months. They will take EPAG for 6 months. Those who cannot visit the NIH Clinical Center within 72 hours will start taking the drugs at home. They will have weekly telephone calls with NIH staff until they visit the Clinical Center. Participants may get h-ATG at the Clinical Center for 4 days. For this, they will have a central line placed. It is a plastic tube inserted into a neck, chest, or arm vein. Participants will repeat most screening tests throughout the study. Participants will have follow-up visits at the Clinical Center at 3 months, 6 months, and annually for 5 years after the start of the study....
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.
Genetic mutations have closely linked to the pathogenesis and prognostication of myeloid cancers. In addition, a number of molecularly targeted agents have been developed in recent years. With the advent of next generation sequencing (NGS), we now are able to detect a wide range of mutations more rapidly, accurately, and economically. In this study, the investigators will use NGS to screen and analyze myeloid-associated gene mutations in the participants, and aim to build up the mutational landscapes of the various myeloid cancers, and investigate how these mutations are linked to clinical outcome.