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Found 54 clinical trials
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

Background Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which …

horse anti-thymocyte globulin
neutrophil count
hematologic disorder
blood test
blood disorder
  • 115 views
  • 21 Apr, 2022
  • 1 location
Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes

Inherited bone marrow failure syndromes (IBMFSs) are a diverse collection of genetic illnesses characterized by various degrees of peripheral cytopenias due to defective single-lineage or multi

Accepts healthy volunteers
  • 0 views
  • 04 Oct, 2022
  • 1 location
The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes.

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

  • 0 views
  • 21 Oct, 2022
  • 1 location
Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study

Background A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders

kostmann's syndrome
anemia
synostosis
fanconi syndrome
dyskeratosis congenita
  • 43 views
  • 24 Oct, 2022
  • 2 locations
French National Registry of Bone Marrow Failures

This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France

  • 0 views
  • 13 Mar, 2021
Clinical Care Consortium of Telomere-Associated Ailments (CCCTAA) Database

Study Description: This protocol will be utilized for the creation and management of a repository of coded clinical data on patients with Telomere Biology Disorders (TBDs) submitted by researchers from CCCTAA member institutions. Objective The primary objective of this study is to develop a shared database (repository) of coded clinical …

  • 0 views
  • 11 Jul, 2022
  • 1 location
Diamond Blackfan Anemia Registry (DBAR) (DBAR)

The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA).

anemia
white blood cell count
malnutrition
platelet count
  • 10 views
  • 27 Apr, 2022
  • 1 location
Investigation of the Genetics of Hematologic Diseases

The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood, bone marrow, hair follicles, nail clippings, urine, saliva …

hematologic disorder
hydroxyurea
blood disorder
sickle cell anemia
sickle cell disease
  • 112 views
  • 15 Jun, 2022
  • 1 location
Collection of Blood, Bone Marrow, Urine, and/or Tissue Samples From Patients With Solid Tumors, Hematological Malignancies or Non-Malignant Hematologic Disorders or HLA Compatible Family Members

Patients with a cancerous solid tumor or a cancerous or non-cancerous blood disorder or a bone marrow failure condition who cannot participate in an NIH treatment protocol or travel to the NIH

malignant solid neoplasm
blood disorder
bone marrow procedure
Accepts healthy volunteers
treatment regimen
  • 43 views
  • 21 Oct, 2022
  • 1 location
The Natural History of GATA2 Deficiency and Related Disorders

Background GATA2 deficiency is a genetic disorder that can cause problems with a person s immune system and other body systems. Some people who have this disorder develop few problems from it. Others can have a wide range of health problems, from skin problems, to hearing loss, to cancer. These …

Accepts healthy volunteers
cancer
  • 34 views
  • 24 Oct, 2022
  • 1 location