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Found 28 clinical trials
Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders

, immune dysregulatory disorders, hemophagocytic lymphohistiocytosis, bone marrow failure syndromes, and hemoglobinopathies. Powerful chemotherapy drugs are often used to condition the patient before

bone marrow procedure
primary cancer
treatment regimen
  • 29 Apr, 2022
  • 1 location
Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

The goal of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are …

bone marrow failure
allogeneic hematopoietic stem cell transplant
stem cell transplantation
  • 17 Nov, 2021
  • 1 location
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of …

bone marrow failure
antithymocyte globulin
  • 22 Jul, 2022
  • 14 locations
Single Arm Prospective Open Label Pilot Study Evaluating Short-Term Safety and Efficacy of Romiplostim in Children With Inherited and Acquired Disorders of Hematopoietic Failure

This is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure disorders including acquired and inherited conditions as a first line of therapy along with standard of …

severe aplastic anemia
thrombocytopenic purpura
aplastic anemia
  • 18 Sep, 2022
  • 1 location
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.

graft-versus-host disease
mycophenolate mofetil
direct bilirubin
  • 07 Mar, 2022
  • 2 locations
UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep

This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment. Single or double unit selected according to current University of Minnesota umbilical cord blood graft selection algorithm.

myeloproliferative disorder
flow cytometry
nodal mass
blood transfusion
renal dysfunction
  • 24 Feb, 2022
  • 1 location
Myeloablative Allo HSCT With Related or Unrelated Donor for Heme Disorders

This is a Phase II study of allogeneic hematopoietic stem cell transplant (HCT) using a myeloablative preparative regimen (of either total body irradiation (TBI); or, fludarabine/busulfan for patients unable to receive further radiation). followed by a post-transplant graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate …

myeloproliferative disorder
flow cytometry
nodal mass
blood transfusion
renal dysfunction
  • 06 May, 2022
  • 1 location
Eltrombopag for Patients With Fanconi Anemia

Background Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new …

fanconi syndrome
platelet count
neutrophil count
chromosome breakage
  • 19 Sep, 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
  • 14 Jul, 2022
  • 1 location
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 and treatments are lacking. Researchers want to better understand long-term outcomes in people with these diseases. Objective To follow people diagnosed with acquired …

  • 21 Jul, 2022
  • 1 location