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Found 52 clinical trials
Fludarabine Based RIC for Bone Marrow Failure Syndromes

This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).

  • 0 views
  • 26 May, 2022
  • 1 location
TPO-Mimetic Use in Children for Hematopoietic Failure

scope of bone marrow failure disorders including acquired and inherited conditions as a first line of therapy along with standard of care.

leukemia
blood transfusion
platelet count
thrombopoietin receptor agonist
cytopenia
  • 0 views
  • 07 Oct, 2022
  • 1 location
Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Non-Malignant Hematological Disorders in Children

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 …

fibrosis
blood transfusion
immune thrombocytopenia
pancytopenia
sickle cell anemia
  • 0 views
  • 11 Oct, 2022
  • 1 location
Haploidentical HCT for Severe Aplastic Anemia

This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not …

ejection fraction
red blood cell transfusion
fludarabine
cyclophosphamide
glomerular filtration rate
  • 5 views
  • 07 Oct, 2022
  • 1 location
Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases

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 transplant
thrombosis
methotrexate
platelet count
cell transplantation
  • 0 views
  • 22 Oct, 2022
  • 17 locations
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 …

chronic leukemias
graft versus host disease
bone marrow failure
allogeneic hematopoietic stem cell transplant
acute leukemia
  • 130 views
  • 17 Nov, 2021
  • 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.

pulse oximetry
bone marrow transplant
graft versus host disease
blood transfusion
melphalan
  • 18 views
  • 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.

leukemia
cytopenia
myeloproliferative disorder
thrombocytopenia
tyrosine
  • 102 views
  • 24 Feb, 2022
  • 1 location
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant (ASCENT)

The ASCENT Trial is a single arm, multi-center, phase II study. The primary objective is to determine the rejection-free, severe graft-versus-host disease (GVHD)-free survival in pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing unrelated donor (URD) hematopoietic stem cell transplantation (HSCT) with abatacept added to conventional GVHD prophylaxis. The …

granulocyte colony stimulating factor
acute graft-versus-host disease
cell transplantation
tacrolimus
anemia
  • 4 views
  • 04 Feb, 2022
  • 6 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 (multicenter registry), from diagnosis and throughout follow-up during the natural history of the disease, treated or not. In parallel, biological samples (blood and/or …

  • 0 views
  • 13 Mar, 2021