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Found 63 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
  • 24 Jan, 2021
  • 1 location
TPO-Mimetic Use in Children for Hemotopoietic Failure

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

thrombocytopenic purpura
reticulocyte count
stem cell transplantation
thrombopoietin receptor agonist
cytopenia
  • 0 views
  • 26 Jan, 2021
  • 1 location
TCR Alpha Beta T-cell Depleted Haploidentical HCT 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 …

blood disorder
chest syndrome
immunosuppressant
gilbert's syndrome
stem cell transplantation
  • 0 views
  • 24 Jan, 2021
  • 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) or Telomere Biology Disorders (TBD) 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 …

ejection fraction
fludarabine
cyclophosphamide
red blood cell transfusion
glomerular filtration rate
  • 5 views
  • 04 Apr, 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.

conjugated bilirubin
graft versus host disease
chest syndrome
malignant disease
sickle cell disease
  • 18 views
  • 26 Jan, 2021
  • 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 …

graft versus host disease
acute leukemia
chronic leukemia
acute leukemias
stem cell transplantation
  • 28 views
  • 24 Feb, 2021
  • 1 location
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for …

hla typing
lymphoproliferative disease
malignant disease
malnutrition
graft versus host disease
  • 176 views
  • 24 Jan, 2021
  • 1 location
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.

waldenstrom's macroglobulinemia
acute leukemia
graft versus host disease
gilbert's syndrome
b-cell acute lymphoblastic leukemia
  • 42 views
  • 25 Jan, 2021
  • 1 location
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant

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 mismatched unrelated donor (URD) hematopoietic stem cell transplantation (HSCT) with abatacept added to conventional GVHD prophylaxis. …

blood disorder
stroke
granulocyte colony stimulating factor
abatacept
mycophenolate
  • 4 views
  • 25 Jan, 2021
  • 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