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Found 25 clinical trials
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
chronic leukemia
graft versus host disease
acute leukemias
immunodeficiency
  • 28 views
  • 24 Feb, 2021
  • 1 location
Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease

This pilot trial offers the unique opportunity for both the treatment of multiple myeloma or systemic AL amyloidosis for which hematopoietic stem cell transplantation would be ordinarily indicated and the reversal of end-stage renal failure, while avoiding the risks associated with long-term standard anti-rejection therapy used in renal transplantation. The …

cancer
carcinoma
heart disease
tacrolimus
multiple myeloma
  • 171 views
  • 15 Mar, 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.

ejection fraction
graft versus host disease
stroke
hemoglobin s
pulse oximetry
  • 18 views
  • 26 Jan, 2021
  • 1 location
TPO-Mimetic Use in Children for Hemotopoietic 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 …

pancytopenia
aplastic anemia
romiplostim
neutrophil count
cytopenia
  • 0 views
  • 18 Jun, 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 …

hemoglobinopathy
hurler's syndrome
ejection fraction
transplant conditioning
follicle stimulating hormone
  • 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.

chronic myeloid leukemia
ejection fraction
neutrophil count
myeloproliferative neoplasm
cancer
  • 42 views
  • 25 Jan, 2021
  • 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 …

chronic myeloid leukemia
ejection fraction
t(1;19)
cancer
myelomonocytic leukemia
  • 58 views
  • 01 Jun, 2021
  • 1 location
Eltrombopag for People 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 …

eltrombopag
neutrophil count
danazol
prbcs
genetic disease
  • 162 views
  • 12 Jun, 2021
  • 1 location
Cancer in Inherited Bone Marrow Failure Syndromes

maturation arrest. Pearson's Syndrome: malabsorption, neutropenia, marrow failure, metabolic acidosis; ringed sideroblasts. Other bone marrow failure syndromes: e.g. Revesz Syndrome, WT, IVIC

pancytopenia
bone marrow failure disorders
cancer
dyskeratosis congenita
bone marrow failure syndromes
  • 43 views
  • 04 Jun, 2021
  • 1 location
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