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Found 18 clinical trials
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 …

aplastic anemia
chest syndrome
pancytopenia
hematologic disorders
fibrosis
  • 0 views
  • 15 Sep, 2021
  • 1 location
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders (RIC)

This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source. Recently, reduced intensity conditioning …

campath
bone marrow failure
melphalan
reduced intensity conditioning
histiocytosis
  • 1 views
  • 08 Apr, 2022
  • 1 location
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

hemoglobin e
busulfan
ctx001
  • 0 views
  • 14 Jun, 2022
  • 1 location
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

busulfan
beta thalassemia
hemoglobin e
  • 0 views
  • 21 Jul, 2022
  • 1 location
A Blood Stem Cell Transplant for Sickle Cell Disease

Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option …

chest syndrome
anti-thymocyte globulin
rabbit anti-thymocyte globulin
hemoglobin s
analgesia
  • 71 views
  • 24 Jul, 2022
  • 1 location
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

busulfan
hydroxyurea
  • 0 views
  • 13 Jun, 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.

chest syndrome
melphalan
malignant disease
sickle cell disease
cirrhosis
  • 18 views
  • 07 Mar, 2022
  • 2 locations
EDIT-301 for Autologous HSCT in Subjects With Severe Sickle Cell Disease

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).

busulfan
hydroxyurea
vaso-occlusive crisis
  • 0 views
  • 28 Jun, 2022
  • 13 locations
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.

minimal residual disease
leukemia
myeloproliferative disorder
hematologic disorder
growth factor
  • 0 views
  • 23 May, 2022
  • 1 location
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta …

chest syndrome
beta thalassemia
analgesia
hemoglobin s
hydroxyurea
  • 0 views
  • 25 Mar, 2022
  • 1 location