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Found 210 clinical trials
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 …

blood transfusion
stem cell transplantation
hemoglobin c disease
hydroxyurea
chest syndrome
  • 0 views
  • 25 Mar, 2022
  • 1 location
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

stem cell transplantation
alemtuzumab
anemia
antithymocyte globulin
fludarabine
  • 231 views
  • 22 Apr, 2022
  • 1 location
Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps

hemoglobinopathy
priapism
anti-thymocyte globulin
chest syndrome
tacrolimus
  • 0 views
  • 29 Jun, 2022
  • 1 location
Pilates Physical Activity in Sickle Cell Disease

Sickle cell anemia (SCA) is a life-threatening hereditary hemoglobinopathy characterized by hemoglobin (Hb) polymerization that affects many people worldwide.Reduced physical capacity is common

hemoglobinopathy
  • 0 views
  • 07 Oct, 2022
  • 1 location
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) 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).

vaso-occlusive crisis
hydroxyurea
busulfan
  • 0 views
  • 21 Oct, 2022
  • 15 locations
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 …

blood transfusion
stem cell transplantation
hydroxyurea
tissue type
transcranial doppler ultrasonography
  • 71 views
  • 04 Oct, 2022
  • 1 location
  • 0 views
  • 21 Oct, 2022
  • 2 locations
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 …

gilbert's syndrome
blood transfusion
pulmonary function test
hydroxyurea
stem cell transplantation
  • 0 views
  • 11 Oct, 2022
  • 1 location
The BENeFiTS Trial in Beta Thalassemia Intermedia (PB04-001)

Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers

thalassemia
  • 11 views
  • 03 May, 2022
  • 4 locations
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 …

cyclosporine
melphalan
alemtuzumab
immunodeficiency
severe combined immunodeficiency
  • 1 views
  • 08 Apr, 2022
  • 1 location