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

  • End date
    Jun 11, 2024
  • participants needed
  • sponsor
    Johns Hopkins All Children's Hospital
Updated on 11 October 2022
stem cell transplantation
pulmonary function test
blood disorder
hemoglobin s
gilbert's syndrome
progressive disease
blood transfusion
aplastic anemia
diamond-blackfan anemia
immune thrombocytopenia
chelation therapy
acute chest syndrome
chest syndrome
sickle cell anemia
hematologic disorders
bone marrow failure syndromes
haploidentical hematopoietic cell transplantation


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 from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.

Condition Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
Treatment Haploidentical Hematopoietic Cell Transplantation
Clinical Study IdentifierNCT04356469
SponsorJohns Hopkins All Children's Hospital
Last Modified on11 October 2022


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