Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

    Not Recruiting
  • End date
    Dec 16, 2022
  • participants needed
  • sponsor
    New York Medical College
Updated on 2 June 2021
cell transplantation
hemoglobin s
blood transfusion
iron overload
acute chest syndrome
chest syndrome
sickle cell trait
sickle hemoglobin


This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.

Funding Source - FDA OOPD


The purpose of this study is to investigate host myeloimmunosuppressive conditioning followed by familial haploidentical T cell depleted allogeneic stem cell transplantation in patients with high risk Sickle Cell Disease (SCD). It is hypothesized that it will be safe and well tolerated, and result in sustained donor chimerism, acceptable engraftment and immune reconstitution. Also, that it will limit SCD related organ damage resulting in improved and/or stable neurological, neurocognitive, pulmonary and pulmonary vascular function and health related quality of life (QOL).

Patients 2-20.99 years of age with a diagnosis of high-risk SCD and with an unaffected HLA partially matched family donor and meeting eligibility criteria (inclusion and exclusion criteria) are eligible.

Condition SICKLE CELL ANEMIA, Sickle Cell Disease, Sickle Cell Disease
Treatment CD34 selected T-cell depleted allogeneic SCT
Clinical Study IdentifierNCT01461837
SponsorNew York Medical College
Last Modified on2 June 2021

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