CD34+ Stem Cell Infusion to Augment Graft Function
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|ClinicalTrials.gov Identifier: NCT01856582|
Recruitment Status : Terminated (CliniMACS CD34 Reagent System was FDA approved for clinical use; therefore, patients were treated clinically.)
First Posted : May 17, 2013
Last Update Posted : December 19, 2018
|Condition or disease||Intervention/treatment||Phase|
|Waning Donor Chimerism Waning Immune Function Primary Immunodeficiency Disease(s) Bone Marrow Failure||Biological: CD34+||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||23 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Post Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure Syndromes|
|Study Start Date :||October 2010|
|Actual Primary Completion Date :||August 15, 2018|
|Actual Study Completion Date :||August 15, 2018|
Experimental: CD34+ selected stem cell infusion
An infusion of selected CD34+ stem cells will be given without any preparative regimen.
CD34+ cells are selected using the CliniMACS System; without preparative regimen
- Augmentation of graft function [ Time Frame: 3, 6 and 12 months; annually thereafter until up to 3 years post-infusion ]
Graft function will be measured in two ways. Firstly the percentage of donor chimerism, and secondly measured improvement in immune function; improvement in numbers of circulating white blood cells and/or platelets, and/or clearance of opportunistic viral infections.
- Improvement in immune function studies compared to baseline levels.
- In the case of Wiskott-Aldrich syndrome, improvement of platelet count to >20,000/L.
- Clinical response to infection, if applicable.
- Frequency and characteristics of potential infusion-related toxicity [ Time Frame: up to 12 hours post-infusion ]While there is little expectation of infusion-related toxicity, the incidence of infusion-related reactions will be recorded and evaluated.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01856582
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||Rebecca Marsh, MD||Children's Hospital Medical Center, Cincinnati|