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Waning Donor Chimerism | CD34+ Stem Cell Infusion to Augment Graft Function

Waning Donor Chimerism research study

What is the primary objective of this study?

The purpose of this study is to determine if infusing additional special donor cells will help to improve graft or immune function in previously transplanted children with immune deficiencies and bone marrow failures.

Who is eligible to participate?

Inclusion Criteria: To be eligible for this protocol, patients must have the following: 1. Primary immunodeficiency (e.g. SCID, Wiskott-Aldrich and/or other more rare conditions and other bone marrow failure syndromes) with prior allogeneic stem cell transplant. 2. Waning donor chimerism or immune function that is inadequate to correct their disease or clinical condition, for which primary transplant was given, as determined by their attending physician. 3. Available primary donor. 4. Must not have other organ dysfunction deemed by the attending physician to preclude this procedure. 5. Age < 35 years at time of transplant 6. One of the following must be true: - Patients must have evidence of persistent or recurrent immunodeficiency or thrombocytopenia. -OR- • Primary immunodeficiency disease with known potential to progress to malignant condition if untreated. -OR- • Debilitating secondary disease known to be a consequence of inadequate immune response to known agent or pathogen, uncontrollable by other available medical therapies (e.g. third patient described on page 5). Exclusion Criteria: 1. Absence of an available original donor 2. Failure to sign consent form, or inability to undergo informed consent process 3. Pregnant or lactating female 4. Uncontrolled GVHD

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Waning Donor Chimerism

Waning Immune Function

Primary Immunodeficiency Disease(s)

Bone Marrow Failure

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Biological:CD34+CD34+ cells are selected using the CliniMACS System; without preparative regimen

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

CD34+ selected stem cell infusionAn infusion of selected CD34+ stem cells will be given without any preparative regimen.

Study Status

Active, not recruiting

Start Date: October 2010

Completed Date: July 2018

Phase: Phase 2

Type: Interventional

Design:

Primary Outcome: Augmentation of graft function

Secondary Outcome: Frequency and characteristics of potential infusion-related toxicity

Study sponsors, principal investigator, and references

Principal Investigator: Rebecca Marsh, MD

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Collaborator: Hoxworth Blood Center

More information:https://clinicaltrials.gov/show/NCT01856582

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