- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01856582
CD34+ Stem Cell Infusion to Augment Graft Function
Post Transplant CD34+ Selected Stem Cell Infusion to Augment Graft Function in Children With Primary Immunodeficiency Diseases and Bone Marrow Failure Syndromes
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Ohio
-
Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
To be eligible for this protocol, patients must have the following:
- 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.
- 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.
- Available primary donor.
- Must not have other organ dysfunction deemed by the attending physician to preclude this procedure.
- Age < 35 years at time of transplant
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:
- Absence of an available original donor
- Failure to sign consent form, or inability to undergo informed consent process
- Pregnant or lactating female
- Uncontrolled GVHD
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants Who Showed Successful Augmentation of Graft Function
Time Frame: 12 months
|
Successful augmentation of graft function achieved if donor chimerism is doubled compared to the value immediately pre-infusion at 12 months.
|
12 months
|
Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Rebecca Marsh, MD, Children's Hospital Medical Center, Cincinnati
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2010-2344
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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