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Sponsors and Collaborators: |
University of Utah National Institutes of Health (NIH) |
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Information provided by: | University of Utah |
ClinicalTrials.gov Identifier: | NCT00721864 |
We propose to determine the function of Prion Protein (PrP) in human hematopoietic stem cells. We will use as a model system that provided by nature in PNH patients who have a clone of PrP-null cells and a normal population of PrP+ cells. A competitive engraftment experiment using human PrP-null and Prp+ cells will be performed in a nude mouse followed by engraftment analysis and serial transplantation to assess long term HSC repopulation potential. The contribution of PrP to human LT-HSC proliferation/differentiation will be determined using the cobblestone area forming cell assay.
Condition |
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Paroxysmal Nocturnal Hemoglobinuria (PNH) |
Study Type: | Observational |
Study Design: | Cohort, Prospective |
Official Title: | Role of Prion Protein in Human Hematopoietic Stem Cells |
Whole Blood
Estimated Enrollment: | 20 |
Study Start Date: | May 2006 |
Estimated Study Completion Date: | March 2009 |
Estimated Primary Completion Date: | March 2009 (Final data collection date for primary outcome measure) |
Groups/Cohorts |
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Affected Population
Subjects suspected of having Paroxysmal Nocturnal Hemoglobinuria (PNH)
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Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Patients with paroxysmal nocturnal hemoglobinuria (PNH)
Inclusion Criteria:
Diagnosed with paroxysmal nocturnal hemoglobinuria
Exclusion Criteria:
1. Pregnant Women
Contact: Josef T Prchal, MD | 801-581-4220 | josef.prchal@hsc.utah.edu |
Contact: Kim Hickman, BS | 801-581-3707 | kimberly.hickman@hsc.utah.edu |
United States, Utah | |
University of Utah | Recruiting |
Salt Lake City, Utah, United States, 84132 | |
Contact: Josef T Prchal, MD 801-581-4220 josef.prchal@hsc.utah.edu | |
Contact: Kim Hickman, BS 801-581-3707 kimberly.hickman@hsc.utah.edu | |
Principal Investigator: Josef T Prchal, MD | |
Sub-Investigator: Charles Parker, MD | |
Sub-Investigator: Gerald Spangrude, PhD |
Principal Investigator: | Josef T Prchal, MD | University of Utah |
Responsible Party: | University of Utah ( Josef T. Prchal, MD ) |
Study ID Numbers: | 17790, R01HL5077-12 |
Study First Received: | July 23, 2008 |
Last Updated: | July 23, 2008 |
ClinicalTrials.gov Identifier: | NCT00721864 |
Health Authority: | United States: Institutional Review Board |
Paroxysmal Nocturnal Hemoglobinuria Prion Protein Hematopoietic stem cells Clonal disorder |
Myelodysplastic syndromes Paroxysmal nocturnal hemoglobinuria Urination Disorders Hematologic Diseases Myelodysplasia Myelodysplastic Syndromes Anemia Anemia, Hemolytic |
Hemoglobinuria Signs and Symptoms Proteinuria Preleukemia Urologic Diseases Marchiafava-Micheli disease Hemoglobinuria, Paroxysmal Bone Marrow Diseases |
Urological Manifestations |