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Sponsors and Collaborators: |
University of Minnesota Masonic Cancer Center, University of Minnesota |
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Information provided by: | University of Minnesota |
ClinicalTrials.gov Identifier: | NCT00668564 |
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).
Condition | Intervention |
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Hurler's Syndrome Maroteaux-Lamy Syndrome Sly Syndrome Alpha Mannosidosis Fucosidosis Aspartylglucosaminuria Sphingolipidoses Krabbe Disease Wolman's Disease Niemann-Pick Disease Type B Niemann-Pick Disease, Type C |
Procedure: Stem Cell Transplantation Drug: Campath, Busulfan, Cyclophosphamide |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation |
Estimated Enrollment: | 45 |
Study Start Date: | March 2008 |
Estimated Study Completion Date: | March 2015 |
Estimated Primary Completion Date: | March 2013 (Final data collection date for primary outcome measure) |
Days before Transplant Drug Frequency
This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this is a new protocol to take the place of several older protocols. While survival has been very good on the prior protocols over the past decade, incomplete engraftment has remained somewhat problematic. Therefore, we have modified the preparative regimen somewhat to increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that is more immune suppressive. In addition, we have modified the supportive care regimen. Based on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers of inflammation and oxidative stress for the families that consent to these research studies.
Ages Eligible for Study: | up to 21 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Mucopolysaccharidosis Disorders:
Glycoprotein metabolic disorders:
Other Inherited Diseases of Metabolism:
Exclusion Criteria:
Major organ dysfunction. Evidence of major organ impairment, including:
Contact: Paul Orchard, MD | 612-626-1926 | orcha001@umn.edu |
Contact: Teresa Kvisto, RN | 612-273-2800 | tkvist1@fairview.org |
United States, Minnesota | |
University of MInnesota, Fairview | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Tim Krepski, RN 612-273-2800 | |
Contact: Teresa Kvisto, RN 612-273-2800 tkvist!@fairview.org |
Principal Investigator: | Paul Orchard, MD | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota ( Paul Orchard, M.D. ) |
Study ID Numbers: | 0801M25201, MT2008-02 |
Study First Received: | April 25, 2008 |
Last Updated: | September 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00668564 |
Health Authority: | United States: Institutional Review Board |
Inborn errors of metabolism Sphingolipidoses Recessive Leukodystrophies- GLD, Krabbe disease, MLD Peroxisomal Disorders |
Wolman syndrome Niemann-Pick B patients Niemann-Pick C subtype 2 |
Pick Disease of the Brain Sphingolipidoses Frontotemporal dementia Demyelinating diseases Brain Diseases Alpha-L-iduronidase deficiency Metabolism, Inborn Errors Mucopolysaccharidoses Alemtuzumab Infant, Newborn, Diseases Brain Diseases, Metabolic, Inborn Niemann-Pick Disease Delirium Speech Disorders Cholesterol Ester Storage Disease |
Metabolic Diseases Demyelinating Diseases Lysosomal Storage Diseases Aphasia Language Disorders Cognition Disorders Fucosidosis Niemann-Pick Disease, Type B Niemann-Pick Disease, Type C Delirium, Dementia, Amnestic, Cognitive Disorders Aspartylglycosaminuria Wolman Disease Niemann-Pick Disease, Type A Mucopolysaccharidosis type 6 Niemann-Pick disease, type C1 |
Reticuloendotheliosis Disease Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Lysosomal Storage Diseases, Nervous System Nervous System Diseases Physiological Effects of Drugs Mucinoses Hereditary Central Nervous System Demyelinating Diseases Immunosuppressive Agents |
Pharmacologic Actions Pathologic Processes Therapeutic Uses Syndrome Myeloablative Agonists Antineoplastic Agents, Alkylating Antirheumatic Agents Histiocytosis, Non-Langerhans-Cell Alkylating Agents Carbohydrate Metabolism, Inborn Errors |