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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: | NCT00638820 |
We believe that HSCT will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone architecture, and the anemia and bone marrow failure that is also characteristic of this disease. However, we have found in past studies that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we plan to use a different combination of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors. The purpose of this research is to find a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality.
Condition | Intervention | Phase |
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Osteopetrosis |
Procedure: Stem Cell Transplantation Drug: Campath, Busulfan, Clofarabine Procedure: Total Lymphoid Irradiation |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft |
Enrollment: | 3 |
Study Start Date: | September 2007 |
Estimated Study Completion Date: | December 2008 |
Primary Completion Date: | May 2008 (Final data collection date for primary outcome measure) |
Dose 500 cGy via AP and PA fields (250 cGy ANT and 250 cGy POST). Treated AP and PA with the patient on a specially designed couch on the floor. Doses rate 26 cGy/minute. Photon energy 6 MV used. Prescribed to the mid-thickness at the center of the field. Superior border is 2 cm below the mastoid tip. Inferior border is at the ischial tuberosity. Lateral border covers the axillary lymph nodes. Below the diaphragm the field is wide enough to encompass the liver and spleen and the mesenteric nodes.
Above the diaphragm the field encompasses cervical, infraclavicular, axillary, mediastinal, and hilar nodes.
Central block 2 cm in width partially shields the rectum, vagina, and bladder. Clam shell on the scrotum for all males if possible.
This transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). Additional biologic studies will include microarray analysis, Campath levels just prior to the administration of the graft, and establishment of mesenchymal stem cell lines. In older patients, studies to evaluation osteoclast differentiation and function will also be offered.
Ages Eligible for Study: | up to 45 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Minnesota | |
University of MInnesota, Fairview | |
Minneapolis, Minnesota, United States, 55455 |
Principal Investigator: | Paul Orchard, MD | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota ( Paul Orchard, M.D. ) |
Study ID Numbers: | 0704M06581, MT2007-06 |
Study First Received: | March 11, 2008 |
Last Updated: | September 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00638820 |
Health Authority: | United States: Institutional Review Board |
Clofarabine Osteopetrosis Musculoskeletal Diseases Busulfan Alemtuzumab |
Bone Diseases, Developmental Osteochondrodysplasias Bone Diseases Albers-Schonberg disease |
Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Therapeutic Uses Physiological Effects of Drugs Myeloablative Agonists |
Antineoplastic Agents, Alkylating Alkylating Agents Immunosuppressive Agents Pharmacologic Actions Osteosclerosis |