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Sponsored by: |
St. Jude Children's Research Hospital |
Information provided by: | St. Jude Children's Research Hospital |
ClinicalTrials.gov Identifier: | NCT00705120 |
This protocol was a prospective, Phase I study of allogeneic bone marrow transplantation (BMT) as the primary therapy for Osteogenesis Imperfecta Types II and III. Compatible sibling donors and unrelated donors were stratified and analyzed according to the type of donor. All patients with a sibling donor will received a chemotherapy conditioning regimen; a non-T cell depleted allogeneic marrow, and GVHD prophylaxis. All patients with an unrelated donor will receive a chemoradiotherapy conditioning regimen, a T-cell depleted allogeneic marrow, and GVHD prophylaxis. The primary objective of this study was to investigate the safety and toxicity of these BMT procedures in this particular population.
Condition | Intervention | Phase |
Osteogenesis Imperfecta |
Other: Bone Marrow Cell Transplantation Radiation: Irradiation, Total Body Drug: Cyclophosphamide Drug: Cyclosporin Procedure: Mesenchymal Stem Cell Transplantation Drug: Busulfan |
Phase I |
Genetics Home Reference related topics: | Melnick-Needles syndrome osteogenesis imperfecta |
MedlinePlus related topics: | Bone Marrow Transplantation Osteogenesis Imperfecta |
ChemIDplus related topics: | Cyclophosphamide Cyclosporine Cyclosporin Busulfan |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Treatment of Severe (Types II and III) Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation |
Enrollment: | 9 |
Study Start Date: | November 1995 |
Study Completion Date: | October 2007 |
Primary Completion Date: | July 2000 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
1 | Other: Bone Marrow Cell Transplantation Drug: Cyclophosphamide Drug: Cyclosporin Procedure: Mesenchymal Stem Cell Transplantation Drug: Busulfan |
2 | Other: Bone Marrow Cell Transplantation Radiation: Irradiation, Total Body Drug: Cyclophosphamide Procedure: Mesenchymal Stem Cell Transplantation Drug: Busulfan |
The secondary objective of the protocol assessed the engraftment of donor mesenchymal cells and their ability to increase the synthesis of normal type I procollagen relative to the synthesis of mutated type I procollagen and to assess whether BMT improves the bone structure and the clinical condition of these patients with OI.
Ages Eligible for Study: | 3 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Diagnosis of OI Type II
Diagnosis of OI Type III
Exclusion Criteria:
United States, Tennessee | |||||
St. Jude Children's Research Hospital | |||||
Memphis, Tennessee, United States, 38105 |
St. Jude Children's Research Hospital |
Principal Investigator: | Kimberly Kasow, DO | St. Jude Children's Research Hospital |
Related Info 
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Responsible Party: | St. Jude Children's Research Hospital ( Edwin M Horwitz, MD, PhD ) |
Study ID Numbers: | TOIT |
First Received: | June 23, 2008 |
Last Updated: | June 23, 2008 |
ClinicalTrials.gov Identifier: | NCT00705120 |
Health Authority: | United States: Institutional Review Board |
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