NCT00705120

Treatment of Severe Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation

Treatment of Severe (Types II and III) Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Osteogenesis Imperfecta

Interventions

Type	Name	Description
OTHER	Bone Marrow Cell Transplantation
RADIATION	Irradiation, Total Body
DRUG	Cyclophosphamide
DRUG	Cyclosporin
PROCEDURE	Mesenchymal Stem Cell Transplantation
DRUG	Busulfan

Timeline

• Start date: 1995-11-01
• Primary completion: 2000-07-01
• Completion: 2007-10-01
• First posted: 2008-06-25
• Last updated: 2008-06-25

Locations

1 site across 1 country: United States