Clinical Trial: Treatment of Severe Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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 Summary: 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.
Sponsor: St. Jude Children's Research Hospital

Current Primary Outcome: To investigate the safety and toxicity of allogeneic bone marrow transplantation (BMT) in children with severe Osteogenesis Imperfecta (OI) [ Time Frame: 1 year ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: St. Jude Children's Research Hospital

Dates:
Date Received: June 23, 2008
Date Started: November 1995
Date Completion:
Last Updated: June 23, 2008
Last Verified: June 2008