NCT02172885

Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Hospital de Cruces · Academic / Other
• Sex: All
• Age: 6 Months – 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine the safety and effectiveness of five infusions of characterized HLA-identical MSC in non immunosuppressed children with Osteogenesis Imperfecta (OI).

Detailed description

The principal aim of this trial is to assess the safety of non-mutated HLA-identical Mesenchymal stem cell (MSC) transplantation for OI pediatric patients irrespective of treatment with biphosphonates. Since MSC are inherently non-immunogenic and do not elicit proliferation of allogeneic lymphocytes (in co-culture experiments), a cell therapy based on HLA-identical or histocompatible (at least 5 shared out of 6 HLA antigens) allogenic MSC may be accomplished without subjecting the patients to immunosuppressor treatment. Adverse secondary effects due to immunosuppressor treatment will be avoided using this strategy thus patients may benefit from two cellular infusions. The patients will be followed for 2 years post their fifth and last MSC infusion.

Conditions

• Osteogenesis Imperfecta

Interventions

Type	Name	Description
BIOLOGICAL	Mesenchymal Stem Cells	Mesenchymal Stem Cell Infusions

Timeline

• Start date: 2014-04-01
• Primary completion: 2018-12-01
• Completion: 2018-12-01
• First posted: 2014-06-24
• Last updated: 2023-10-03

Locations

2 sites across 1 country: Spain