Clinical Trial: Repeated Infusions of Mesenchymal Stromal Cells in Children With Osteogenesis Imperfecta

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

Official Title: A Pilot Study to Assess the Safety and Feasibility of Repeated Infusions of Mesenchymal Stromal Cells (MSC) in Children With Osteogenesis Imperfecta

Brief Summary:

This is a study to evaluate the safety and effectiveness of repeated Mesenchymal Stromal Cells (MSC) infusions to patients with Type II or III osteogenesis imperfecta (OI).

Participants will receive MSC infusions approximately every 4 months to complete a total of 6 infusions over 20 months. Participants will be followed for 4 months post their last MSC infusion.


Detailed Summary:

This is a pilot study to evaluate the safety and efficacy of repeated MSC infusions to subjects with OI. This study will evaluate subjects on two separate strata. Stratum A will include subjects with a diagnosis of Type II or Type III osteogenesis imperfecta who have previously undergone a bone marrow transplant. Stratum A may use previously harvested and cryopreserved bone marrow mononuclear cells from original BMT donor or freshly harvested or cryopreserved bone marrow mononuclear cells obtained from a haploidentical healthy parent or sibling. Stratum B will include subjects with Type II or III osteogenesis imperfecta who have not undergone a bone marrow transplant. Stratum B will only receive freshly harvested or cryopreserved bone marrow mononuclear cells from a haploidentical healthy parent or sibling.

Participants will receive MSC infusions approximately every 4 months to complete a total of 6 infusions over 20 months. Participants will be followed for 12 months post their last MSC infusion.


Sponsor: Nationwide Children's Hospital

Current Primary Outcome: To determine the safety or repeated infusions of donor-derived and MSCs in subjects with severe osteogenesis imperfecta > 5years after an allogeneic bone marrow transplant and no prior bone marrow transplant. [ Time Frame: Completion of study ]

Original Primary Outcome: To determine the safety or repeated infusions of donor-derived and haploidentical MSCs in subjects with severe osteogenesis imperfecta > 5years after an allogeneic bone marrow transplant and no prior bone marrow transplant. [ Time Frame: Completion of study ]

Current Secondary Outcome:

  • To determine if MSCs elicit an immune response after repeated infusions. [ Time Frame: Completion of study ]
  • To determine the change in clinical course (growth, bone mineral content, fracture rate, development/activities) of subjects after experimental MSC intervention therapy as compared with each subject's own pre-MSC intervention therapy. [ Time Frame: Completion of Study ]


Original Secondary Outcome:

  • To determine whether haploidentical MSCs engraft in bone and bone marrow after infusion without a cytotoxic conditioning. [ Time Frame: Completion of study ]
  • To determine if haploidentical MSCs elicit an immune response after repeated infusions. [ Time Frame: Completion of study ]
  • To determine if an anti-haplo MSC immune response is found, whether it can be suppressed by peri-infusion steroid therapy. [ Time Frame: Completion of study ]
  • To determine the change in clinical course (growth, bone mineral content, fracture rate, development/activities) of subjects after experimental MSC intervention therapy as compared with each subject's own pre-MSC intervention therapy. [ Time Frame: Completion of Study ]


Information By: Nationwide Children's Hospital

Dates:
Date Received: February 1, 2010
Date Started: February 2010
Date Completion:
Last Updated: April 23, 2015
Last Verified: April 2015