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Repeated Infusions of Mesenchymal Stromal Cells in Children With Osteogenesis Imperfecta (STOD3)

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ClinicalTrials.gov Identifier: NCT01061099
Recruitment Status : Completed
First Posted : February 2, 2010
Last Update Posted : April 24, 2015
Sponsor:
Information provided by (Responsible Party):
Edwin Horwitz, Nationwide Children's Hospital

February 1, 2010
February 2, 2010
April 24, 2015
February 2010
March 2013   (Final data collection date for primary outcome measure)
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 ]
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 ]
Complete list of historical versions of study NCT01061099 on ClinicalTrials.gov Archive Site
  • 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 ]
  • 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 ]
Not Provided
Not Provided
 
Repeated Infusions of Mesenchymal Stromal Cells in Children With Osteogenesis Imperfecta
A Pilot Study to Assess the Safety and Feasibility of Repeated Infusions of Mesenchymal Stromal Cells (MSC) in Children With Osteogenesis Imperfecta

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.

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.

Interventional
Phase 1
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Osteogenesis Imperfecta Type II
  • Osteogenesis Imperfecta Type III
Biological: Mesenchymal Stromal Cells
Both cohorts will receive multiple infusions of ex-vivo expanded MSCs. 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 only receive freshly harvested or cryopreserved bone marrow
Other Name: ex-vivo expanded MSCs
  • Active Comparator: Stratum A
    Subjects with a diagnosis of Type II or Type III osteogenesis imperfecta who have previously undergone a bone marrow transplant. Intervention: Mesenchymal Stromal Cells.
    Intervention: Biological: Mesenchymal Stromal Cells
  • Active Comparator: Stratum B
    Subjects with Type II or III osteogenesis imperfecta who have not undergone a bone marrow transplant. Intervention: Mesenchymal Stromal Cells.
    Intervention: Biological: Mesenchymal Stromal Cells

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
5
12
February 2014
March 2013   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Subjects less than or equal to 19 years of age at the time of enrollment
  • Children with a diagnosis of severe of Type II or III osteogenesis imperfecta
  • Parent or sibling greater than or equal to 18 years of age, donor willing to or has already undergone HLA typing, and willing and able to provide bone marrow
  • BMT greater than 5 years ago for Stratum A

Exclusion Criteria:

  • Dependent on supplemental oxygen
  • Concurrent Infection
Sexes Eligible for Study: All
up to 19 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01061099
2008-4-5947
No
Not Provided
Not Provided
Edwin Horwitz, Nationwide Children's Hospital
Nationwide Children's Hospital
Not Provided
Principal Investigator: Alix Seif, MD Children's Hospital of Philadelphia
Nationwide Children's Hospital
April 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP