Trial record 1 of 21 for:    "Osteogenesis Imperfecta"
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Repeated Infusions of Mesenchymal Stromal Cells in Children With Osteogenesis Imperfecta (STOD3)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Edwin Horwitz, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT01061099
First received: February 1, 2010
Last updated: October 23, 2013
Last verified: October 2013
  Purpose

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.


Condition Intervention Phase
Osteogenesis Imperfecta Type II
Osteogenesis Imperfecta Type III
Biological: Mesenchymal Stromal Cells
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study to Assess the Safety and Feasibility of Repeated Infusions of Mesenchymal Stromal Cells (MSC) in Children With Osteogenesis Imperfecta

Resource links provided by NLM:


Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To determine if MSCs elicit an immune response after repeated infusions. [ Time Frame: Completion of study ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 12
Study Start Date: February 2010
Estimated Study Completion Date: December 2013
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
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 Names:
  • Mesenchymal Stromal Cells
  • ex-vivo expanded MSCs
Active Comparator: Stratum B
Subjects with Type II or III osteogenesis imperfecta who have not undergone a bone marrow transplant. Intervention: Mesenchymal Stromal Cells.
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 Names:
  • Mesenchymal Stromal Cells
  • ex-vivo expanded MSCs

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   up to 19 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01061099

Locations
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19105
Sponsors and Collaborators
Edwin Horwitz
Investigators
Principal Investigator: Ed Horwitz, MD Children's Hospital of Philadelphia
  More Information

Publications:
Responsible Party: Edwin Horwitz, Attending Physician, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT01061099     History of Changes
Other Study ID Numbers: 2008-4-5947
Study First Received: February 1, 2010
Last Updated: October 23, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital of Philadelphia:
Type II or Type III Osteogenesis imperfecta
OI
Mesenchymal Stromal Cells
MSC

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases

ClinicalTrials.gov processed this record on July 22, 2014