Osteogenesis Imperfecta Type III Clinical Trial
— STOD3Official title:
A Pilot Study to Assess the Safety and Feasibility of Repeated Infusions of Mesenchymal Stromal Cells (MSC) in Children With Osteogenesis Imperfecta
Verified date | April 2015 |
Source | Nationwide Children's Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 5 |
Est. completion date | February 2014 |
Est. primary completion date | March 2013 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 19 Years |
Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Nationwide Children's Hospital |
United States,
Horwitz EM, Gordon PL, Koo WK, Marx JC, Neel MD, McNall RY, Muul L, Hofmann T. Isolated allogeneic bone marrow-derived mesenchymal cells engraft and stimulate growth in children with osteogenesis imperfecta: Implications for cell therapy of bone. Proc Natl Acad Sci U S A. 2002 Jun 25;99(13):8932-7. — View Citation
Horwitz EM, Prockop DJ, Fitzpatrick LA, Koo WW, Gordon PL, Neel M, Sussman M, Orchard P, Marx JC, Pyeritz RE, Brenner MK. Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta. Nat Med. 1999 Mar;5(3):309-13. — View Citation
Horwitz EM, Prockop DJ, Gordon PL, Koo WW, Fitzpatrick LA, Neel MD, McCarville ME, Orchard PJ, Pyeritz RE, Brenner MK. Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta. Blood. 2001 Mar 1;97(5):1227-31. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | Completion of study | Yes | |
Secondary | To determine if MSCs elicit an immune response after repeated infusions. | Completion of study | Yes | |
Secondary | 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. | Completion of Study | Yes |
Status | Clinical Trial | Phase | |
---|---|---|---|
Not yet recruiting |
NCT05559801 -
Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)
|
Phase 1/Phase 2 | |
Completed |
NCT03118570 -
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
|
Phase 2 |