Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study

This study has been completed.
Sponsor:
Information provided by:
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00187018
First received: September 12, 2005
Last updated: February 15, 2008
Last verified: May 2007
  Purpose

Osteogenesis imperfecta (OI) is a genetic disease for which there is currently no known cure. OI causes the osteoblasts (bone-forming cells in the body) to grow poorly, which slows the growth of children with the disease and causes their bones to bend and break easily. Some forms of osteogenesis imperfecta may cause severe disability and even death. In previous research studies performed at St. Jude, it was found that children treated with bone marrow transplant (infusion of healthy immature blood-forming cells) began to grow faster, had more minerals (material that helps make the bones strong) in their bones, and broke their bones less often than before the bone marrow transplant. Several months after the bone marrow transplant however, body growth once again began to slow down. In this research study, children with osteogenesis imperfecta will receive another infusion of bone marrow cells but without any chemotherapy. The marrow cells will come from the same bone marrow donor as their previous bone marrow transplant. It is hoped that by removing the CD3+ cells (a type of white blood cells that attack other cells that are not like themselves) from the donated bone marrow, the subject's body will be infused quite safely and that body growth and bone strength will increase. The CD3+ cells will be removed from the donor bone marrow by use of a machine called the CliniMACS System. This machine has not been approved for use in the United States by the Food and Drug Administration (FDA). The use of this device is considered experimental.


Condition Intervention
Osteogenesis Imperfecta
Procedure: Bone marrow transplant

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study

Resource links provided by NLM:


Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • To find out the effects (good and bad) of bone marrow cell infusions using donor bone marrow that has had CD3+ cells removed
  • To find out if there is any effect on the growth rate of children with osteogenesis imperfecta who receive donor bone marrow which has had CD3+ cells removed
  • To find out if there is any effect on the total bone mineral content of children with OI
  • who receive donor bone marrow which has had CD3+ cells removed

Secondary Outcome Measures:
  • To find out the effect of the CD3 washed-out marrow cell therapy on the growth rate of the children

Estimated Enrollment: 14
Study Start Date: March 2004
Study Completion Date: August 2007
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must have been previously enrolled on TOIT protocol at St. Jude Children's Research Hospital
  • Must have original bone marrow donor available and willing to participate as a donor
  • Normal liver function
  • Hemoglobin >10gm/dl
  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: NCT00187018

Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Investigators
Principal Investigator: Gregory Hale, M.D. St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided by St. Jude Children's Research Hospital

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Gregory Hale, MD / Prinicipal Investigator, St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00187018     History of Changes
Other Study ID Numbers: STOD2
Study First Received: September 12, 2005
Last Updated: February 15, 2008
Health Authority: United States: Food and Drug Administration

Keywords provided by St. Jude Children's Research Hospital:
Collagen Disease

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