NCT00187018

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Mar 2004

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2004

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

September 12, 2005

Completed
4 days until next milestone

First Posted

Study publicly available on registry

September 16, 2005

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2007

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2007

Completed
Last Updated

March 4, 2015

Status Verified

May 1, 2007

Enrollment Period

3.4 years

First QC Date

September 12, 2005

Last Update Submit

March 3, 2015

Conditions

Osteogenesis Imperfecta

Keywords

Collagen Disease

Outcome Measures

Primary Outcomes (4)

  • 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 Outcomes (1)

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

Interventions

Bone marrow transplantBIOLOGICAL

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Publications (1)

  • Otsuru S, Gordon PL, Shimono K, Jethva R, Marino R, Phillips CL, Hofmann TJ, Veronesi E, Dominici M, Iwamoto M, Horwitz EM. Transplanted bone marrow mononuclear cells and MSCs impart clinical benefit to children with osteogenesis imperfecta through different mechanisms. Blood. 2012 Aug 30;120(9):1933-41. doi: 10.1182/blood-2011-12-400085. Epub 2012 Jul 24.

    PMID: 22829629DERIVED

Related Links

MeSH Terms

Conditions

Osteogenesis ImperfectaCollagen Diseases

Interventions

Bone Marrow Transplantation

Condition Hierarchy (Ancestors)

OsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesConnective Tissue DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Tissue TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Officials

  • Gregory Hale, M.D.

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 12, 2005

First Posted

September 16, 2005

Study Start

March 1, 2004

Primary Completion

August 1, 2007

Study Completion

August 1, 2007

Last Updated

March 4, 2015

Record last verified: 2007-05

Locations

US(1)