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Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow Transplantation

This study has been completed.
Sponsor:
Collaborators:
Drexel University
Wayne State University
Information provided by:
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00186914
First received: September 9, 2005
Last updated: February 18, 2008
Last verified: February 2008
History of Changes
  Purpose

Osteodysplasia or poorly formed bones, "brittle bones" is a genetic disease with no known proven treatments. Some forms of osteodysplasia may cause severe disability and even death.

Eligible patients were limited to those children with Osteodysplasia who had undergone a previous allogeneic bone marrow transplant at St. Jude. The study intervention involved an infusion of a specified number of ex vivo expanded stromal cells obtained from the bone marrow of the same donor from whom they received their primary transplant procedure. These bone marrow stromal cells can become bone-forming cells, called osteoblasts. Participants then received 2 infusions of ex vivo expanded, gene marked cells not less than 6 months after bone marrow transplantation. The second cell infusion occurred between 14 to 21 days after the first infusion in the absence of toxicity. The goal of the study was to evaluate the safety and toxicity of these infusions.


Condition Intervention Phase
Osteodysplasia
 Procedure: Marrow stromal cell infusion
 Phase 1

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: Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow Transplantation: A Phase I Study

Resource links provided by NLM:

U.S. FDA Resources

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

Primary Outcome Measures:
  • To evaluate the safety of the stromal cell infusion [ Time Frame: June 2001 ] [ Designated as safety issue: Yes ]

Enrollment: 8
Study Start Date: July 1999
Study Completion Date: January 2008
Primary Completion Date: November 2001 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1 Procedure: Marrow stromal cell infusion

This is a pilot study of infusions of ex vivo expanded, gene marked donor bone marrow stromal cells following allogeneic bone marrow transplantation. The study is a within patient dose escalation safety evaluation. It is believed that this patient population may benefit from these donor stromal cell infusions.

As the stromal cells will be obtained from the original stem cell donor, no conditioning is required.

Patients will receive two infusions of mesenchymal cells (MSC) approximately 14 to 21 days apart unless there is unacceptable toxicity after the first infusion. The first dose of cells to be given will be 1x10^6 MSC/kg and the second dose of cells will be 5x10^6 MSC/kg. The patients will be followed for approximately 28 days following the second infusion for any toxicity. Only after all six patients have safely completed both infusions will we consider 5 x 10^6 MSC/kg a safe dose of allogeneic mesenchymal stem cells for infusion.

Other Names:
  • Marrow stromal cell infusion
  • Gene marking
  • mesenchymal cells

Detailed Description:
  • To determine whether these ex vivo expanded, gene marked marrow stromal cells will engraft in the recipient's bone, bone marrow, and/or skin.
  • To begin to investigate whether high proliferative mesenchymal progenitor cells can be expanded ex vivo and retain their progenitor potential in vivo.
  • To begin to investigate whether ex vivo expanded bone marrow stromal cells can ameliorate the skeletal dysplasias associated with various genetic disorders.
  Eligibility

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

Inclusion Criteria:

  • Patients with an appropriate genetic disorder who have had an allogeneic bone marrow transplant and currently maintain complete or mixed hematopoietic chimerism
  • Patient must be < or equal to 18Kg

Exclusion Criteria:

  • Presence of infection
  • Presence of GVHD (graft versus host disease)or the need for prophylaxis
  • Concurrent participation in any investigational study that could potentially confound the interpretation of the safety parameters being investigated in this study.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00186914

Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Drexel University
Wayne State University
Investigators
Principal Investigator: Kimberly Kasow, DO St. Jude Children's Research Hospital
  More Information

Additional Information:
St. Jude Children's Research Hospital  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Kimberly Kasow, DO / Principal Investigator, St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00186914     History of Changes
Other Study ID Numbers: STOD
Study First Received: September 9, 2005
Last Updated: February 18, 2008
Health Authority: United States: Food and Drug Administration

Keywords provided by St. Jude Children's Research Hospital:
Bone marrow transplant
bone marrow stromal cells
allogeneic transplant
Osteodysplasia
 Osteogenesis imperfecta
Gene marking
Stromal cell

ClinicalTrials.gov processed this record on May 22, 2013