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Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow Transplantation

NCT00186914 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2015-03-04

No results posted yet for this study

Summary

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.

Conditions

  • Osteodysplasia

Interventions

BIOLOGICAL

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.

Sponsors & Collaborators

Principal Investigators

  • Kimberly Kasow, DO · St. Jude Children's Research Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1999-07-31
Primary Completion
2001-11-30
Completion
2008-01-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00186914 on ClinicalTrials.gov