Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow Transplantation

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.

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Key information

Conditions

Osteodysplasia Bone Diseases Bone Diseases, Developmental Carbohydrate Metabolism, Inborn Errors Collagen Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Musculoskeletal Diseases Nutritional and Metabolic Diseases Osteochondrodysplasias Osteogenesis Imperfecta Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Marrow stromal cell infusion

Biological

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: Gene marking, mesenchymal cells

Primary outcomes

To evaluate the safety of the stromal cell infusion

Time frame: June 2001

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Collaborators

Drexel University
Wayne State University

Registry information

Official study title

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

Important dates

Study start: 1999
Primary completion: 2001
Study completion: 2008
First posted: Sep 16, 2005
Registry last updated: Mar 4, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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