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Treatment of Severe Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation

NCT ID: NCT00705120

Last Updated: 2008-06-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

1995-11-30

Study Completion Date

2007-10-31

Brief Summary

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This protocol was a prospective, Phase I study of allogeneic bone marrow transplantation (BMT) as the primary therapy for Osteogenesis Imperfecta Types II and III. Compatible sibling donors and unrelated donors were stratified and analyzed according to the type of donor. All patients with a sibling donor will received a chemotherapy conditioning regimen; a non-T cell depleted allogeneic marrow, and GVHD prophylaxis. All patients with an unrelated donor will receive a chemoradiotherapy conditioning regimen, a T-cell depleted allogeneic marrow, and GVHD prophylaxis. The primary objective of this study was to investigate the safety and toxicity of these BMT procedures in this particular population.

Detailed Description

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The secondary objective of the protocol assessed the engraftment of donor mesenchymal cells and their ability to increase the synthesis of normal type I procollagen relative to the synthesis of mutated type I procollagen and to assess whether BMT improves the bone structure and the clinical condition of these patients with OI.

Conditions

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Osteogenesis Imperfecta

Keywords

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Osteogenesis Imperfecta Bone Marrow Cell Transplantation Mesenchymal Stem Cells Stem Cell Transplantation, Mesenchymal

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Group Type OTHER

Bone Marrow Cell Transplantation

Intervention Type OTHER

Cyclophosphamide

Intervention Type DRUG

Cyclosporin

Intervention Type DRUG

Mesenchymal Stem Cell Transplantation

Intervention Type PROCEDURE

Busulfan

Intervention Type DRUG

2

Group Type OTHER

Bone Marrow Cell Transplantation

Intervention Type OTHER

Irradiation, Total Body

Intervention Type RADIATION

Cyclophosphamide

Intervention Type DRUG

Mesenchymal Stem Cell Transplantation

Intervention Type PROCEDURE

Busulfan

Intervention Type DRUG

Interventions

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Bone Marrow Cell Transplantation

Intervention Type OTHER

Irradiation, Total Body

Intervention Type RADIATION

Cyclophosphamide

Intervention Type DRUG

Cyclosporin

Intervention Type DRUG

Mesenchymal Stem Cell Transplantation

Intervention Type PROCEDURE

Busulfan

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Patient has diagnosis of OI Type II or III. Because there are no specific, defined clinical criteria consistently used to make this diagnosis, we provide the following clinical guidelines to assist in diagnosis. Any appropriate combinations of the following clinical findings will be acceptable.

Diagnosis of OI Type II

* Antenatal ultrasonography (if performed for other indications) by established obstetric impressions including short femurs, a small thoracic cage and poorly mineralized bones. Analysis of collagen synthesized from cultured cells obtained from chorionic villus sampling (CVS) may establish the diagnosis; however, no CVS will be performed specifically for enrollment into this study.
* Clinical examination including prematurity, low birth weight, characteristic facies (blue sclera, beaked nose, extremely soft calvarium), "frog-leg" hips, small thoracic cavity, fractures at birth or shortly thereafter, loose skin or lax joints that cannot be readily explained by other factors.
* Radiographic evaluation demonstrating various aspects of the characteristic picture of telescoped femur, bowed tibias, beaded ribs, flattened vertebral bodies and virtual absence of calvarial mineralization.

Diagnosis of OI Type III

* Antenatal ultrasonography (performed for other indications) by established obstetric impressions for this more moderate form of OI. Chorionic villus sampling will be accepted as above.
* Clinical examination including short stature, bony deformities, many fractures at birth or shortly thereafter. Blue scleras and dental abnormalities are also common.
* Radiographic abnormalities including thin, osteopenic bones of the limbs with evidence of fractures, growth plate abnormalities, and an undermineralized calvarium.
* Diagnosis of other diseases with possibly similar presentation to OI (e.g. hypophosphatasia and rickets) should be excluded by obtaining a serum calcium, phosphate and alkaline phosphatase. These parameters can be expected to be within normal limits (alkaline phosphatase may be somewhat elevated) in patients with OI.
* Age less than 3 years at time of transplant.
* Parents or legal guardians must sign an informed consent indicating that they are aware this is a research study and have been told of its possible benefits and toxic side effects, including treatment related mortality. Patients or their guardians will be given a copy of the consent form.
* Identification of a suitable bone marrow donor.
* Any donor must be of sufficient size so that adequate bone marrow may be harvested.
* HLA mismatched sibling or unrelated donor. DNA typing will be per- formed on unrelated donors. Donors must be a 6/6 match or a 5/6 match (with serologic mismatch at a single Class I allele or mismatch at a single DR1 allele).

Exclusion Criteria

* Patients who are ventilatory dependent due to primary lung parenchymal disease prior to BMT.
* Patients with evidence of basilar invagination/compression.
Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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St. Jude Children's Research Hospital

OTHER

Sponsor Role lead

Responsible Party

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St. Jude Children's Research Hospital

Principal Investigators

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Kimberly Kasow, DO

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Locations

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St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Countries

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United States

Related Links

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http://www.stjude.org

Related Info

Other Identifiers

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TOIT

Identifier Type: -

Identifier Source: org_study_id