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Stem Cell Transplantation for Children Affected With Osteopetrosis

Primary Purpose

Osteopetrosis

Status
Terminated
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Stem Cell Transplantation
Miltenyi Biotec CliniMACS
Systemic chemotherapy and antibodies
Sponsored by
St. Jude Children's Research Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteopetrosis focused on measuring Osteopetrosis, Autosomal recessive bone disease, Haploidentical stem cell transplantation, Allogeneic stem cell transplantation, T-cell depletion methodology, Miltenyi Biotec CliniMACS stem cell selection device

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Clinical diagnosis of malignant osteopetrosis as documented by bone marrow biopsy and radiographic imaging A suitable hematopoietic stem cell donor is available Exclusion Criteria: Participant has the Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant Symptomatic cardiac disease or evidence of significant cardiac dysfunction by ECHO (shortening fraction <30%) Creatinine clearance ≤ 40ml/min/1.73m^2 Bilirubin ≥ 3mg/dL SGPT ≥ 500 U/L Evidence of current severe infection which would preclude ablative chemotherapy or a successful transplantation Karnofsky or Lansky score < 70 noting expected abnormalities

Sites / Locations

  • St. Jude Children's Research Hospital

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

1

Arm Description

Outcomes

Primary Outcome Measures

Engraftment
To determine the need for blood or platelet transfusions and the presence of donor cells being present in the transplant recipient's bone marrow or peripheral blood by 100 day after transplantation for children with malignant infantile osteopetrosis who have received a haploidentical stem cell graft.

Secondary Outcome Measures

Full Information

First Posted
September 1, 2005
Last Updated
April 24, 2017
Sponsor
St. Jude Children's Research Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT00145587
Brief Title
Stem Cell Transplantation for Children Affected With Osteopetrosis
Official Title
Allogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
January 2011
Overall Recruitment Status
Terminated
Why Stopped
Due to the principal investigator having left the institution.
Study Start Date
July 2004 (undefined)
Primary Completion Date
February 2009 (Actual)
Study Completion Date
February 2009 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
St. Jude Children's Research Hospital

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation. Stem cells provided from an HLA identical matched sibling donor is the standard of care, but not feasible for the majority of patients. In addition, due to the potentially rapid progression of this disease, the time to identify a suitable HLA matched unrelated donor is not optimal. Therefore this study is designed to test the hypothesis that children with osteopetrosis can properly engraft hematopoietic stem cells that are donated from a partially matched parental donor, or "haploidentical" stem cell donor that are processed on the investigational device, CliniMACS selection system.
Detailed Description
The primary objective of this trial will be answered strictly by those patients enrolled who receive a haploidentical stem cell donor graft. Patients with a matched sibling donor will be offered participation in this clinical trial and will receive a standard myeloablative conditioning regimen followed by the infusion of an unmanipulated bone marrow graft. However, data from these transplant recipients will be reported in a descriptive manner only. Secondary Objectives in this trial include the following: To describe the outcome of children with MIOP who receive hematopoietic stem cells from a matched sibling donor or a haploidentical donor utilizing a uniform approach one year from transplant To estimate the fraction of children with MIOP who have a genetic defect correlating to the osteopetrosis phenotype To assess carrier-state of the genetic mutation in parents with an affected child To assess carrier-state of the genetic mutation in siblings of affected children To estimate the effect of age at the time of hematopoietic stem cell transplantation on the overall outcome of children with MIOP To describe the kinetics of select cytokine expression before and after transplantation

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteopetrosis
Keywords
Osteopetrosis, Autosomal recessive bone disease, Haploidentical stem cell transplantation, Allogeneic stem cell transplantation, T-cell depletion methodology, Miltenyi Biotec CliniMACS stem cell selection device

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Other
Intervention Type
Procedure
Intervention Name(s)
Stem Cell Transplantation
Other Intervention Name(s)
Haploidentical stem cell transplant, Allogeneic stem cell transplant
Intervention Description
An infusion of HLA partially matched family member donor stem cells processed through the use of the investigational Miltenyi Biotec CliniMACS device.
Intervention Type
Device
Intervention Name(s)
Miltenyi Biotec CliniMACS
Other Intervention Name(s)
T-cell depletion
Intervention Description
Stem cell selection device
Intervention Type
Drug
Intervention Name(s)
Systemic chemotherapy and antibodies
Other Intervention Name(s)
Transplantation for Osteopetrosis
Intervention Description
Haploidentical stem cell transplant recipients will receive a reduced intensity conditioning regimen consisting of OKT-3, Fludarabine, Thiotepa , and Melphalan followed by an infusion of a T-cell depleted donor stem cell product. Rituximab will be administered within 24 hours of the infusion in an effort to prevent post transplantation lymphoproliferative disorders (PTLPD). In addition to T-cell depletion of the donor product, cyclosporine will be provided as prophylaxis for (GVHD)Graft versus Host Disease Recipients of a matched sibling donor product will receive a myeloablative conditioning regimen consisting of busulfan and cyclophosphamide. Cyclosporine will be administered for GVHD prophylaxis.
Primary Outcome Measure Information:
Title
Engraftment
Description
To determine the need for blood or platelet transfusions and the presence of donor cells being present in the transplant recipient's bone marrow or peripheral blood by 100 day after transplantation for children with malignant infantile osteopetrosis who have received a haploidentical stem cell graft.
Time Frame
100 days post-transplant

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Clinical diagnosis of malignant osteopetrosis as documented by bone marrow biopsy and radiographic imaging A suitable hematopoietic stem cell donor is available Exclusion Criteria: Participant has the Carbonic Anhydrase II (CAII) deficiency osteopetrosis variant Symptomatic cardiac disease or evidence of significant cardiac dysfunction by ECHO (shortening fraction <30%) Creatinine clearance ≤ 40ml/min/1.73m^2 Bilirubin ≥ 3mg/dL SGPT ≥ 500 U/L Evidence of current severe infection which would preclude ablative chemotherapy or a successful transplantation Karnofsky or Lansky score < 70 noting expected abnormalities
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kimberly A Kasow, DO
Organizational Affiliation
St. Jude Children's Research Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States

12. IPD Sharing Statement

Links:
URL
http://www.stjude.org
Description
St. Jude Children's Research Hospital

Learn more about this trial

Stem Cell Transplantation for Children Affected With Osteopetrosis

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