Stem Cell Transplantation for Children Affected With Osteopetrosis

NCT00145587 | Terminated Results

• 1 other identifier: OPBMT2
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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.

  100 days post-transplant

Study Arms (1)

1

OTHER

Procedure: Stem Cell Transplantation; Device: Miltenyi Biotec CliniMACS; Drug: Systemic chemotherapy and antibodies

Interventions

Stem Cell Transplantation PROCEDURE

An infusion of HLA partially matched family member donor stem cells processed through the use of the investigational Miltenyi Biotec CliniMACS device.

Also known as: Haploidentical stem cell transplant, Allogeneic stem cell transplant

1

Miltenyi Biotec CliniMACS DEVICE

Stem cell selection device

Also known as: T-cell depletion

1

Systemic chemotherapy and antibodies DRUG

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.

Also known as: Transplantation for Osteopetrosis

1

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Clinical diagnosis of malignant osteopetrosis as documented by bone marrow biopsy and radiographic imaging
• A suitable hematopoietic stem cell donor is available

You may not qualify if:

• 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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital

MeSH Terms

Conditions

Osteopetrosis

Interventions

Stem Cell Transplantation; Antibodies; Transplantation

Condition Hierarchy (Ancestors)

Osteosclerosis; Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Surgical Procedures, Operative; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Limitations and Caveats

This study closed prior to completion of enrollment due to the principal investigator having left the institution.

Results Point of Contact

• Title: Kimberly Kasow, DO
• Organization: St. Jude Children's Research Hospital

kimberly_kasow@med.unc.edu

901-595-3300

Study Officials

• Kimberly A Kasow, DO

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2005
• First Posted: September 5, 2005
• Study Start: July 1, 2004
• Primary Completion: February 1, 2009
• Study Completion: February 1, 2009
• Last Updated: May 30, 2017
• Results First Posted: August 22, 2011

Record last verified: 2011-01

Locations

US (1)