Stem Cell Transplantation for Children Affected With Osteopetrosis

• Status: Terminated
• Trial end date: 2009-02-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: St. Jude Children's Research Hospital
• Treatments: Antibodies

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