Stem Cell Transplantation for Children Affected With Osteopetrosis

Stopped early · Not applicable

Conditions studied: Osteopetrosis

In brief

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.

Key facts

Study ID

NCT00145587

Run by

St. Jude Children's Research Hospital

People needed

15

Starts

2004-07-01

Expected to finish

2009-02-01

Last updated by the study team

2017-05-30

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

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

Where it is running

• St. Jude Children's Research Hospital — Memphis, Tennessee, United States

Full record on ClinicalTrials.gov

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