Stem Cell Transplantation for Children Affected With Osteopetrosis
Osteopetrosis
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
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
Other
1