Status:
TERMINATED
Stem Cell Transplantation for Children Affected With Osteopetrosis
Lead Sponsor:
St. Jude Children's Research Hospital
Conditions:
Osteopetrosis
Eligibility:
All Genders
Phase:
NA
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 tra...
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 part...
Eligibility Criteria
Inclusion
- Clinical diagnosis of malignant osteopetrosis as documented by bone marrow biopsy and radiographic imaging
- A suitable hematopoietic stem cell donor is available
Exclusion
- 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
Key Trial Info
Start Date :
July 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2009
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT00145587
Start Date
July 1 2004
End Date
February 1 2009
Last Update
May 30 2017
Active Locations (1)
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1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105