Status:
COMPLETED
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Lead Sponsor:
Duke University
Conditions:
Non Malignant Disorders
Immunodeficiencies
Eligibility:
All Genders
Up to 21 years
Phase:
PHASE1
Brief Summary
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimen...
Detailed Description
Myeloablative doses of chemotherapy and/or radiation therapy are employed with the primary purpose of eradicating malignant cells. Additionally, these regimens exert varying degree of immunosuppressio...
Eligibility Criteria
Inclusion
- 0-21 years of age with a diagnosis of a immunodeficiency, congenital marrow failure syndrome, inborn error of metabolism, or hereditary anemia
- Appropriately matched related or unrelated umbilical cord blood unit with a cell dose ≥ 3 x 10e7cells/kg
- Performance score (lansky or karnofsky) greater than or equal to 70
- Adequate organ function (Creatinine ≤ 2.0 mg/dl and creatinine clearance ≥ 50 ml/min/1.73 m2; Hepatic transaminases (ALT/AST) ≤ 4 x normal; Shortening fraction \>26% or ejection fraction \>40% or \> 80% of normal value for age; Pulmonary function tests demonstrating CVC or FEV1/FVC of \>60% of predicted for age.)
- Informed consent
- Not pregnant or breast feeding
- Minimum life expectancy of at least 6 months
- HIV negative
- No uncontrolled infections at the time of cytoreduction
- Disease specific inclusion criteria
Exclusion
- Patients with hemoglobinopathies \> 3 years of age
- UCB unit with a total nucleated cell count \< 3 x 10e7/kg or \> 2 antigen mismatching
- Available HLA-matched related living donor able to donate without previous UCB donation
- Allogeneic hematopoietic stem cell transplant within the previous 6 months
- Any active malignancy, MDS, or any history of malignancy
- Severe acquired aplastic anemia
- DLCO \< 60% of normal value for age; requirement for supplemental oxygen
- Uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms)
- Pregnancy or nursing mother
- HIV/HTLV seropositive, Hep B surface antigen positive, or HCV RNA positive by PCR
- Any condition that precludes serial follow-up
Key Trial Info
Start Date :
October 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2014
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT00744692
Start Date
October 1 2008
End Date
April 1 2014
Last Update
August 13 2014
Active Locations (1)
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1
Duke University Medical Center Pediatric Blood and Marrow Transplant Program
Durham, North Carolina, United States, 27705