Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Completed · Phase 1
Conditions studied: Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
In brief
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) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
Key facts
- Study ID
- NCT00744692
- Run by
- Duke University
- People needed
- 22
- Starts
- 2008-10-01
- Expected to finish
- 2014-04-01
- Last updated by the study team
- 2014-08-13
Who can join
Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Duke University Medical Center Pediatric Blood and Marrow Transplant Program — Durham, North Carolina, United States
Full record on ClinicalTrials.gov
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