Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

• Status: Completed
• Trial end date: 2014-04-01
• Target enrollment: 0
• Participant gender: All

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) regimens in pediatric patients < 21 years receiving cord blood transplantation for non-malignant disorders.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Duke University
• Treatments: Fludarabine; Hydroxyurea; Melphalan; Thiotepa

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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