Overview

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