Phase 1
N=22
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Non Malignant Disorders · Immunodeficiencies · Congenital Marrow Failures · Hemoglobinopathies · Inborn Errors of Metabolism
Bottom Line
View on ClinicalTrials.gov: NCT00744692 ↗Enrolled (actual)
22
Serious AEs
81.8%
Results posted
Aug 2014
Primary outcome: Primary: Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders. — 88 % of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Unrelated Umbilical Cord Blood Transplant (Biological); Reduced Intensity Conditioning (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Duke University
- Primary completion
- Dec 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders. |
88 | — |
| SECONDARY To Describe the Pace of Neutrophil Recovery |
20 | — |
| SECONDARY To Evaluate the Pace of Immune Reconstitution. |
805 | — |
| SECONDARY To Determine the Overall Survival at day180 Post-transplant |
81.8 | — |
| SECONDARY To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV) |
22.7 | — |
| SECONDARY To Describe the Incidence of Grade 3-4 Organ Toxicity |
— | — |
| SECONDARY To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure |
— | — |
| SECONDARY To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant |
— | — |
| SECONDARY To Describe the Pace of Platelet Recovery |
48 | — |
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.
Eligibility 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 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
Data sourced from ClinicalTrials.gov (NCT00744692). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.