N/A
N=2
Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia
Leukemia · Myelodysplasia · Acute Lymphocytic Leukemia · Acute Myelogenous Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT02007863 ↗Enrolled (actual)
2
Serious AEs
0.0%
Results posted
Dec 2015
Primary outcome: Primary: Number of Successful Unrelated Cord Blood (UCB) Transplants — 2 participants
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Umbilical Cord Blood Transfusion (Procedure); Fludarabine (Drug); Busulfan (Drug); Melphalan (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- University of Miami
- Primary completion
- Jun 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Successful Unrelated Cord Blood (UCB) Transplants |
2 | — |
Summary
Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.
Eligibility Criteria
Inclusion Criteria
- Patients must be up to 21 years of age
- Patients cannot receive total body irradiation (TBI) because of:
- Young age - = 5 abnormalities)
- Requiring > 2 cycles chemotherapy to obtain CR;
- Second or greater CR.
- First relapse with 25% blasts on bone marrow examination on Day 14 of induction therapy)]; or,
- Hypodiploidy ( 40%, or shortening fraction > 26%
- Hepatic: Bilirubin ≤ 2.5 mg/dL; and alanine transaminase (ALT), aspartate transaminase (AST) and Alkaline Phosphatase ≤ 5 x upper limit of normal (ULN)
- Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70 mL/min/1.73 m2.
- Pulmonary: Diffusing capacity of the lungs for carbon monoxide (DLCO), Forced expiratory volume in 1 second (FEV1), Forced vital capacity (FVC) (diffusion capacity) > 50% of predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation > 92% of room air.
Exclusion Criteria
- Pregnant (B-positive HCG) or breastfeeding.
- Evidence of HIV infection or HIV positive serology.
- Current uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms).
- Autologous transplant < 6 months prior to enrollment.
- Prior autologous transplant for the disease for which the UCB transplant will be performed.
- Allogeneic hematopoietic stem cell transplant < 6 months prior to enrollment.
- Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment
- Active CNS leukemia.
- Requirement of supplemental oxygen.
- HLA-matched related donor able to donate.
Data sourced from ClinicalTrials.gov (NCT02007863). 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.