Phase 2
N=160
Donor Umbilical Cord Blood Transplant With or Without Ex-vivo Expanded Cord Blood Progenitor Cells in Treating Patients With Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or Myelodysplastic Syndromes
Acute Biphenotypic Leukemia · Acute Lymphoblastic Leukemia in Remission · Acute Myeloid Leukemia in Remission · Chronic Myelogenous Leukemia · Myelodysplastic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01690520 ↗Enrolled (actual)
160
Serious AEs
64.4%
Results posted
Jul 2021
Primary outcome: Primary: Time to Neutrophil Engraftment — 20.0; 22.0 days
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Cyclophosphamide (Drug); Cyclosporine (Drug); Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion (Biological); Fludarabine Phosphate (Drug); Mycophenolate Mofetil (Drug); Thiotepa (Drug); Total-Body Irradiation (Radiation); Umbilical Cord Blood Transplantation (Procedure)
- Age
- Pediatric, Adult, Older Adult · 0+ yrs
- Sex
- All
- Sponsor
- Nohla Therapeutics, Inc.
- Primary completion
- Sep 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Time to Neutrophil Engraftment |
20.0; 22.0 | — |
| SECONDARY Time to Platelet Engraftment (20k) |
40.0; 38.0 | — |
| SECONDARY Overall Survival |
52; 57 | — |
| SECONDARY Non-relapse Mortality |
16; 16 | — |
| SECONDARY Proportion of Patients With Severe Acute Graft Versus Host Disease |
0.14; 0.16 | — |
| SECONDARY Proportion of Participants With Chronic Graft Versus Host Disease |
27; 23 | — |
Summary
This randomized phase II trial studies how well donor umbilical cord blood transplant with or without ex-vivo expanded cord blood progenitor cells works in treating patients with acute myeloid leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, or myelodysplastic syndromes. Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's cells. When the healthy stem cells and ex-vivo expanded cord blood progenitor cells are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether giving donor umbilical cord blood transplant plus ex-vivo expanded cord blood progenitor cells is more effective than giving a donor umbilical cord blood transplant alone.
Eligibility Criteria
Inclusion Criteria
- Age criteria:
- High dose TBI regimen: 6 months to = = 15% for age
- Patients in which adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the principal investigator prior to enrollment
- Acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
- High risk first complete remission (CR1) (for example, but not limited to: t(9;22), t(1;19), t(4;11) or other mixed-lineage leukemia [MLL] rearrangements, hypodiploid); or high risk (HR) as defined by referring institution treatment protocol greater than 1 cycle to obtain CR; second complete remission (CR2) or greater
- All patients must be in CR as defined by = 15% for age
- Patients in which adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the principal investigator prior to enrollment
- Chronic myelogenous leukemia excluding refractory blast crisis; to be eligible in first chronic phase (CP1) patient must have failed or be intolerant to tyrosine kinase inhibitor therapy
- Myelodysplasia (MDS) International Prognostic Scoring System (IPSS) intermediate (Int)-2 or high risk (i.e., refractory anemia with excess blasts [RAEB], refractory anemia with excess blasts in transformation [RAEBt]) or refractory anemia with severe pancytopenia or high risk cytogenetics; blasts must be = 16 years old) >= 70 or Eastern Cooperative Oncology Group (ECOG) 0-1
- Lansky ( = 60
- Adults: calculated creatinine clearance must be > 60 mL and serum creatinine = 60 mL/min
- Total serum bilirubin must be 60% normal
- For pediatric patients unable to perform pulmonary function tests, oxygen (O2) saturation > 92% on room air
- May not be on supplemental oxygen
- Left ventricular ejection fraction > 45% OR
- Shortening fraction > 26%
- Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria
- Uncontrolled viral or bacterial infection at the time of study enrollment
- Active or recent (prior 6 month) invasive fungal infection without infectious disease (ID) consult and approval
- History of human immunodeficiency virus (HIV) infection
- Pregnant or breastfeeding
- Prior myeloablative transplant containing full dose TBI (greater than 8 Gy)
- Central nervous system (CNS) leukemic involvement not clearing with intrathecal chemotherapy and/or cranial radiation prior to initiation of conditioning; diagnostic lumbar puncture is to be performed per protocol
- Patients >= 45 years: comorbidity score of 5 or higher
Data sourced from ClinicalTrials.gov (NCT01690520). 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.