Phase 2
N=15
UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
Acute Leukemia · Acute Myeloid Leukemia · Acute Lymphoblastic Leukemia/Lymphoma · Burkitt's Lymphoma · Natural Killer Cell Malignancies
Bottom Line
View on ClinicalTrials.gov: NCT02722668 ↗Enrolled (actual)
15
Serious AEs
0.0%
Results posted
Jul 2025
Primary outcome: Primary: Probability of Acute Graft Versus Host Disease (GVHD) — 0; 30 Percent of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Fludarabine (Drug); Cyclophosphamide (Drug); MMF (Drug); Sirolimus (Drug); TBI (Radiation); Umbilical cord blood cell infusion (Biological); ATG (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Probability of Acute Graft Versus Host Disease (GVHD) |
0; 30 | — |
| SECONDARY Incidence of Acute GVHD |
0; 10 | — |
| SECONDARY Transplant Related Mortality |
20; 20 | — |
| SECONDARY Chimerism |
20; 100 | — |
| SECONDARY Chimerism |
20; 100 | — |
| SECONDARY Chimerism |
20; 100 | — |
| SECONDARY Chimerism |
20; 100 | — |
| SECONDARY Neutrophil Engraftment |
80; 90 | — |
Summary
This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment. Single or double unit selected according to current University of Minnesota umbilical cord blood graft selection algorithm.
Eligibility Criteria
Inclusion Criteria
- Age, Performance Status, and Graft Criteria
- 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk. Favorable risk AML is defined as having one of the following:
- t(8,21) without cKIT mutation
- inv(16) or t(16;16) without cKIT mutation
- Normal karyotype with mutated NPM1 and wild type FLT-ITD
- Normal karyotype with double mutated CEBPA
- Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
- Acute lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL. High risk ALL is defined as having one of the following:
- Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
- 30 years of age or older at diagnosis
- White blood cell counts of greater than 30,000/mcL (B-ALL) or greater than 100,000/mcL (T-ALL) at diagnosis
- CNS leukemia involvement during the course of disease
- Slow cytologic response (>10% lymphoblasts in bone marrow on Day 14 of induction therapy)
- Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy
- Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR
- Chronic myelogenous leukemia in chronic or accelerated phase, or CML blast crisis in morphological remission ( 12 months, are eligible after at least two prior therapies. Patients with bulky disease should be considered for debulking chemotherapy before transplant. Patients with refractory disease are eligible, unless bulky disease and an estimated tumor doubling time of less than one month.
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy if chemotherapy sensitive.
- Relapsed Multiple Myeloma that is chemotherapy sensitive and has failed or ineligible for an autologous transplant.
- Plasma Cell Leukemia after initial therapy if achieved at least in partial remission; or relapsed and achieved subsequent remission (CR/PR)
- Acquired Bone marrow failure syndromes, except for Fanconi anemia
- Myeloproliferative Neoplasms/Myelofibrosis
- Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
- Additional Criteria for Bulky Disease (lymphomas)
- If stable disease is best response, the largest residual nodal mass must 40%. For children that are not able to cooperate with MUGA and echocardiography, such should be clearly stated in the physician's note.
- Pulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.
- Liver: Transaminases ≤ 5 x upper limit of normal (ULN) and total bilirubin ≤ 2.5 mg/dL except for patients with Gilbert's syndrome or hemolysis
- Renal: Creatinine ≤ 2.0 mg/dl (adults) and creatinine clearance ≥ 40 mL/min (pediatrics). Adults with a creatinine > 1.2 mg/dl or a history of renal dysfunction must have estimated creatinine clearance ≥ 40 ml/min/1.73m^2. Adequate performance status is defined as Karnofsky score ≥ 70% (≥ 16 years of age) or Lansky score ≥ 50 (pediatrics)
- Sexually active females of childbearing potential and males with partners of child-bearing potential must agree to use adequate birth control during study treatment.
- Voluntary wri
Data sourced from ClinicalTrials.gov (NCT02722668). 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.