StemRegenin-1 Expanded vs Unexpanded UCB for High Risk Heme Malignancies

• Status: Not yet recruiting
• Trial end date: 0000-00-00
• Target enrollment: 30
• Participant gender: Both

Summary

This is an open label, interventional, randomized phase II trial comparing StemRegenin-1 (SR-1) cultured umbilical cord blood (experimental arm) to unmanipulated umbilical cord blood (standard of care arm) transplantation after a myeloablative CY/FLU/TBI conditioning. A 2:1 randomization will be employed with a higher chance of being assigned to the experimental arm.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Cyclophosphamide; Fludarabine; Lenograstim; Mycophenolate mofetil
• Last Updated: 2016-11-07

Criteria

Inclusion Criteria:

• Must have a partially HLA matched UCB unit with a pre-cryopreserved TNC dose >2.5 x 107 per kilogram recipient weight. HLA matching is initially based on 4 of 6 HLA-A and B (at low or intermediate resolution by molecular typing) and DRB1 (at high resolution by molecular typing).

• Eligible Diseases

• Acute myelogenous leukemia (AML) at the following stages:

• Intermediate to high risk leukemia in first complete remission (CR1) based on institutional criteria.

• Any second or subsequent CR.

• Secondary AML with prior malignancy that has been in remission for at least 12 months.

• Acute lymphocytic leukemia (ALL) at the following stages:

• High risk first remission.

1. Ph+ ALL, or

2. MLL rearrangement with slow early response at Day 14, or

3. Hypodiploidy (< 44 chromosomes or DNA index < 0.81), or

4. End of induction M3 bone marrow, or

5. End of induction M2 with M2-3 at Day 42.

• High risk second CR based on institutional criteria (eg, for children, bone marrow relapse <36 months from induction or T-lineage bone marrow relapse or very early isolated central nervous system (CNS) relapse <6 months from diagnosis, or slow re-induction (stage M2-3 at day 28 after induction) regardless of length remission.

• Any third or subsequent CR.

• Biphenotypic/undifferentiated leukemia in CR

• Chronic myelogenous leukemia (CML) excluding refractory blast crisis

• Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia

• Other Inclusion Criteria

• Karnofsky score >70% (16 years and older) or a Lansky play score >70 (children <16 years) - appendix II

• Adequate organ function defined as:

• 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.

• Hepatic: Bilirubin ≤2.5 x mg/dL; AST, ALT, alkaline phosphatase <5 x upper limit of normal,

• Pulmonary function: DLCO, FEV1, FEC (diffusion capacity) >50% of predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then normal O2 saturation on room air.

• Cardiac: Left ventricular ejection fraction at rest must be >45%

• Available 'back-up' HSPC graft (e.g, second partially HLA matched UCB unit, haploidentical related donor).

• Voluntary written consent signed (adult or parental) before performance of any study-related procedure not part of normal medical care

Exclusion Criteria:

• Pregnant or breast feeding. The agents used in this study may be teratogenic to a fetus and there is no information on the excretion of agents into breast milk. Females of childbearing potential must have a blood test or urine study within 14 days prior to study enrollment to rule out pregnancy.

• Evidence of human immunodeficiency virus (HIV) infection or known HIV positive serology.

• Active bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms).

• Prior autologous or allogeneic transplant within past 12 months.

• Other active malignancy.

• Inability to receive TBI 1320 cGy (e.g., extensive prior therapy including >12 months alkylator therapy or >6 months alkylator therapy with extensive radiation. Or prior Y-90 ibritumomab (Zevalin) or I-131 tostumomab (Bexxar), as part of their salvage therapy.