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

NCT02765997 | Withdrawn Phase 2 FDA Drug

• 2 other identifiers: 2016LS006MT2016-01
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Acute Myeloid Leukemia; Acute Lymphocytic Leukemia; Chronic Myelogenous Leukemia; Myelodysplasia

Keywords

AML; ALL; MDS

Outcome Measures

Primary Outcomes (1)

• Neutrophil Recovery

  Percentage of patients with neutrophil recovery

  Day 14 after transplantation

Secondary Outcomes (3)

• Secondary Graft Failure

  Day 100 after transplantation

• Platelet Recovery

  Day 100 after transplantation

• Transplant-Related Mortality

  6 months after transplantation

Study Arms (2)

Unmanipulated UCB

ACTIVE COMPARATOR

Subjects will receive unmanipulated umbilical cord blood transplantation after a myeloablative CY/FLU/TBI conditioning.

Biological: Unmanipulated UCB

StemRegenin-1 UCB

EXPERIMENTAL

Subjects will receive StemRegenin-1 (SR-1) cultured umbilical cord blood transplantation after a myeloablative CY/FLU/TBI conditioning.

Biological: SR-1 UCB

Interventions

Unmanipulated UCB BIOLOGICAL

Unmanipulated UCB infusion given on Day 0. All patients will receive the same conditioning and immunoprophylaxis for the prevention of acute and chronic GVHD, previously demonstrated to offer the best outcomes in recipients of partially HLA matched UCB. Standard supportive care, including the use of Neupogen \[G-CSF\] and prophylactic anti-bacterial, protozoal, viral and fungal agents, will also be prescribed. Supportive care will be modified throughout the transplant course at the treating physician's judgement.

Also known as: Unmanipulated Umbilical Cord Blood

Unmanipulated UCB

SR-1 UCB BIOLOGICAL

SR-1 UCB infusion given on Day 0. All patients will receive the same conditioning and immunoprophylaxis for the prevention of acute and chronic GVHD, previously demonstrated to offer the best outcomes in recipients of partially HLA matched UCB. Standard supportive care, including the use of Neupogen \[G-CSF\] and prophylactic anti-bacterial, protozoal, viral and fungal agents, will also be prescribed. Supportive care will be modified throughout the transplant course at the treating physician's judgement.

Also known as: StemRegenin-1 cultured umbilical cord blood

StemRegenin-1 UCB

Eligibility Criteria

• Age: 2 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• Ph+ ALL, or
• MLL rearrangement with slow early response at Day 14, or
• Hypodiploidy (\< 44 chromosomes or DNA index \< 0.81), or
• End of induction M3 bone marrow, or
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

University of Minnesota Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Anemia, Refractory, with Excess of Blasts

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Anemia, Refractory; Anemia; Myelodysplastic Syndromes

Study Officials

• John Wagner, MD

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2016
• First Posted: May 9, 2016
• Study Start: April 1, 2017
• Primary Completion: June 1, 2020
• Study Completion: June 1, 2022
• Last Updated: December 5, 2017

Record last verified: 2017-12

Locations

US (1)