NCT03301597

Brief Summary

Phase 2 open-label, multi-center, randomized, controlled, dose-finding study of safety and efficacy of NLA101 to reduce the rate of infections associated with CIN in adult subjects with AML.

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
146

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Jan 2018

Shorter than P25 for phase_2

Geographic Reach
3 countries

36 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 27, 2017

Completed
7 days until next milestone

First Posted

Study publicly available on registry

October 4, 2017

Completed
4 months until next milestone

Study Start

First participant enrolled

January 24, 2018

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 18, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 18, 2019

Completed
Last Updated

March 30, 2021

Status Verified

March 1, 2021

Enrollment Period

1.1 years

First QC Date

September 27, 2017

Last Update Submit

March 25, 2021

Conditions

Keywords

Chemotherapy-induced NeutropeniaBacterial InfectionsFungal Infections

Outcome Measures

Primary Outcomes (1)

  • Recurrent Event Rate of Grade 3 or Higher Bacterial or Fungal Infection

    From randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, whichever is later

Secondary Outcomes (5)

  • Event rate of grade 3 or higher documented bacterial and fungal infections per cycle of chemotherapy

    From randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, whichever is later

  • Incidence and duration of filgrastim (or biosimilar) administration

    From randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, whichever is later

  • Overall Response Rate

    From randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, whichever is later

  • Incidence and duration of complications due to infections

    From randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, whichever is later

  • Incidence and duration of febrile neutropenia

    From randomization through follow-up of 84 days post randomization, 30 days post last infusion of NLA101, or 30 days post last infusion of chemotherapy for Control Arm, whichever is later

Study Arms (4)

Control Arm

OTHER

The Control Arm will receive standard of care (SOC) chemotherapy without the infusion of NLA101. SOC chemotherapy will be determined by local PI and must be a standard regimen for untreated de novo or secondary AML that will result in moderate to severe myelosuppression and will be given with curative intent.

Drug: Standard of Care (SOC) chemotherapy

Low Dose Arm

EXPERIMENTAL

The Low Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of low-dose NLA101.

Biological: NLA101Drug: Standard of Care (SOC) chemotherapy

Medium Dose Arm

EXPERIMENTAL

The Medium Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of medium-dose NLA101.

Biological: NLA101Drug: Standard of Care (SOC) chemotherapy

High Dose Arm

EXPERIMENTAL

The High Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of high-dose NLA101.

Biological: NLA101Drug: Standard of Care (SOC) chemotherapy

Interventions

NLA101BIOLOGICAL

NLA101 is a universal donor "off-the-shelf" ex-vivo expanded hematopoietic stem and progenitor cell (HSPC) product that is cryopreserved and ready for immediate use.

Also known as: Dilanubicel
High Dose ArmLow Dose ArmMedium Dose Arm

The SOC chemotherapy regimen for each patient will be determined by local PI. Regimen must be a standard AML regimen that will result in moderate to severe myelosuppression and have curative intent.

Control ArmHigh Dose ArmLow Dose ArmMedium Dose Arm

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age ≥ 18 (or legal age of majority for sites outside US).
  • Untreated de novo or secondary acute myeloid leukemia (AML), including AML that has progressed from myelodysplastic syndrome (MDS), and histologically documented diagnosis
  • Eligible for at least 2 cycles of standard of care AML chemotherapy that will result in moderate to severe myelosuppression and have curative intent
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of 0, 1, or 2 or Karnofsky Status of 50 to 100.
  • Adequate cardiac, renal, and hepatic functions.

You may not qualify if:

  • Extramedullary disease in the absence of bone marrow or blood involvement
  • Acute promyelocytic leukemia (APL) with PML-RARA
  • Prior AML therapy, with the exception of intrathecal chemotherapy or emergent radiation for myeloid sarcoma.
  • Concurrent malignancy requiring active treatment with chemotherapy, immunotherapy, or radiation
  • Prior allotransplant, including allogeneic hematopoietic cell transplant or solid organ allogeneic transplant
  • Known hypersensitivity or history of hypersensitivity to dimethylsulfoxide (DMSO)
  • Active/chronic human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV) infection

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (36)

UC San Diego Moores Cancer Center

La Jolla, California, 92093, United States

Location

USC Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

Winship Cancer Institute, Emory University

Atlanta, Georgia, 30322, United States

Location

University of Chicago Medical Center

Chicago, Illinois, 60637, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Norton Cancer Institute, St. Matthews Campus

Louisville, Kentucky, 40207, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of Nebraska Medical Center - Fred & Pamela Buffett Cancer Center

Omaha, Nebraska, 68198, United States

Location

Westchester Medical Center

Hawthorne, New York, 10532, United States

Location

Weill Cornell Medical College - NewYork-Presbyterian Hospital

New York, New York, 10021, United States

Location

Icahn School of Medicine at Mount Sinai and Mount Sinai Health System

New York, New York, 10029, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Stony Brook University

Stony Brook, New York, 11794, United States

Location

Duke University Heath System, Duke Cancer Center

Durham, North Carolina, 27710, United States

Location

Wake Forest Baptist Health

Winston-Salem, North Carolina, 27157, United States

Location

Geisinger Medical Center

Danville, Pennsylvania, 17822, United States

Location

West Penn Hospital

Pittsburgh, Pennsylvania, 15224, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Swedish Cancer Institute

Seattle, Washington, 98104, United States

Location

Seattle Cancer Care Alliance

Seattle, Washington, 98109, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

Froedtert Hospital and The Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

St. Vincent's Hospital Sydney

Darlinghurst, New South Wales, 2010, Australia

Location

St. George Hospital

Kogarah, New South Wales, 2217, Australia

Location

Calvary Mater Newcastle

Waratah, New South Wales, 2298, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

Austin Health

Heidelberg, Victoria, 3084, Australia

Location

Epworth HealthCare

Richmond, Victoria, 3121, Australia

Location

Royal Perth Hospital

Perth, Western Australia, 6000, Australia

Location

Gachon University Gil Medical Center

Incheon, 21565, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

The Catholic University of Korea's Seoul St. Mary's Hospital

Seoul, 06591, South Korea

Location

MeSH Terms

Conditions

Leukemia, Myeloid, AcuteBacterial InfectionsMycoses

Interventions

Standard of CareDrug Therapy

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBacterial Infections and MycosesInfections

Intervention Hierarchy (Ancestors)

Quality Indicators, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and EvaluationTherapeutics

Study Officials

  • Martin S Tallman, MD

    Memorial Sloan Kettering Cancer Center

    STUDY CHAIR
  • Naval G Daver, MD

    M.D. Anderson Cancer Center

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Masking Details
This is an open-label study.
Purpose
PREVENTION
Intervention Model
PARALLEL
Model Details: Subjects will be randomized to 1 of 3 Investigational Arms or a Control Arm in a 1:1:1:1 ratio.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 27, 2017

First Posted

October 4, 2017

Study Start

January 24, 2018

Primary Completion

March 18, 2019

Study Completion

March 18, 2019

Last Updated

March 30, 2021

Record last verified: 2021-03

Locations