NLA101 in Adults Receiving High Dose Chemotherapy for AML (LAUNCH)

A Phase 2 Open-Label, Multi-Center, Randomized, Controlled, Dose-Finding Study of NLA101 in Adults Receiving High Dose Chemotherapy for Acute Myeloid Leukemia

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.

Study Overview

• Status: Terminated
• Conditions: Leukemia, Myeloid, Acute
• Intervention / Treatment: Drug: Standard of Care (SOC) chemotherapy; Biological: NLA101

Detailed Description

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 chemotherapy induced neutropenia (CIN) in adult subjects with AML.

Eligible subjects with untreated de novo or secondary AML and per local institutional standards planned to receive at least two cycles of chemotherapy with curative intent will be enrolled into the study and randomized 1:1:1:1 to 1 of 3 Investigational Arms (Standard of Care [SOC] chemotherapy + low, medium, or high dose NLA101) or a Control Arm (SOC chemotherapy).

Subjects randomized to an Investigational Arm will be eligible to receive a single fixed assigned dose of NLA101 after the first cycle of chemotherapy, and up to 2 additional identical cell doses after subsequent chemotherapy cycles (one NLA101 infusion per cycle). Subjects randomized to the Control Arm will be followed for up to 3 cycles of chemotherapy.

All subjects will be followed for 84 days following randomization, or 30 days post final infusion of NLA101, or 30 days post the day after the last chemotherapy infusion for Control Arm, whichever is longer.

• Study Type: Interventional
• Enrollment (Actual): 146
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Darlinghurst, New South Wales, Australia, 2010
      • St. Vincent's Hospital Sydney
    • Kogarah, New South Wales, Australia, 2217
      • St. George Hospital
    • Waratah, New South Wales, Australia, 2298
      • Calvary Mater Newcastle
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
  • Victoria
    • Heidelberg, Victoria, Australia, 3084
      • Austin Health
    • Richmond, Victoria, Australia, 3121
      • Epworth Healthcare
  • Western Australia
    • Perth, Western Australia, Australia, 6000
      • Royal Perth Hospital
• Korea, Republic of
  • Incheon, Korea, Republic of, 21565
    • Gachon University Gil Medical Center
  • Seoul, Korea, Republic of, 03080
    • Seoul National University Hospital
  • Seoul, Korea, Republic of, 06351
    • Samsung Medical Center
  • Seoul, Korea, Republic of, 06591
    • The Catholic University of Korea's Seoul St. Mary's Hospital
• United States
  • California
    • La Jolla, California, United States, 92093
      • UC San Diego Moores Cancer Center
    • Los Angeles, California, United States, 90033
      • USC Norris Comprehensive Cancer Center
    • Sacramento, California, United States, 95817
      • University of California Davis Comprehensive Cancer Center
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic Florida
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Winship Cancer Institute, Emory University
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Medical Center
    • Maywood, Illinois, United States, 60153
      • Loyola University Medical Center
  • Kentucky
    • Louisville, Kentucky, United States, 40207
      • Norton Cancer Institute, St. Matthews Campus
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • Nebraska
    • Omaha, Nebraska, United States, 68198
      • University of Nebraska Medical Center - Fred & Pamela Buffett Cancer Center
  • New York
    • Hawthorne, New York, United States, 10532
      • Westchester Medical Center
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
    • New York, New York, United States, 10021
      • Weill Cornell Medical College - NewYork-Presbyterian Hospital
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai and Mount Sinai Health System
    • Stony Brook, New York, United States, 11794
      • Stony Brook University
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Heath System, Duke Cancer Center
    • Winston-Salem, North Carolina, United States, 27157
      • Wake Forest Baptist Health
  • Pennsylvania
    • Danville, Pennsylvania, United States, 17822
      • Geisinger Medical Center
    • Pittsburgh, Pennsylvania, United States, 15224
      • West Penn Hospital
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas Md Anderson Cancer Center
  • Washington
    • Seattle, Washington, United States, 98109
      • Seattle Cancer Care Alliance
    • Seattle, Washington, United States, 98104
      • Swedish Cancer Institute
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • University of Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Froedtert Hospital and the Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Criteria:

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Control Arm; 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; 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.
Experimental: Low Dose Arm; The Low Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of low-dose NLA101.	Drug: Standard of Care (SOC) chemotherapy; 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.; Biological: NLA101; 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.; Other Names: Dilanubicel
Experimental: Medium Dose Arm; The Medium Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of medium-dose NLA101.	Drug: Standard of Care (SOC) chemotherapy; 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.; Biological: NLA101; 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.; Other Names: Dilanubicel
Experimental: High Dose Arm; The High Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of high-dose NLA101.	Drug: Standard of Care (SOC) chemotherapy; 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.; Biological: NLA101; 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.; Other Names: Dilanubicel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Nohla Therapeutics, Inc.
• Investigators: Study Chair: Martin S Tallman, MD, Memorial Sloan Kettering Cancer Center; Study Chair: Naval G Daver, MD, M.D. Anderson Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): January 24, 2018
• Primary Completion (Actual): March 18, 2019
• Study Completion (Actual): March 18, 2019

Study Registration Dates

• First Submitted: September 27, 2017
• First Submitted That Met QC Criteria: October 3, 2017
• First Posted (Actual): October 4, 2017

Study Record Updates

• Last Update Posted (Actual): March 30, 2021
• Last Update Submitted That Met QC Criteria: March 25, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Keywords: Bacterial Infections; Fungal Infections; Chemotherapy-induced Neutropenia
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: NLA-0101-CIN-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No