NLA101 in Adults Receiving High Dose Chemotherapy for AML

Phase 2

Terminated

• Conditions: Leukemia, Myeloid, Acute
• Interventions: Biological: NLA101; Drug: Standard of Care (SOC) chemotherapy

• Registration Number: NCT03301597
• Lead Sponsor: Nohla Therapeutics, Inc.

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.

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 Timeline

• Study First Submitted: 2017-09-27
• Study First Submitted QC: 2017-10-03
• Study First Posted: 2017-10-04
• Study Start: 2018-01-24
• Primary Completion: 2019-03-18
• Study Completion: 2019-03-18
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-25
• Last Update Posted: 2021-03-30

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 146

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Low Dose Arm	Standard of Care (SOC) chemotherapy	The Low Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of low-dose NLA101.
Control Arm	Standard of Care (SOC) chemotherapy	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.
High Dose Arm	NLA101	The High Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of high-dose NLA101.
Medium Dose Arm	NLA101	The Medium Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of medium-dose NLA101.
Medium Dose Arm	Standard of Care (SOC) chemotherapy	The Medium Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of medium-dose NLA101.
High Dose Arm	Standard of Care (SOC) chemotherapy	The High Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of high-dose NLA101.
Low Dose Arm	NLA101	The Low Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of low-dose NLA101.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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 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
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

Trial Locations

Locations (36)

UC San Diego Moores Cancer Center; 🇺🇸 La Jolla, California, United States

USC Norris Comprehensive Cancer Center; 🇺🇸 Los Angeles, California, United States

University of California Davis Comprehensive Cancer Center; 🇺🇸 Sacramento, California, United States

Mayo Clinic Florida; 🇺🇸 Jacksonville, Florida, United States

Winship Cancer Institute, Emory University; 🇺🇸 Atlanta, Georgia, United States

University of Chicago Medical Center; 🇺🇸 Chicago, Illinois, United States

Loyola University Medical Center; 🇺🇸 Maywood, Illinois, United States

Norton Cancer Institute, St. Matthews Campus; 🇺🇸 Louisville, Kentucky, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Scroll for more (26 remaining)