LAUNCH: NLA101 in Adults Receiving High Dose Chemotherapy for AML

Sponsor

Nohla Therapeutics, Inc. (Industry)

Overall Status

Terminated

CT.gov ID

NCT03301597

Collaborator

(none)

146

Enrollment

Locations

Arms

13.7

Actual Duration (Months)

4.1

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Leukemia, Myeloid, Acute	Biological: NLA101 Drug: Standard of Care (SOC) chemotherapy	Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

146 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Subjects will be randomized to 1 of 3 Investigational Arms or a Control Arm in a 1:1:1:1 ratio.Subjects will be randomized to 1 of 3 Investigational Arms or a Control Arm in a 1:1:1:1 ratio.

Masking:

Single (Outcomes Assessor)

Masking Description:

This is an open-label study.

Primary Purpose:

Prevention

Official Title:

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

Actual Study Start Date :

Jan 24, 2018

Actual Primary Completion Date :

Mar 18, 2019

Actual Study Completion Date :

Mar 18, 2019

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

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

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UC San Diego Moores Cancer Center	La Jolla	California	United States	92093
2	USC Norris Comprehensive Cancer Center	Los Angeles	California	United States	90033
3	University of California Davis Comprehensive Cancer Center	Sacramento	California	United States	95817
4	Mayo Clinic Florida	Jacksonville	Florida	United States	32224
5	Winship Cancer Institute, Emory University	Atlanta	Georgia	United States	30322
6	University of Chicago Medical Center	Chicago	Illinois	United States	60637
7	Loyola University Medical Center	Maywood	Illinois	United States	60153
8	Norton Cancer Institute, St. Matthews Campus	Louisville	Kentucky	United States	40207
9	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
10	Mayo Clinic	Rochester	Minnesota	United States	55905
11	University of Nebraska Medical Center - Fred & Pamela Buffett Cancer Center	Omaha	Nebraska	United States	68198
12	Westchester Medical Center	Hawthorne	New York	United States	10532
13	Weill Cornell Medical College - NewYork-Presbyterian Hospital	New York	New York	United States	10021
14	Icahn School of Medicine at Mount Sinai and Mount Sinai Health System	New York	New York	United States	10029
15	Memorial Sloan Kettering Cancer Center	New York	New York	United States	10065
16	Stony Brook University	Stony Brook	New York	United States	11794
17	Duke University Heath System, Duke Cancer Center	Durham	North Carolina	United States	27710
18	Wake Forest Baptist Health	Winston-Salem	North Carolina	United States	27157
19	Geisinger Medical Center	Danville	Pennsylvania	United States	17822
20	West Penn Hospital	Pittsburgh	Pennsylvania	United States	15224
21	The University of Texas MD Anderson Cancer Center	Houston	Texas	United States	77030
22	Swedish Cancer Institute	Seattle	Washington	United States	98104
23	Seattle Cancer Care Alliance	Seattle	Washington	United States	98109
24	University of Wisconsin	Madison	Wisconsin	United States	53792
25	Froedtert Hospital and The Medical College of Wisconsin	Milwaukee	Wisconsin	United States	53226
26	St. Vincent's Hospital Sydney	Darlinghurst	New South Wales	Australia	2010
27	St. George Hospital	Kogarah	New South Wales	Australia	2217
28	Calvary Mater Newcastle	Waratah	New South Wales	Australia	2298
29	Royal Adelaide Hospital	Adelaide	South Australia	Australia	5000
30	Austin Health	Heidelberg	Victoria	Australia	3084
31	Epworth HealthCare	Richmond	Victoria	Australia	3121
32	Royal Perth Hospital	Perth	Western Australia	Australia	6000
33	Gachon University Gil Medical Center	Incheon		Korea, Republic of	21565
34	Seoul National University Hospital	Seoul		Korea, Republic of	03080
35	Samsung Medical Center	Seoul		Korea, Republic of	06351
36	The Catholic University of Korea's Seoul St. Mary's Hospital	Seoul		Korea, Republic of	06591