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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03301597




Registration number
NCT03301597
Ethics application status
Date submitted
27/09/2017
Date registered
4/10/2017
Date last updated
30/03/2021

Titles & IDs
Public title
NLA101 in Adults Receiving High Dose Chemotherapy for AML
Scientific 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
Secondary ID [1] 0 0
NLA-0101-CIN-01
Universal Trial Number (UTN)
Trial acronym
LAUNCH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Leukemia, Myeloid, Acute 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - NLA101
Treatment: Drugs - Standard of Care (SOC) chemotherapy

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.

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

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

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


Other interventions: 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.

Treatment: Drugs: 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.
Intervention code [1] 0 0
Other interventions
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Recurrent Event Rate of Grade 3 or Higher Bacterial or Fungal Infection
Timepoint [1] 0 0
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 [1] 0 0
Event rate of grade 3 or higher documented bacterial and fungal infections per cycle of chemotherapy
Timepoint [1] 0 0
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 [2] 0 0
Incidence and duration of filgrastim (or biosimilar) administration
Timepoint [2] 0 0
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 [3] 0 0
Overall Response Rate
Timepoint [3] 0 0
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 [4] 0 0
Incidence and duration of complications due to infections
Timepoint [4] 0 0
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 [5] 0 0
Incidence and duration of febrile neutropenia
Timepoint [5] 0 0
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
Key inclusion criteria
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/01/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/03/2019
Sample size
Target
Accrual to date
Final
146
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
Recruitment hospital [1] 0 0
St. Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [2] 0 0
St. George Hospital - Kogarah
Recruitment hospital [3] 0 0
Calvary Mater Newcastle - Waratah
Recruitment hospital [4] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [5] 0 0
Austin Health - Heidelberg
Recruitment hospital [6] 0 0
Epworth HealthCare - Richmond
Recruitment hospital [7] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
2217 - Kogarah
Recruitment postcode(s) [3] 0 0
2298 - Waratah
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
3084 - Heidelberg
Recruitment postcode(s) [6] 0 0
3121 - Richmond
Recruitment postcode(s) [7] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Kentucky
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Nebraska
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
United States of America
State/province [14] 0 0
Wisconsin
Country [15] 0 0
Korea, Republic of
State/province [15] 0 0
Incheon
Country [16] 0 0
Korea, Republic of
State/province [16] 0 0
Seoul

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Nohla Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/ct2/show/NCT03301597
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Martin S Tallman, MD
Address 0 0
Memorial Sloan Kettering Cancer Center
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03301597