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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03301597
Ethics application status
Date submitted
27/09/2017
Date registered
3/10/2017
Date last updated
21/11/2018

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 grade 3 or higher fever or hypothermia
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
Gender
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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/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
Richie Phan, CTM
Address 0 0
Country 0 0
Phone 0 0
206-519-5318
Fax 0 0
Email 0 0
RichieP@NohlaTherapeutics.com
Contact person for scientific queries

No data has been provided for results reporting
Summary results
Not applicable