Leukemia, Myeloid, AcutePhase 2LAUNCHNCT03301597
NLA101 in Adults Receiving High Dose Chemotherapy for AML
A Phase 2 study testing NLA101 in people with Leukemia, Myeloid, Acute, run by Nohla Therapeutics, Inc. at 36 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- NLA101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 146 participants
- Where
- 36 sitesin 3 countries
- Study dates
- 24 Jan 2018to 18 Mar 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
36 sites in 3 countries.
36 sites in 3 countries
United States25 sites
Winship Cancer Institute, Emory University
Atlanta, Georgia, 30322
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Chicago Medical Center
Chicago, Illinois, 60637
Geisinger Medical Center
Danville, Pennsylvania, 17822
Duke University Heath System, Duke Cancer Center
Durham, North Carolina, 27710
Westchester Medical Center
Hawthorne, New York, 10532
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Mayo Clinic Florida
Jacksonville, Florida, 32224
UC San Diego Moores Cancer Center
La Jolla, California, 92093
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Norton Cancer Institute, St. Matthews Campus
Louisville, Kentucky, 40207
University of Wisconsin
Madison, Wisconsin, 53792
Loyola University Medical Center
Maywood, Illinois, 60153
Froedtert Hospital and The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Weill Cornell Medical College - NewYork-Presbyterian Hospital
New York, New York, 10021
Icahn School of Medicine at Mount Sinai and Mount Sinai Health System
New York, New York, 10029
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
University of Nebraska Medical Center - Fred & Pamela Buffett Cancer Center
Omaha, Nebraska, 68198
West Penn Hospital
Pittsburgh, Pennsylvania, 15224
Mayo Clinic
Rochester, Minnesota, 55905
University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817
Swedish Cancer Institute
Seattle, Washington, 98104
Seattle Cancer Care Alliance
Seattle, Washington, 98109
Stony Brook University
Stony Brook, New York, 11794
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157
Australia7 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
St. Vincent's Hospital Sydney
Darlinghurst, New South Wales, 2010
Austin Health
Heidelberg, Victoria, 3084
St. George Hospital
Kogarah, New South Wales, 2217
Royal Perth Hospital
Perth, Western Australia, 6000
Epworth HealthCare
Richmond, Victoria, 3121
Calvary Mater Newcastle
Waratah, New South Wales, 2298
South Korea4 sites
Gachon University Gil Medical Center
Incheon, 21565
Seoul National University Hospital
Seoul, 03080
Samsung Medical Center
Seoul, 06351
The Catholic University of Korea's Seoul St. Mary's Hospital
Seoul, 06591
About this study
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
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.
Read the 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.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- NLA101, Standard of Care (SOC) chemotherapy
- Keywords
- Chemotherapy-induced Neutropenia, Bacterial Infections, Fungal Infections
- Medical subject headings
- Leukemia, Myeloid, Acute, Bacterial Infections, Mycoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NLA101Biological
Also called Dilanubicel
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.
Standard of Care (SOC) chemotherapyDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Control Arm | Other | 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. Receives: Drug: Standard of Care (SOC) chemotherapy |
| Low Dose Arm | Experimental | The Low Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of low-dose NLA101. Receives: Biological: NLA101; Drug: Standard of Care (SOC) chemotherapy |
| Medium Dose Arm | Experimental | The Medium Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of medium-dose NLA101. Receives: Biological: NLA101; Drug: Standard of Care (SOC) chemotherapy |
| High Dose Arm | Experimental | The High Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of high-dose NLA101. Receives: Biological: NLA101; Drug: Standard of Care (SOC) chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recurrent Event Rate of Grade 3 or Higher Bacterial or Fungal Infection
Measured: 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 outcomes (5)
Event rate of grade 3 or higher documented bacterial and fungal infections per cycle of chemotherapy
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2017
- Study start24 Jan 2018
- Primary completion18 Mar 2019
- Study completion18 Mar 2019
Why it stopped: Development plan under review.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Subjects will be randomized to 1 of 3 Investigational Arms or a Control Arm in a 1:1:1:1 ratio.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 146 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nohla Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Martin S Tallman, MD, Study chair, Memorial Sloan Kettering Cancer CenterNaval G Daver, MD, Study chair, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03301597
- Sponsor's protocol ID
- NLA-0101-CIN-01
- Acronym
- LAUNCH
- Status last verified
- Mar 2021
- First submitted
- 27 Sep 2017
- Last updated
- 30 Mar 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03301597
- Acronym / sponsor ID
- LAUNCH / NLA-0101-CIN-01
- Conditions
- Leukemia, Myeloid, Acute
- Keywords
- Chemotherapy-induced Neutropenia, Bacterial Infections, Fungal Infections
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Development plan under review.
- Enrolment
- 146 participants (actual)
- Lead sponsor
- Nohla Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Jan 2018 (actual)
- Primary completion
- 18 Mar 2019 (actual)
- Study completion
- 18 Mar 2019 (actual)
- First posted
- 4 Oct 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 4
- Model description
- Subjects will be randomized to 1 of 3 Investigational Arms or a Control Arm in a 1:1:1:1 ratio.
| Arm | Type | Receives |
|---|---|---|
| Control Arm | Other | Drug: Standard of Care (SOC) chemotherapyThe 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. |
| Low Dose Arm | Experimental | Biological: NLA101, Drug: Standard of Care (SOC) chemotherapyThe Low Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of low-dose NLA101. |
| Medium Dose Arm | Experimental | Biological: NLA101, Drug: Standard of Care (SOC) chemotherapyThe Medium Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of medium-dose NLA101. |
| High Dose Arm | Experimental | Biological: NLA101, Drug: Standard of Care (SOC) chemotherapyThe High Dose Arm will receive standard of care (SOC) chemotherapy with the infusion of high-dose NLA101. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NLA101Also: DilanubicelNLA101 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. | Biological | High Dose Arm, Low Dose Arm, Medium Dose Arm |
| Standard of Care (SOC) chemotherapyThe 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. | Drug | Control Arm, High Dose Arm, Low Dose Arm, Medium Dose Arm |
Primary outcome
Recurrent Event Rate of Grade 3 or Higher Bacterial or Fungal Infection
Time frame: 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
5 secondary outcomes
Event rate of grade 3 or higher documented bacterial and fungal infections per cycle of chemotherapy
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Results and safety
The study was stopped early (reason given: Development plan under review.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Martin S Tallman, MD study chair
Memorial Sloan Kettering Cancer Center
Naval G Daver, MD study chair
M.D. Anderson Cancer Center
36 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NLA101 in people with Leukemia, Myeloid, Acute, run by Nohla Therapeutics, Inc. at 36 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 36 sites in United States, Australia, South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Development plan under review.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recurrent Event Rate of Grade 3 or Higher Bacterial or Fungal Infection (time frame: 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2021.