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Acute Myeloid LeukemiaPhase 1/2ENTO in AMLNCT02343939

Entospletinib Monotherapy and in Combination With Chemotherapy in Adults With Acute Myeloid Leukemia (AML)

A Phase 1/2 study testing Entospletinib, Daunorubicin and Cytarabine in people with Acute Myeloid Leukemia, run by Gilead Sciences at 17 sites in 3 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study will evaluate the efficacy, safety, and tolerability of entospletinib when administered as monotherapy or in combination with chemotherapy in adults with acute myeloid leukemia (AML). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Entospletinib, Daunorubicin and Cytarabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
148 participants
Where
17 sitesin 3 countries
Study dates
1 Jul 2015to 21 Feb 2019
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Adults with AML in need of treatment
  • Group A : Individuals ≥ 18 years of age with previously untreated AML by World Health Organization (WHO) criteria who are able and should receive up to 2 cycles of induction chemotherapy with 7+3 as determined by the treating physician
  • Group B: Individuals > 70 years of age with previously untreated AML by WHO criteria; or individuals ≤ 70 years of age with previously untreated AML who refuse or are unable to receive chemotherapy with 7+3 as determined by the treating physician
  • Group C: Individuals ≥ 18 years of age with relapsed/refractory AML by WHO criteria; or with relapsed/refractory AML with mixed-lineage leukemia (MLL); or with previously untreated AML by WHO criteria and who would have met disease eligibility criteria for Group A or B but refuse or are unable to receive chemotherapy and hypomethylating agent as determined by the treating physician

Who cannot join

  • Known active central nervous system or leptomeningeal lymphoma
  • Subjects with acute promyelocytic leukemia (M3)
  • Treatment with proton pump inhibitors (PPIs) within 7 days prior to enrollment.
  • NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Trial locations and contacts

17 sites in 3 countries.

17 sites in 3 countries

United States14 sites
  • Dana Farber Cancer Institute

    Boston, Massachusetts

  • University of Chicago

    Chicago, Illinois

  • University Hospitals Case Medical Center

    Cleveland, Ohio

  • Ohio State University

    Columbus, Ohio

  • Henry Ford Health System

    Detroit, Michigan

  • Karmanos Cancer Institute

    Detroit, Michigan

  • Duke Cancer Center

    Durham, North Carolina

  • University of Kansas Medical Center Research Institute, Inc

    Fairway, Kansas

  • Saint Francis Cancer Center

    Greenville, South Carolina

  • Indiana University

    Indianapolis, Indiana

  • UCLA

    Los Angeles, California

  • Loyola University Medical Center

    Maywood, Illinois

  • Weill Cornell Medical College - New York - Presbyterian Hospital

    New York, New York

  • Oregon Health & Science University

    Portland, Oregon, 97239

Other trials in United States

Canada2 sites
  • Jewish General Hospital

    Montreal, Quebec

  • Princess Margaret

    Toronto, Ontario

Other trials in Canada

Germany1 site
  • Universitätsklinikum Frankfurt Medizinische Klinik II

    Frankfurt, 60590

Other trials in Germany

About this study

Official title: A Phase 1b/2 Study of Entospletinib (GS-9973) Monotherapy and in Combination With Chemotherapy in Patients With Acute Myeloid Leukemia (AML)

This study will evaluate the efficacy, safety, and tolerability of entospletinib when administered as monotherapy or in combination with chemotherapy in adults with acute myeloid leukemia (AML).

Being tested
Entospletinib, Daunorubicin, Cytarabine, Decitabine, Azacitidine
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EntospletinibDrug

    Also called GS-9973, ENTO

    Tablet(s) administered orally every 12 hours

  • DaunorubicinDrug

    60 mg/m^2 administered intravenously daily on Days 1 to 3 for up to two 14-day induction cycles

  • CytarabineDrug

    100 mg/m^2 administered intravenously daily on Days 1 to 7 for up to two 14-day cycles

  • DecitabineDrug

    20 mg/m^2 administered intravenously

  • AzacitidineDrug

    75 mg/m^2 administered intravenously or subcutaneously

Study groups in NCT02343939
Study groupRoleWhat they receive
Entospletinib + daunorubicin + cytarabine (Group A)ExperimentalDose Escalation: Entospletinib up to 400 mg for 14 days and then entospletinib up to 400 mg in combination with daunorubicin and cytarabine for up to two 14-day cycles. Dose Expansion: Entospletinib 400 mg for 14 days and then entospletinib 400 mg in combination with daunorubicin and cytarabine for up to two 14-day cycles. Some participants will have the option to receive post-induction therapy with entospletinib 400 mg in combination with cytarabine/cytosine arabinoside (ARA-C). Participants may receive maintenance therapy with 28-day cycles of entospletinib 400 mg for up to twelve 28-day cycles, if the participant is not eligible for stem cell transplant.
Receives: Drug: Entospletinib; Drug: Daunorubicin; Drug: Cytarabine
Entospletinib + decitabine (Group B)ExperimentalDose Escalation: Entospletinib 400 mg for 14 days and then entospletinib 400 mg in combination with decitabine for 10 days beginning on Day 1 of every 28-day cycle (at least 2 cycles of induction therapy but no more than 4 cycles). Participants who are intolerant of decitabine may switch to entospletinib monotherapy maintenance at any time after completing the first 2 cycles. Dose Expansion: Entospletinib 400 mg for 14 days for the safety run-in participants or Entospletinib 400 mg for 5 days for the randomization participants. Then entospletinib 400 mg in combination with decitabine or azacitidine (at least 2 cycles of induction therapy but no more than 4 cycles). Some participants will have the option to receive maintenance therapy with entospletinib in combination with decitabine or azacitidine. Participants who are intolerant of decitabine or azacitidine may switch to entospletinib monotherapy maintenance at any time after completing the first 2 cycles.
Receives: Drug: Entospletinib; Drug: Decitabine; Drug: Azacitidine
Entospletinib (Group C)ExperimentalDose Escalation: Entospletinib up to 800 mg for 28-day cycles until the participant meets criteria for study treatment discontinuation per the study protocol. Dose Expansion: Entospletinib 400 mg for 28-day cycles until the participant meets criteria for study treatment discontinuation per the protocol.
Receives: Drug: Entospletinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs)

    DLTs refer to toxicities experienced during the first 28 days of study treatment that have been judged to be clinically significant and related to study treatment. DLT assessment was applicable only for Phase 1b and Phase 2 safety run-in participants.

    Measured: Group A: Cycle 0 Day 1 to Cycle 2 Day 28; Group B: Cycle 0 Day 1 to Cycle 1 Day 28; Group C: Cycle 1 Day 1 to Cycle 1 Day 28 (Cycle length: for Cycle 0 = 14 days, for all other cycles = 28 days)

  2. Percentage of Participants With Morphologic Complete Remission (CR) at the End of Induction

    Clinical response was assessed according to the International Working Group criteria (Cheson 2003). Morphologic CR included CR and cytogenetic CR (CRc). CR required all of the following: < 5% blasts in bone marrow aspirate; Neutrophils ≥ 1,000/microliter (mcL); Platelets ≥ 100,000/mcL; No extramedullary disease; No blasts with Auer rods detected; and Independent of transfusions. CRc, in addition to CR criteria, required reversion to a normal karyotype with an abnormal karyotype at the time of diagnosis.

    Measured: At the end of induction (Group A: up to end of Cycle 2; Group B: up to end of Cycle 4) (cycle length = up to 28 days); Group C: From Day 1 until meeting the criteria for study treatment discontinuation (up to approximately 3 years)

  3. Percentage of Participants With Composite Complete Remission at the End of Induction

    Clinical response was assessed according to the International Working Group criteria (Cheson 2003). Composite complete remission included CR, CRc, and morphologic complete remission with incomplete blood count recovery (CRi). CR required all of the following: < 5% blasts in bone marrow aspirate; Neutrophils ≥ 1,000/mcL; Platelets ≥ 100,000/mcL; No extramedullary disease; No blasts with Auer rods detected; and Independent of transfusions. CRc, in addition to CR criteria, required reversion to a normal karyotype with an abnormal karyotype at the time of diagnosis. CRi required all of the CR criteria except the criterion of neutrophils and platelets.

    Measured: At the end of induction (Group A: up to end of Cycle 2; Group B: up to end of Cycle 4) (cycle length = up to 28 days); Group C: From Day 1 until meeting the criteria for study treatment discontinuation (up to approximately 3 years)

  4. Percentage of Participants With Overall Response at the End of Induction

    Clinical response was assessed according to the International Working Group criteria (Cheson 2003). Overall response included CR, CRc, CRi, and partial remission (PR). CR required all of the following: < 5% blasts in bone marrow aspirate; Neutrophils ≥ 1,000/mcL; Platelets ≥ 100,000/mcL; No extramedullary disease; No blasts with Auer rods detected; and Independent of transfusions. CRc, in addition to CR criteria, required reversion to a normal karyotype with an abnormal karyotype at the time of diagnosis. CRi required all of the CR criteria except the criterion of neutrophils and platelets. PR required all of the following: ≥ 50% decrease in blasts in bone marrow aspirate to a range of 5% to 25%; Neutrophils ≥ 1,000/mcL; Platelets ≥ 100,000/mcL; Independent of transfusions; and A value of ≤ 5% blasts was also considered a PR if Auer rods were detected.

    Measured: At the end of induction (Group A: up to end of Cycle 2; Group B: up to end of Cycle 4) (cycle length = up to 28 days); Group C: From Day 1 until meeting the criteria for study treatment discontinuation (up to approximately 3 years)

Secondary outcomes (5)
  1. Duration of Exposure of Entospletinib

    Measured: First dose date up to approximately 3 years

  2. Event Free Survival (EFS)

    EFS was defined as the time interval from the start of the study therapy until the date of treatment failure, acute myeloid leukemia (AML) relapse, or death from any cause, whichever occurred first. Participants who received other anti-cancer therapy (prior to the event if any) were censored. Median EFS was analyzed using Kaplan-Meier (KM) method.

    Measured: First dose date up to approximately 38 months

  3. Overall Survival (OS)

    OS was defined as the time interval from the start of the study therapy to death from any cause. Median OS was analyzed using KM method.

    Measured: First dose date up to approximately 38 months

  4. Percentage of Participants Experiencing Treatment-Emergent Adverse Events

    Measured: First dose date up to the last dose date plus 30 days (maximum: 18 months)

  5. Percentage of Participants Who Experienced Laboratory Abnormalities

    Treatment-emergent laboratory abnormalities were defined as values that increase at least one toxicity grade from baseline. The most severe graded abnormality from all tests was counted for each participant.

    Measured: First dose date up to the last dose date plus 30 days (maximum: 18 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jan 2015 (estimated)
  2. Study start1 Jul 2015
  3. Primary completion4 Sep 2018
  4. Study completion21 Feb 2019

Resources

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 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
148 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02343939
Sponsor's protocol ID
GS-US-339-1559
Other IDs
2016-003353-16, 2016
Acronym
ENTO in AML
Status last verified
Nov 2019
First submitted
16 Jan 2015
Last updated
15 Nov 2019
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Entospletinib, Daunorubicin and Cytarabine in people with Acute Myeloid Leukemia, run by Gilead Sciences at 17 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 4 inclusion and 4 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 15 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in United States, Canada, Germany. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Yes. Results are posted in the registry (first posted 15 Nov 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Percentage of Participants Experiencing Dose Limiting Toxicities (DLTs) (time frame: Group A: Cycle 0 Day 1 to Cycle 2 Day 28; Group B: Cycle 0 Day 1 to Cycle 1 Day 28; Group C: Cycle 1 Day 1 to Cycle 1 Day 28 (Cycle length: for Cycle 0 = 14 days, for all other cycles = 28 days)). The study lists 4 primary outcomes in all. 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 15 Nov 2019.

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