NCT01814826

Brief Summary

The purpose of this study is to establish the maximum tolerated dose (MTD), and to assess the safety and tolerability of MLN4924 (pevonedistat) in combination with azacitidine in treatment naive participants with AML who were 60 years of age or older.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
64

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Apr 2013

Longer than P75 for phase_1

Geographic Reach
1 country

8 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 18, 2013

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 20, 2013

Completed
21 days until next milestone

Study Start

First participant enrolled

April 10, 2013

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 3, 2016

Completed
1.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 8, 2018

Completed
1.9 years until next milestone

Results Posted

Study results publicly available

March 3, 2020

Completed
Last Updated

March 3, 2020

Status Verified

February 1, 2020

Enrollment Period

3.1 years

First QC Date

March 18, 2013

Results QC Date

December 5, 2019

Last Update Submit

February 18, 2020

Conditions

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (3)

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Baseline up to 30 days after the last dose of study drug (up to 5 years)

  • Number of Participants With TEAEs Related to Clinically Significant Laboratory Evaluation Findings

    Baseline up to 30 days after the last dose of study drug (up to 5 years)

  • Number of Participants With TEAEs Related to Clinically Significant Vital Sign Findings

    Baseline up to 30 days after the last dose of study drug (up to 5 years)

Secondary Outcomes (27)

  • Dose-escalation Phase, Cmax: Maximum Observed Plasma Concentration for MLN4924

    Cycle 1 Day 1 pre-dose and at multiple time points (up to 24 hours) post-dose; Cycle 1 Day 5 pre-dose and at multiple time points (up to 48 hours) post-dose (Cycle length is equal to [=] 28 days)

  • Maximum Tolerated Dose (MTD) Expansion Phase, Cmax: Maximum Observed Plasma Concentration for MLN4924

    Cycle 1 Day 1 pre-dose and at multiple time points (up to 24 hours) post-dose; Cycle 1 Day 5 pre-dose and at multiple time points (up to 48 hours) post-dose (Cycle length = 28 days)

  • Dose-escalation Phase, Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for MLN4924

    Cycle 1 Day 1 pre-dose and at multiple time points (up to 24 hours) post-dose; Cycle 1 Day 5 pre-dose and at multiple time points (up to 48 hours) post-dose (Cycle length = 28 days)

  • MTD Expansion Phase, Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for MLN4924

    Cycle 1 Day 1 pre-dose and at multiple time points (up to 24 hours) post-dose; Cycle 1 Day 5 pre-dose and at multiple time points (up to 48 hours) post-dose (Cycle length = 28 days)

  • Dose-escalation Phase, Ctrough: Observed Plasma Concentration at the End of the Dosing Interval for MLN4924

    Cycle 1 Day 1 pre-dose and at multiple time points (up to 24 hours) post-dose; Cycle 1 Day 5 pre-dose and at multiple time points (up to 48 hours) post-dose (Cycle length = 28 days)

  • +22 more secondary outcomes

Study Arms (1)

MLN4924 and Azacitidine

EXPERIMENTAL
Drug: MLN4924Drug: Azacitidine

Interventions

MLN4924 intravenously (IV) in AML participants in a 28-day cycle: * MLN4924 on Days 1, 3, and 5 for Cycle 1 and all subsequent cycles

Also known as: Pevonedistat
MLN4924 and Azacitidine

Azacitidine (IV) or subcutaneously in AML participants in a 28-day cycle: \- Azacitidine Days 1, 2, 3, 4, 5, 8, 9 in Cycle 1 and for all subsequent cycles

MLN4924 and Azacitidine

Eligibility Criteria

Age60 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Participants with world health organization (WHO)-defined AML, 60 years of age or older, who are unlikely to benefit from standard induction therapy, defined as having at least 1 of the following:
  • Greater than or equal to 75 years of age.
  • Antecedent hematologic disease.
  • Known adverse cytogenetic risk.
  • Eastern Cooperative Oncology Group (ECOG) PS = 2.
  • Participant must not have received definitive treatment for AML, defined as any prior chemotherapy with antileukemic activity.
  • ECOG PS 0 to 2.
  • Expected survival longer than 3 months from enrollment in the study.
  • Female participants who are post menopausal, surgically sterile, or agree to practice 2 effective methods of contraception or agree to practice true abstinence.
  • Male participants who agree to practice effective barrier contraception or agree to practice true abstinence.
  • Voluntary written consent must be given before performance of any study-related procedure.
  • Suitable venous access for the study-required blood sampling.
  • Clinical laboratory values as specified below within 3 days before the first dose of any study drug:
  • Total bilirubin must be less than or equal to (\<=) the upper limit of the normal range (ULN).
  • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) must be\<=2.5\*ULN.
  • +6 more criteria

You may not qualify if:

  • Previous treatment with azacitidine or decitabine.
  • Known favorable cytogenetic risk.
  • Any serious medical or psychiatric illness.
  • Treatment with any investigational products.
  • Known hypersensitivity to azacitidine or mannitol.
  • Acute promyelocytic leukemia as diagnosed by morphologic examination of bone marrow, by fluorescent in situ hybridization or cytogenetics of peripheral blood or bone marrow, or by other accepted analysis.
  • Active uncontrolled infection or severe infectious disease.
  • Major surgery within 14 days before the first dose of study drug.
  • Life-threatening illness unrelated to cancer.
  • Clinically uncontrolled central nervous system (CNS) involvement.
  • WBC count greater than (\>) 50,000/ mcL.
  • Prothrombin time (PT) or activated partial thromboplastin time (aPTT) \>1.5\* ULN or a history of coagulopathy or bleeding disorder
  • Known human immunodeficiency virus (HIV) positive.
  • Known hepatitis B surface antigen-positive, or known or suspected active hepatitis C infection
  • Known hepatic cirrhosis or severe pre-existing hepatic impairment.
  • +14 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Stanford University

Stanford, California, 94305-5826, United States

Location

Hospital Corporation of America-HealthOne, LLC

Denver, Colorado, 80218, United States

Location

Mayo Clinic - Jacksonville, FL

Jacksonville, Florida, 32224, United States

Location

University of Miami School of Medicine

Miami, Florida, 33136, United States

Location

UNC-Chapel Hill School of Medicine

Chapel Hill, North Carolina, 27599, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Methodist Hospital

San Antonio, Texas, 78229, United States

Location

Related Publications (2)

  • Swords RT, Coutre S, Maris MB, Zeidner JF, Foran JM, Cruz J, Erba HP, Berdeja JG, Tam W, Vardhanabhuti S, Pawlikowska-Dobler I, Faessel HM, Dash AB, Sedarati F, Dezube BJ, Faller DV, Savona MR. Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, combined with azacitidine in patients with AML. Blood. 2018 Mar 29;131(13):1415-1424. doi: 10.1182/blood-2017-09-805895. Epub 2018 Jan 18.

  • Faessel HM, Mould DR, Zhou X, Faller DV, Sedarati F, Venkatakrishnan K. Population pharmacokinetics of pevonedistat alone or in combination with standard of care in patients with solid tumours or haematological malignancies. Br J Clin Pharmacol. 2019 Nov;85(11):2568-2579. doi: 10.1111/bcp.14078. Epub 2019 Sep 4.

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

pevonedistatAzacitidine

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Results Point of Contact

Title
Medical director
Organization
Takeda

Study Officials

  • Medical Monitor

    Millennium Pharmaceuticals, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Masking
NONE
Purpose
OTHER
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 18, 2013

First Posted

March 20, 2013

Study Start

April 10, 2013

Primary Completion

May 3, 2016

Study Completion

April 8, 2018

Last Updated

March 3, 2020

Results First Posted

March 3, 2020

Record last verified: 2020-02

Data Sharing

IPD Sharing
Will share

Takeda makes patient-level, de-identified data sets and associated documents available for all interventional studies after applicable marketing approvals and commercial availability have been received (or program is completely terminated), an opportunity for the primary publication of the research and final report development has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com for details). To obtain access, researchers must submit a legitimate academic research proposal for adjudication by an independent review panel, who will review the scientific merit of the research and the requestor's qualifications and conflict of interest that can result in potential bias. Once approved, qualified researchers who sign a data sharing agreement are provided access to these data in a secure research environment.

Locations