NCT03839771

Brief Summary

AML and MDS-EB2 are malignancies of the bone marrow. The standard treatment for these diseases is chemotherapy. Patients participating have a special type of this disease because the leukemia cells (blasts) have developed an error in the genetic material (DNA). This error is called an IDH1 mutation or an IDH2 mutation (a mutation is a change in the DNA), which leads to changes in specific substances in the leukemia cells. This trial will investigate whether the addition of the new drugs Ivosidenib (for patients with IDH1 mutation) or Enasidenib (for patients with IDH2 mutation) to the standard treatment of chemotherapy controle the disease more effectively and for a longer period.

Trial Health

87
On Track

Trial Health Score

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

Strong global presence with extensive site network
Enrollment
968

participants targeted

Target at P75+ for phase_3

Timeline
102mo left

Started Mar 2019

Longer than P75 for phase_3

Geographic Reach
13 countries

165 active sites

Status
active not recruiting

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

Study Progress46%
Mar 2019Sep 2034

First Submitted

Initial submission to the registry

February 6, 2019

Completed
9 days until next milestone

First Posted

Study publicly available on registry

February 15, 2019

Completed
14 days until next milestone

Study Start

First participant enrolled

March 1, 2019

Completed
8.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 17, 2027

Expected
7.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 19, 2034

Last Updated

October 2, 2024

Status Verified

September 1, 2024

Enrollment Period

8.1 years

First QC Date

February 6, 2019

Last Update Submit

October 1, 2024

Conditions

Outcome Measures

Primary Outcomes (1)

  • Event-free survival (EFS)

    EFS is defined as the time from randomization to failure to achieve CR or CRi after remission induction, death after achieving CR or CRi or relapse after achieving CR or CRi, whichever occurs first. A patient is said to have failed to achieve CR or CRi after induction therapy, if his/her best response during or at completion of the induction treatment is less than CRi. Patients who achieved CR/CRi after remission induction and are not known to have relapsed or died will be censored at the date of last clinical assessment.

    Approximately up to 60 months following first patient enrollment

Secondary Outcomes (11)

  • Overall survival (OS)

    Approximately up to 84 months following first patient enrollment

  • Relapse-free survival (RFS) after CR/CRi

    Approximately up to 60 months following first patient enrollment

  • Cumulative incidence of relapse (CIR) after CR/CRi

    Approximately up to 60 months following first patient enrollment

  • Cumulative incidence of death (CID) after CR/CRi

    Approximately up to 60 months following first patient enrollment

  • Complete remission without minimal residual disease (CRMRD-) rate after induction cycle 2

    Approximately up to 60 months following first patient enrollment

  • +6 more secondary outcomes

Study Arms (2)

Arm A: Placebo

PLACEBO COMPARATOR

Cycle 1: day 1-start cycle 2 \| Cycle 2: day 1 - start consolidation treatment \| Consolidation treatment: day 1 - start maintenance \| Maintenance treatment: day 1- day 730 (2 years) \| The dosage for Placebo for AG-120 (IDH1): 500 mg dose/day The dosage for Placebo for AG-221 (IDH2): 100mg dose/day

Drug: Placebo for AG-120Drug: Placebo for AG-221

Arm B: Ivosidenib (IDH1) or Enasidenib (IDH2)

EXPERIMENTAL

Cycle 1: day 1-start cycle 2 \| Cycle 2: day 1 - start consolidation treatment \| Consolidation treatment: day 1 - start maintenance \| Maintenance treatment: day 1- day 730 (2 years) \| The dosage for AG-120 (IDH1): 500 mg dose/day The dosage for AG-221 (IDH2): 100mg dose/day

Drug: AG-120Drug: AG-221

Interventions

AG-120DRUG

250mg tablets

Also known as: Ivosidenib
Arm B: Ivosidenib (IDH1) or Enasidenib (IDH2)

250mg tablets

Arm A: Placebo
AG-221DRUG

100mg tablets

Also known as: Enasidenib
Arm B: Ivosidenib (IDH1) or Enasidenib (IDH2)

100mg tablets

Arm A: Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age ≥18 years
  • Newly diagnosed AML or MDS-EB2 defined according to WHO criteria, with a documented IDH1 or IDH2 gene mutation (as determined by the clinical trial assay) at a specific site (IDH1 R132, IDH2 R140, IDH2 R172). AML may be secondary to prior hematological disorders, including MDS, and/or therapy-related (in which prior disease should have been documented to have existed for at least 3 months). Patients may have had previous treatment with hypomethylating agents (HMAs) for MDS. HMAs have to be stopped at least four weeks before registration
  • Patients with dual mutant FLT3 and IDH1 or IDH2 mutations may be enrolled only if, for medical or other reasons, treatment with a FLT3 inhibitor is not considered.
  • Considered to be eligible for intensive chemotherapy.
  • ECOG/WHO performance status ≤ 2
  • Adequate hepatic function as evidenced by:
  • Serum total bilirubin ≤ 2.5 × upper limit of normal (ULN) unless considered due to Gilbert's disease (e.g. a mutation in UGT1A1) (only for patients in IDH2 cohort), or leukemic involvement of the liver - following written approval by the (Co)Principal Investigator.
  • Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤ 3.0 × ULN, unless considered due to leukemic involvement of the liver, following written approval by the Principal Investigator.
  • Adequate renal function as evidenced by creatinine clearance \> 40 mL/min based on the Cockroft-Gault formula for glomerular filtration rate (GFR).
  • Able to understand and willing to sign an informed consent form (ICF).
  • Written informed consent
  • Female patient must either:
  • o Be of nonchildbearing potential: Postmenopausal (defined as at least 1 year without any menses) prior to screening, or Documented surgically sterile or status posthysterectomy (at least 1 month prior to screening)
  • o Or, if of childbearing potential: Agree not to try to become pregnant during the study and for 6 months after the final study drug administration And have a negative urine or serum pregnancy test at screening And, if heterosexually active, agree to consistently use highly effective\* contraception per locally accepted standards in addition to a barrier method starting at screening and throughout the study period and for 6 months after the final study drug administration.
  • Highly effective forms of birth control include:
  • +12 more criteria

You may not qualify if:

  • Prior chemotherapy for AML or MDS-EB2 (with the exception of HMA). Hydroxyurea is allowed for the control of peripheral leukemic blasts in patients with leukocytosis (e.g., white blood cell \[WBC\] counts \> 30x109/L).
  • Dual IDH1 and IDH2 mutations.
  • Acute promyelocytic leukemia (APL) with PML-RARA or one of the other pathognomonic variant fusion genes/chromosome translocations.
  • Blast crisis after chronic myeloid leukemia (CML).
  • Known allergy or suspected hypersensitivity to Ivosidenib or Enasidenib and/or any exipients.
  • Taking medications with narrow therapeutic windows with potential interaction with investigational medication (see Appendix I), unless the patient can be transferred to other medications prior to enrolling or unless the medications can be properly monitored during the study.
  • Taking P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) transporter-sensitive substrate medications (see Appendix J) unless the patient can be transferred to other medications within ≥ 5 half-lives prior to administration of ivosidenib or enasidenib, or unless the medications can be properly monitored during the study.
  • Breast feeding at the start of study treatment.
  • Active infection, including hepatitis B or C or HIV infection that is uncontrolled at randomization. An infection controlled with an approved or closely monitored antibiotic/antiviral/antifungal treatment is allowed.
  • Patients with a currently active second malignancy. Patients are not considered to have a currently active malignancy if they have completed therapy and are considered by their physician to be at \< 30% risk of relapse within one year. However, patients with the following history/concurrent conditions are allowed:
  • Basal or squamous cell carcinoma of the skin
  • Carcinoma in situ of the cervix
  • Carcinoma in situ of the breast
  • Incidental histologic finding of prostate cancer
  • Significant active cardiac disease within 6 months prior to the start of study treatment, including New York Heart Association (NYHA) Class III or IV congestive heart failure (appendix G); myocardial infarction, unstable angina and/or stroke; or left ventricular ejection fraction (LVEF) \< 40% by ultrasound or MUGA scan obtained within 28 days prior to the start of study treatment.
  • +8 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (181)

AU-Adelaide-FLINDERS

Adelaide, Australia

Location

AU-Adelaide-RAH

Adelaide, Australia

Location

AU-Brisbane-PAH

Brisbane, Australia

Location

AU-Camperdown-RPA

Camperdown, Australia

Location

AU-Canberra-CANBERRAHOSPITAL

Canberra, Australia

Location

AU-Douglas-TOWNSVILLE

Douglas, Australia

Location

AU-Hobart TAS-RHOBART

Hobart, Australia

Location

AU-Launceston TAS-LAUNCESTON

Launceston, Australia

Location

AU-Melbourne-ALFRED

Melbourne, Australia

Location

AU-Melbourne-AUSTIN

Melbourne, Australia

Location

AU-Melbourne-MONASH

Melbourne, Australia

Location

AU-Melbourne-RMELBOURNE

Melbourne, Australia

Location

AU-Melbourne-SVHM

Melbourne, Australia

Location

AU-Perth-FSH

Perth, Australia

Location

AU-Perth-RPH

Perth, Australia

Location

AU-Perth-SCGH

Perth, Australia

Location

AU-Sydney-CONCORD

Sydney, Australia

Location

AU-Sydney-RNSH

Sydney, Australia

Location

AU-Sydney-SVHS

Sydney, Australia

Location

AU-Sydney-WSAH

Sydney, Australia

Location

AU-Waratah-CALVARYMATER

Waratah, Australia

Location

AT-Graz-MEDUNIGRAZ

Graz, Austria

Location

AT-Innsbruck-IMED

Innsbruck, Austria

Location

AT-Linz-ORDENSKLINIKUM

Linz, Austria

Location

AT-Vienna-HANUSCH

Vienna, Austria

Location

BE-Antwerpen-ZNASTUIVENBERG

Antwerp, Belgium

Location

BE-Brugge-AZBRUGGE

Bruges, Belgium

Location

BE-Brussel-BORDET

Brussels, Belgium

Location

BE-Brussel-UZBRUSSEL

Brussels, Belgium

Location

BE-Bruxelles-STLUC

Brussels, Belgium

Location

BE-Gent-UZGENT

Ghent, Belgium

Location

BE-Haine-Saint-Paul-JOLIMONT

Haine-Saint-Paul, Belgium

Location

BE-Hasselt-VIRGAJESSE

Hasselt, Belgium

Location

BE-Leuven-UZLEUVEN

Leuven, Belgium

Location

BE-Liege-CHRCITADELLE

Liège, Belgium

Location

BE-Liege-CHULIEGE

Liège, Belgium

Location

BE-Roeselare-AZDELTA

Roeselare, Belgium

Location

BE-Yvoir-MONTGODINNE

Yvoir, Belgium

Location

EE-Tartu-TARTU

Tartu, Estonia

Location

FI-Helsinki-HUS

Helsinki, Finland

Location

FI-Tampere-TAYS

Tampere, Finland

Location

FR-Amiens-CHUAMIENS

Amiens, France

Location

FR-Angers-CHUANGERS

Angers, France

Location

FR-Argenteuil-CHARGENTEUIL

Argenteuil, France

Location

FR-Bayonne-CHCOTEBASQUE

Bayonne, France

Location

FR-Besançon Cedex-JEANMINJOZ

Besançon, France

Location

FR-Bobigny-AVICENNE

Bobigny, France

Location

FR-Chambery-CHMETROPOLESAVOIE

Chambéry, France

Location

FR-Le Chesnay cedex-CHVERSAILLES

Chesnay, France

Location

FR-Clamart-HIAPERCY

Clamart, France

Location

FR-Clermont-Ferrand-ESTAING

Clermont-Ferrand, France

Location

FR-Créteil cedex-CHUMONDOR

Créteil, France

Location

FR-Grenoble cedex 9-CHUGRENOBLE

Grenoble, France

Location

FR-Lens-CHLENS

Lens, France

Location

FR-Lille-CHULILLE

Lille, France

Location

FR-Limoges-CHULIMOGES

Limoges, France

Location

FR-Lyon Pierre Benite cedex-LYONSUD

Lyon, France

Location

FR-Lyon-LEONBERARD

Lyon, France

Location

FR-Marseille-IPC

Marseille, France

Location

FR-Montpellier-STELOI

Montpellier, France

Location

FR-Mulhouse-GHRMSA

Mulhouse, France

Location

FR-Nantes-CHUNANTES

Nantes, France

Location

FR-Nice-CAL

Nice, France

Location

FR-Nice-LARCHET

Nice, France

Location

FR-Orléans-CHORLEANS

Orléans, France

Location

FR-Paris cedex 10-SAINTLOUIS

Paris, France

Location

FR-Paris cedex 12-SAINTANTOINE

Paris, France

Location

FR-Paris cedex 15-NECKER

Paris, France

Location

FR-Pessac Cedex-CHUBORDEAUX

Pessac, France

Location

FR-Poitiers-CHUPOITERS

Poitiers, France

Location

FR-Reims-CHREIMS

Reims, France

Location

FR-Rennes cedex 9-CHURENNES

Rennes, France

Location

FR-Rouen cedex-BECQUEREL

Rouen, France

Location

FR-Strasbourg cedex-HAUTEPIERRE

Strasbourg, France

Location

FR-Toulouse-CHUTOULOUSE

Toulouse, France

Location

FR-Tours cedex-BRETONNEAU

Tours, France

Location

FR-Vandoeuvre Les Nancy-CHRUNANCY

Vandœuvre-lès-Nancy, France

Location

FR-Villejuif-GUSTAVEROUSSY

Villejuif, France

Location

DE-Bad Saarow-HELIOSBADSAAROW

Bad Saarow, Germany

Location

DE-Berlin-CAMPUSBENFRANKLIN

Berlin, Germany

Location

DE-Berlin-CAMPUSVIRCHOW

Berlin, Germany

Location

DE-Berlin-VIVANTESNEUKOLLN

Berlin, Germany

Location

DE-Berlin-VIVANTESURBAN

Berlin, Germany

Location

DE-Bochum-RUB

Bochum, Germany

Location

DE-Bonn-UNIBONN

Bonn, Germany

Location

DE-Braunschweig-KLINIKUMBRAUNSCHWEIG

Braunschweig, Germany

Location

DE-Bremen-KBM

Bremen, Germany

Location

DE-Dortmund-JOHODORTMUND

Dortmund, Germany

Location

DE-Düsseldorf-MEDUNIDUESSELDORF

Düsseldorf, Germany

Location

DE-Essen-KEM

Essen, Germany

Location

DE-Esslingen-KLINIKUMESSLINGEN

Esslingen am Neckar, Germany

Location

DE-Flensburg-MALTESER

Flensburg, Germany

Location

DE-Giessen-UKGM

Giessen, Germany

Location

DE-Goch-KKLE

Goch, Germany

Location

DE-Hamburg-ASKLEPIOSSTGEORG

Hamburg, Germany

Location

DE-Hamburg-ASKLEPIOS

Hamburg, Germany

Location

DE-Hamburg-UKE

Hamburg, Germany

Location

DE-Hamm-EVKHAMM

Hamm, Germany

Location

DE-Hanau-KLINIKUMHANAU

Hanau, Germany

Location

DE-Hannover-MHHANNOVER

Hanover, Germany

Location

DE-Hannover-SILOAHKRH

Hanover, Germany

Location

DE-Herne-MARIENHOSPITALHERNE

Herne, Germany

Location

DE-Homburg-UNIKLINIKSAARLAND

Homburg, Germany

Location

DE-Karlsruhe-KLINIKUMKARLSRUHE

Karlsruhe, Germany

Location

DE-Lebach-CARITASKHLEBACH

Lebach, Germany

Location

DE-Lemgo-KLINIKUMLIPPE

Lemgo, Germany

Location

DE-Ludwigshafen-KLILU

Ludwigshafen, Germany

Location

DE-Luedenscheid-KLINIKUMLUEDENSCHEID

Lüdenscheid, Germany

Location

DE-Magdeburg-OVGU

Magdeburg, Germany

Location

DE-Mainz-KLINKUNIMAINZ

Mainz, Germany

Location

DE-Mainz-UNIMEDIZINMAINZ

Mainz, Germany

Location

DE-Meschede-HOCHSAUERLAND

Meschede, Germany

Location

DE-Minden-MUEHLENKREISKLINKEN

Minden, Germany

Location

DE-München-IRZTUM

München, Germany

Location

DE-München-MEDUNIMUNCHIN

München, Germany

Location

DE-Offenburg-ORTENAUKLINIKUM

Offenburg, Germany

Location

DE-Oldenburg-KLINIKUMOLDENBURG

Oldenburg, Germany

Location

DE-Passau-KLINIKUMPASSAU

Passau, Germany

Location

DE-Stuttgart-DIAKSTUTTGART

Stuttgart, Germany

Location

DE-Stuttgart-KLINIKUMSTUTTGART

Stuttgart, Germany

Location

DE-Traunstein-TSSOB

Traunstein, Germany

Location

DE-Trier-MUTTERHAUS

Trier, Germany

Location

DE-Tübingen-MEDUNITUEBINGEN

Tübingen, Germany

Location

DE-Ulm-UNIKLINKULM

Ulm, Germany

Location

DE-Villingen-Schwenningen-SBKVS

Villingen-Schwenningen, Germany

Location

DE-Wuppertal-HELIOSGESUNDHEIT

Wuppertal, Germany

Location

IE-Cork-CUH

Cork, Ireland

Location

IE-Dublin 8-STJAMES

Dublin, Ireland

Location

IE-Dublin 9-BEAUMONT

Dublin, Ireland

Location

IE-Galway-UHGALWAY

Galway, Ireland

Location

LT-Vilnius-SANTA

Vilnius, Lithuania

Location

LU-Luxembourg-CHL

Luxembourg, Luxembourg

Location

NL-Den Bosch-JBZ

's-Hertogenbosch, Netherlands

Location

NL-Amersfoort-MEANDERMC

Amersfoort, Netherlands

Location

NL-Amsterdam-AMC

Amsterdam, Netherlands

Location

NL-Amsterdam-OLVG

Amsterdam, Netherlands

Location

NL-Amsterdam-VUMC

Amsterdam, Netherlands

Location

NL-Arnhem-RIJNSTATE

Arnhem, Netherlands

Location

NL-Breda-AMPHIA

Breda, Netherlands

Location

NL-Delft-RDGG

Delft, Netherlands

Location

NL-Dordrecht-ASZ

Dordrecht, Netherlands

Location

NL-Eindhoven-MAXIMAMC

Eindhoven, Netherlands

Location

NL-Enschede-MST

Enschede, Netherlands

Location

NL-Groningen-UMCG

Groningen, Netherlands

Location

NL-Leeuwarden-MCL

Leeuwarden, Netherlands

Location

NL-Leiden-LUMC

Leiden, Netherlands

Location

NL-Maastricht-MUMC

Maastricht, Netherlands

Location

NL-Nieuwegein-ANTONIUS

Nieuwegein, Netherlands

Location

NL-Nijmegen-RADBOUDUMC

Nijmegen, Netherlands

Location

NL-Rotterdam-ErasmusMC

Rotterdam, Netherlands

Location

NL-Den Haag-HAGA

The Hague, Netherlands

Location

NL-Utrecht-UMCUTRECHT

Utrecht, Netherlands

Location

NL-Zwolle-ISALA

Zwolle, Netherlands

Location

NO-Bergen-HELSEBERGEN

Bergen, Norway

Location

NO-Oslo-OSLOUH

Oslo, Norway

Location

NO-Stavanger-HELSESTAVANGER

Stavanger, Norway

Location

NO-Tromsø-NORTHNOORWEGEN

Tromsø, Norway

Location

NO-Trondheim-STOLAV

Trondheim, Norway

Location

ES-Barcelona-CLINICUB

Barcelona, Spain

Location

ES-Barcelona-GERMANTRIALS

Barcelona, Spain

Location

ES-Barcelona-ICODURANREYNALS

Barcelona, Spain

Location

ES-Barcelona-MUTUATERRASSA

Barcelona, Spain

Location

ES-Barcelona-PARCDESALUTMAR

Barcelona, Spain

Location

ES-Barcelona-SANTPAU

Barcelona, Spain

Location

ES-Barcelona-VHEBRON

Barcelona, Spain

Location

ES-Girona-ICSTRUETA

Girona, Spain

Location

ES-Madrid-CSGREGORIOMARANON

Madrid, Spain

Location

ES-Palma-SSIB

Palma, Spain

Location

ES-Tarragona-JOAN

Tarragona, Spain

Location

ES-Valencia-MALVARROSA

Valencia, Spain

Location

SE-Lund-SUH

Lund, Sweden

Location

SE-Stockholm-KAROLINSKAHUDDINGE

Stockholm, Sweden

Location

SE-Uppsala-UPPSALAUH

Uppsala, Sweden

Location

CH-Basel-USB

Basel, Switzerland

Location

CH-Bellinzona-IOSI

Bellinzona, Switzerland

Location

CH-Bern-INSEL

Bern, Switzerland

Location

CH-Fribourg-HFR

Fribourg, Switzerland

Location

CH-Geneve (14)-HCUGE

Geneva, Switzerland

Location

CH-Luzern-LUKS

Lucerne, Switzerland

Location

CH-St. Gallen-KSSG

Sankt Gallen, Switzerland

Location

CH-Zürich-USZ

Zurich, Switzerland

Location

Related Links

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

ivosidenibenasidenib

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • B.J. Wouters

    Erasmus MC / HOVON

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 6, 2019

First Posted

February 15, 2019

Study Start

March 1, 2019

Primary Completion (Estimated)

April 17, 2027

Study Completion (Estimated)

September 19, 2034

Last Updated

October 2, 2024

Record last verified: 2024-09

Data Sharing

IPD Sharing
Will not share

Locations