Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation (AGILE)

A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of AG-120 in Combination With Azacitidine in Subjects ≥ 18 Years of Age With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

Study AG120-C-009 is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial to evaluate the efficacy and safety of AG-120 (ivosidenib) + azacitidine vs placebo + azacitidine in adult participants with previously untreated IDH1m AML who are considered appropriate candidates for non-intensive therapy. The primary endpoint is event-free survival (EFS). The key secondary efficacy endpoints are overall survival (OS), rate of complete remission (CR), rate of CR and complete remission with partial hematologic recovery (CRh), and overall response rate (ORR). Participants eligible for study treatment based on Screening assessments will be randomized 1:1 to receive oral AG-120 or matched placebo, both administered in combination with subcutaneous (SC) or intravenous (IV) azacitidine. An estimated 200 participants will take part in the study.

Study Overview

• Status: Active, not recruiting
• Conditions: Leukemia, Myeloid, Acute; Untreated AML; Newly Diagnosed Acute Myeloid Leukemia (AML); AML Arising From Myelodysplastic Syndrome (MDS)
• Intervention / Treatment: Drug: Placebo; Drug: AG-120; Drug: Azacitidine

• Study Type: Interventional
• Enrollment (Actual): 146
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Royal Prince Alfred Hospital
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
    • Bedford park, South Australia, Australia, 5042
      • Flinders Medical Centre
• Austria
  • Salzburg, Austria, 5020
    • Salzburger Landeskliniken
  • Wien, Austria, 1130
    • Krankenhaus Hietzing mit Neurologischem Zentrum Rosenhügel
• Brazil
  • Rio De Janeiro, Brazil, 20230-130
    • Instituto Nacional de Câncer
  • Sao Paulo, Brazil, 08270-070
    • Hospital Santa Marcelina
  • Sao Paulo, Brazil, 01308-050
    • Hospital Sírio Libanês
  • Sao Paulo, Brazil, 01321-001
    • Hospital Sao Jose
  • Sao Paulo
    • Campinas, Sao Paulo, Brazil, 13083-878
      • Unicamp Universidade Estadual de Campinas
    • Jau, Sao Paulo, Brazil, 17210-120
      • Hospital Amaral Carvalho
• Canada
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3E 0V9
      • Cancer Care Manitoba
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • University Health Network
• China
  • Beijing, China
    • Peking Union Medical College Hospital
  • Guangzhou, China, 510080
    • Guangdong Provincial People's Hospital
  • Hangzhou, China, 310003
    • The First Affiliated Hospital, College of Medicine, Zhejiang University
  • Tianjin, China, 300020
    • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
  • Henan
    • Zhengzhou, Henan, China, 450008
      • Henan Cancer Hospital
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • West China Hospital Sichuan University
• Czechia
  • Ostrava, Czechia
    • Fakultni Nemocnice Ostrava
• France
  • Brest, France, 29609
    • CHRU de Brest - Hôpital Morvan
  • Caen, France, 14000
    • Institut dHematologie de Basse Normandie
  • Grenoble, France, 38043
    • CHU de Grenoble
  • Le Chesnay, France, 78 157
    • Centre Hospitalier de Versailles CHV Hopital Andre Mignot
  • Paris, France, 75015
    • Groupe Hospitalier Necker Enfants Malades
  • Poitiers, France, 86021
    • CHRU de Poitiers La Miletrie
  • Strasbourg, France, 67200
    • Hopital de Hautepierre
  • Toulouse, France, 31059
    • EDOG - Institut Claudius Regaud - PPDS
  • Villejuif, France, 94805
    • Institut Gustave Roussy
  • Gironde
    • Pessac, Gironde, France, 33604
      • Hopital Haut Leveque
  • Indre-et-Loire
    • Tours, Indre-et-Loire, France, 37044
      • Hôpital Bretonneau
  • Loire-Atlantique
    • Nantes, Loire-Atlantique, France, 44093
      • Hôtel Dieu - Nantes
  • Rhone
    • Pierre-benite, Rhone, France, 69495
      • Centre Hospitalier Lyon Sud
  • Sarthe
    • Le Mans, Sarthe, France, 72037
      • Centre Hospitalier Le Mans
• Germany
  • Berlin, Germany, 13353
    • Charité - Universitätsmedizin Berlin
  • Hannover, Germany, 30625
    • Medizinische Hochschule Hannover
  • Leipzig, Germany, 04103
    • Universitätsklinikum Leipzig
  • Munchen, Germany, 81377
    • LMU Klinikum der Universität München
  • Ulm, Germany, 89081
    • Universitätsklinikum Ulm
  • Nordrhein-Westfalen
    • Essen, Nordrhein-Westfalen, Germany, 45122
      • Universitätsklinikum Essen
  • Sachsen
    • Chemnitz, Sachsen, Germany, 09113
      • Klinikum Chemnitz gGmbH
• Israel
  • Petah Tikva, Israel, 49100
    • Rabin Medical Center - PPDS
  • Rehovot, Israel, 7610000
    • Kaplan Medical Center
• Italy
  • Meldola, Italy, 47014
    • Istituto Scientifico Romagnolo Per Lo Studio E La Cura Dei Tumori IRST - PPDS
  • Milano, Italy, 20132
    • Ospedale San Raffaele S.r.l. - PPDS
  • Milano, Italy, 20162
    • ASST Grande Ospedale Metropolitano Niguarda - Presidio Ospedaliero Ospedale Niguarda Ca' Granda
  • Pavia, Italy, 27100
    • Fondazione IRCCS Policlinico San Matteo di Pavia
  • Rimini, Italy, 47900
    • Ospedale Infermi di Rimini
  • Torino, Italy, 10126
    • Azienda Ospedaliera Citta della Salute e della Scienza di Torino
  • Lombardia
    • Varese, Lombardia, Italy, 21100
      • ASST dei Sette Laghi - Ospedale Di Circolo E Fondazione Macchi
• Japan
  • Fukui, Japan, 910-1193
    • University of Fukui Hospital
  • Himeji, Japan, 670-8540
    • Japanese Red Cross Society Himeji Hospital
  • Kobe, Japan
    • Kobe City Medical Center General Hospital
  • Ehime
    • Matsuyama, Ehime, Japan, 790-8524
      • Matsuyama Red Cross Hospital
• Korea, Republic of
  • Busan, Korea, Republic of, 602-739
    • Pusan National University Hospital
  • Seoul, Korea, Republic of, 110-744
    • Seoul National University Hospital
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital Yonsei University Health System
  • Gyeonggido
    • Goyang-si, Gyeonggido, Korea, Republic of, 10408
      • National Cancer Center
    • Suwon-si, Gyeonggido, Korea, Republic of, 16499
      • Ajou University Hospital
• Mexico
  • Culiacan, Mexico, 80230
    • SINACOR
  • Mexico, Mexico, 14000
    • Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
• Netherlands
  • Nijmegen, Netherlands, 6525 GA
    • Universitair Medisch Centrum Groningen
  • Noord-Holland
    • Amsterdam, Noord-Holland, Netherlands, 1081 HV
      • VU Medisch Centrum
• Poland
  • Gdansk, Poland, 80-214
    • Uniwersyteckie Centrum Kliniczne
  • Dolnoslaskie
    • Wroclaw, Dolnoslaskie, Poland, 50-367
      • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
  • Mazowieckie
    • Warszawa, Mazowieckie, Poland, 02-776
      • Instytut Hematologii i Transfuzjologii
• Russian Federation
  • Kaluga, Russian Federation, 248007
    • Kaluga Regional Clinical Hospital
  • Moscow, Russian Federation, 129301
    • City Clinical Hospital # 40
• Spain
  • Barcelona, Spain, 08036
    • Hospital Clínic de Barcelona
  • Barcelona, Spain, 08035
    • Hospital Universitario Vall d'Hebron - PPDS
  • Madrid, Spain, 28034
    • Hospital Universitario Ramon Y Cajal
  • Madrid, Spain, 28040
    • Hospital Universitario Fundacion Jimenez Diaz
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre
  • Madrid, Spain, 28007
    • Hospital General Universitario Gregorio Marañón
  • Sevilla, Spain, 41013
    • Hospital Universitario Virgen del Rocio - PPDS
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe de Valencia
  • Zaragoza, Spain, 50009
    • Hospital Clínico Universitario Lozano Blesa
  • A Coruna
    • Santiago de Compostela, A Coruna, Spain, 15706
      • CHUS H. Clinico U. de Santiago
  • Baleares
    • Palma de Mallorca, Baleares, Spain, 07010
      • Hospital Universitario Son Espases
  • Barcelona
    • Badalona, Barcelona, Spain, 08916
      • Hospital Universitario Germans Trias i Pujol
  • Las Palmas
    • Las Palmas de Gran Canaria, Las Palmas, Spain, 35010
      • Hospital Universitario de Gran Canaria Doctor Negrin
• Taiwan
  • Changhua City, Taiwan, 500
    • Changhua Christian Medical Foundation Changhua Christian Hospital
  • Kaohsiung, Taiwan, 807
    • Kaohsiung Medical University Hospital
  • Taichung City, Taiwan, 40447
    • China Medical University Hospital
  • Tainan City, Taiwan, 736
    • Chi Mei Medical Center, Liouying
  • Taipei, Taiwan
    • National Taiwan University Hospital
• United Kingdom
  • West Midlands
    • Birmingham, West Midlands, United Kingdom, B9 5SS
      • Birmingham Heartlands Hospital
• United States
  • Kentucky
    • Louisville, Kentucky, United States, 40207
      • Norton Cancer Institute - Suburban
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Be ≥ 18 years of age and meet at least 1 of the following criteria defining ineligibility for intensive induction chemotherapy (IC): ≥ 75 years old, Eastern Cooperative Oncology Group Performance Status (ECOG PS) score of 2, severe cardiac disorder (e.g., congestive heart failure requiring treatment, left ventricular ejection fraction (LVEF), ≤50%, or chronic stable angina), severe pulmonary disorder (e.g., diffusing capacity of the lungs for carbon monoxide ≤65% or forced expiratory volume in 1 second ≤65%), creatinine clearance <45 mL/minute, bilirubin >1.5 times the upper limit of normal (ULN) and/or have any other comorbidity that the Investigator judges to be incompatible with intensive IC and must be reviewed and approved by the Medical Monitor before study enrollment.
2. Have previously untreated AML, defined according to World Health Organization (WHO) criteria, with ≥ 20% leukemic blasts in the bone marrow. Participants with extramedullary disease alone (i.e., no detectable bone marrow and no detectable peripheral blood AML) are not eligible for the study.
3. Have an isocitrate dehydrogenase 1 (IDH1) mutation.
4. Have an ECOG PS score of 0 to 2.
5. Have adequate hepatic function.
6. Have adequate renal function.
7. Have agreed to undergo serial blood and bone marrow sampling.
8. Be able to understand and willing to sign an informed consent form (ICF).
9. Be willing to complete Quality of Life assessments during the study
10. If female with reproductive potential, must have a negative serum pregnancy test prior to the start of study therapy. Females of reproductive potential, as well as fertile men and their female partners of reproductive potential, must agree to use 2 effective forms of contraception.

Exclusion Criteria:

1. Are candidates for and willing to receive intensive induction chemotherapy (IC) for their AML.
2. Have received any prior treatment for AML with the exception of hydroxyurea.
3. Have received a hypomethylating agent for myelodysplastic syndrome (MDS).
4. Participants who had previously received an experimental agent for MDS may not be randomized until a washout period has elapsed since the last dose of that agent.
5. Have received prior treatment with an IDH1 inhibitor.
6. Have a known hypersensitivity to any of the components of AG-120, matched placebo, or azacitidine.
7. Are female and pregnant or breastfeeding.
8. Have an active, uncontrolled, systemic fungal, bacterial, or viral infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment.
9. Have a prior history of cancer other than MDS or myeloproliferative disorder, unless the participant has been free of the disease for ≥ 1 year prior to the start of study treatment.
10. Have had significant active cardiac disease within 6 months prior to the start of the study treatment.
11. Have any condition that increases the risk of abnormal ECG or cardiac arrhythmia.
12. Have a condition that limits the ingestion or absorption of drugs administered by mouth.
13. Have uncontrolled hypertension (systolic blood pressure [BP] > 180 mmHg or diastolic BP > 100 mmHg).
14. Have clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia.
15. Have immediate, life-threatening, severe complications of leukemia, such as uncontrolled bleeding, pneumonia with hypoxia or sepsis, and/or disseminated intravascular coagulation.
16. Have any other medical or psychological condition deemed by the Investigator to be likely to interfere with the participant's ability to give informed consent or participate in the study.
17. Are taking medications that are known to prolong the QT interval unless they can be transferred to other medications within ≥5 half-lives prior to dosing, or unless the medications can be properly monitored during the study. (If equivalent medication is not available, heart rate corrected QT interval [QTc] will be closely monitored.)
18. Have a known medical history of progressive multifocal leukoencephalopathy.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AG-120 + Azacitidine; Participants received AG-120 500 mg orally, once daily (QD) in combination with azacitidine 75 milligrams per square meter per day (mg/m^2/day) subcutaneously (SC) or intravenously (IV), on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation.	Drug: AG-120; Tablets administered orally; Other Names: Ivosidenib; Drug: Azacitidine; Administered SC or IV; Other Names: Vidaza®
Placebo Comparator: Placebo + Azacitidine; Participants received AG-120 matching placebo orally, QD in combination with azacitidine 75 mg/m^2/day SC or IV, on Days 1-7, or on Days 1-5 and 8-9, of each 28-day cycle for a minimum of 6 cycles until death, disease relapse, disease progression, development of unacceptable toxicity (adverse event), confirmed pregnancy, withdrawal by participant or protocol violation .	Drug: Placebo; Tablets administered orally; Drug: Azacitidine; Administered SC or IV; Other Names: Vidaza®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Event-Free Survival (EFS)	EFS was defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure was defined as failure to achieve complete remission (CR) by Week 24. CR: Bone marrow blasts <5% and no Auer rods; absence of extramedullary disease; Absolute neutrophil count (ANC) ≥1.0 × 10^9 per litre (10^9/L) (1000 per microlitre [1000/μL]); platelet count ≥100 × 10^9/L (100,000/μL); independence of red blood cell transfusions. Participants who had an EFS event (relapse or death) after, 2 or more missing disease assessments were censored at the last adequate disease assessment documenting no relapse before the missing assessments. The reported data represents the Kaplan-Meier median value.	Up to Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete Remission Rate (CR Rate)	CR rate is defined as the proportion of participants who achieve a CR. A Cochran-Mantel-Haenszel (CMH) test will be used to compare CR rate between the 2 treatment arms.	Up to approximately 52 months
Overall Survival (OS)	OS is defined as the time from date of randomization to the date of death due to any cause. Kaplan-Meier (KM) curves and KM estimates of OS will be presented for each treatment arm.	Up to approximately 52 months
CR + Complete Remission With Partial Hematologic (CRh) Rate	CR + CRh rate is defined as the proportion of participants who achieve a CR or CRh. CRh is defined as a CR with partial recovery of peripheral blood counts (less than 5% bone marrow blasts, absolute neutrophil count (ANC) greater than 0.5 × 10^9/liter (L) 500/microliter (μL)], and platelets greater than 50 × 10^9/L [50,000/μL]). A CMH test will be used to compare the CR + CRh rate between the 2 treatment arms.	Up to approximately 52 months
Objective Response Rate (ORR)	ORR is defined as the rate of CR, CR with incomplete hematologic recovery (CRi) (including CR with incomplete platelet recovery [CRp]), partial remission (PR), and morphologic leukemia-free state (MLFS). The best response is calculated using the following hierarchy: CR, followed by CRi (including CRp), followed by PR and MLFS. A summary of best response by treatment arm will be produced. A CMH test will be used to compare ORR between the 2 treatment arms.	Up to approximately 52 months
CR + CRi (Including CRp) Rate	The CR + CRi (including CRp) rate is defined as the proportion of participants who achieve a CR or CRi (including CRp). A CMH test will be used to compare the CR + CRi (including CRp) rate between the 2 treatment arms.	Up to approximately 52 months
Duration of CR (DOCR)	DOCR will be calculated as the date of the first occurrence of CR to the date of first documented disease relapse, or death. DOCR is only defined for participants who achieve a CR.	Up to approximately 52 months
Duration of CRh (DOCRh)	DOCRh will be calculated as the date of the first occurrence of CR or CRh to the date of first documented disease relapse or death. DOCRh is only defined for participants who achieve a CR or CRh.	Up to approximately 52 months
Duration of Response (DOR)	DOR will be calculated as the date of the first response to the date of first documented disease relapse, disease progression, or death. DOR is only defined for participants who achieve a CR, CRi (including CRp), PR, and/or MLFS.	Up to approximately 52 months
Duration of CRi (DOCRi)	DOCRi will be calculated as the date of the first occurrence of CR or CRi (including CRp) to the date of the first documented relapse or death. DOCRi is only defined for participants who achieve a CR or CRi (including CRp).	Up to approximately 52 months
Time to CR (TTCR)	TTCR will be assessed from the date of randomization to the date of first occurrence of CR. TTCR is only defined for participants who achieve a CR.	Up to approximately 52 months
Time to CRh (TTCRh)	TTCRh will be assessed from the date of randomization to the date of first occurrence of CR or CRh. TTCRh is only defined for participants who achieve a CR or CRh.	Up to approximately 52 months
Time to Response (TTR)	TTR will be assessed from the date of randomization to the date of the first response. TTR is only defined for participants who achieve a CR, CRi (including CRp), PR, and/or MLFS.	Up to approximately 52 months
Time to CRi (TTCRi)	TTCRi will be assessed from the date of randomization to the date of first occurrence of CR or CRi (including CRp). TTCRi is only defined for participants who achieve a CR or CRi (including CRp).	Up to approximately 52 months
Units of Platelets and Red Blood Cells (RBC) Infused	All measures that are indicative of clinical benefit are measured like number of units of platelet and RBC infused.	Up to approximately 52 months
Rate of Infection		Up to approximately 52 months
Percentage of Participants With CR With IDH1 Mutation Clearance (MC)	CR with IDH1 MC is defined as a response of CR where there is no evidence of the IDH1 mutation by molecular techniques to below the level of detection (0.02%-0.04%) for ≥1 on-treatment time point. A CMH test will be used to compare the rate of CR between 2 treatment arms.	Up to approximately 52 months
Percentage of Participants With Abnormalities in Vital Sign Measurements	Vital signs will include body temperature, respiratory rate, blood pressure, and heart rate.	Up to approximately 52 months
Percentage of Participants With Abnormalities in Eastern Cooperative Oncology Group Performance Status (ECOG PS)		Up to approximately 52 months
Percentage of Participants With Abnormalities in 12-lead Electrocardiograms (ECGs)		Up to approximately 52 months
Percentage of Participants With Abnormalities in Echocardiogram (ECHO) or Multi-Gated Acquisition (MUGA) for Left Ventricular Ejection Fraction (LVEF)	LVEF is determined by ECHO or MUGA scan in participants.	Up to approximately 52 months
Percentage of Participants With Abnormalities in Clinical Laboratory Tests	Clinical laboratory assessments will include hematology, serum chemistry, coagulation.	Up to approximately 52 months
Percentage of Participants With Adverse Events (AEs)	An AE is defined as any untoward medical occurrence in a clinical investigation participant administered an investigational medicinal product; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug.	Up to approximately 52 months
Percentage of Participants With AEs of Special Interest (AESIs)	AESIs are AEs that are not solicited local or systemic AEs, they are predefined AEs that required close monitoring and prompt reporting to the sponsor. AESIs include protocol-specified QT prolongation, isocitrate dehydrogenase (IDH) differentiation syndrome and leukocytosis.	Up to approximately 52 months
Percentage of Participants With Serious Adverse Events (SAEs)	An SAE is defined as an untoward medical occurrence, significant hazard, contraindication, side effect or precaution that at any dose: results in death, is life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or is medically significant.	Up to approximately 52 months
Percentage of Participants With Adverse Events Leading to Discontinuation or Death	An AE is defined as any untoward medical occurrence in a clinical investigation participant administered an investigational medicinal product; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug.	Up to approximately 52 months
Percentage of Participants Using Concomitant Medications	Participants receiving concomitant medications will be adequately monitored by ECG controls, drug concentration (where applicable), and serum electrolytes (i.e., potassium and magnesium).	Up to approximately 52 months
Number of Days Spent Hospitalized		Up to approximately 52 months
Change From Baseline in the European Organisation for Research and Treatment of Cancer (EORTC) QLC-C30 Questionnaire	The EORTC QLQ-C30 questionnaire measures quality of life and consists of 30 questions that are incorporated into 5 functional domains (physical, role, cognitive, emotional, and social); a global health status/global quality of life; 3 symptom scales (fatigue, pain, and nausea and vomiting); and 6 single items that assess additional symptoms (dyspnea, appetite loss, sleep disturbance, constipation, and diarrhea) and the perceived financial burden of treatment experienced by participants with cancer.	Up to approximately 52 months
Change From Baseline in the EORTC EQ-5D-5L Questionnaire	The EORTC EQ-5D-5L questionnaire measures quality of life and spans 5 dimensions, including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, which are used to build a composite of the participant's health status.	Up to approximately 52 months
Percentage of Participants With Drug Exposure, Dose Modifications and Dose Intensities	The number of doses administered, total dose, duration of treatment, dose intensity, and the proportion of participants with dose modifications, will be summarized by treatment arm.	Up to approximately 52 months
Circulating Plasma Concentration of AG-120	Serial blood samples will be drawn before and after dosing of study treatment in order to determine circulating plasma concentrations.	Up to approximately 52 months
Circulating Plasma Concentration of 2-HG	Serial blood samples will be drawn before and after dosing of study treatment in order to determine circulating plasma concentrations.	Up to approximately 52 months

Collaborators and Investigators

• Sponsor: Institut de Recherches Internationales Servier

Publications and helpful links

General Publications

• Montesinos P, Recher C, Vives S, Zarzycka E, Wang J, Bertani G, Heuser M, Calado RT, Schuh AC, Yeh SP, Daigle SR, Hui J, Pandya SS, Gianolio DA, de Botton S, Dohner H. Ivosidenib and Azacitidine in IDH1-Mutated Acute Myeloid Leukemia. N Engl J Med. 2022 Apr 21;386(16):1519-1531. doi: 10.1056/NEJMoa2117344.

Study record dates

Study Major Dates

• Study Start (Actual): June 26, 2017
• Primary Completion (Actual): March 18, 2021
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: May 30, 2017
• First Submitted That Met QC Criteria: May 30, 2017
• First Posted (Actual): June 1, 2017

Study Record Updates

• Last Update Posted (Actual): April 5, 2024
• Last Update Submitted That Met QC Criteria: April 3, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; Leukemia; Azacitidine; AG-120; ivosidenib
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Myelodysplastic Syndromes; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Preleukemia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Azacitidine; Ivosidenib
• Other Study ID Numbers: AG120-C-009

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

• used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
• where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

• sponsored by Servier
• with a first patient enrolled as of 1 January 2004 onwards
• for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

• IPD Sharing Time Frame: After Marketing Authorisation in EEA or US if the study is used for the approval.
• IPD Sharing Access Criteria: Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Study Data/Documents

1. Individual Participant Data Set
2. Study Protocol
3. Statistical Analysis Plan
4. Informed Consent Form
5. Clinical Study Report
6. Study-level clinical trial data

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No