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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03173248




Registration number
NCT03173248
Ethics application status
Date submitted
30/05/2017
Date registered
1/06/2017
Date last updated
21/09/2020

Titles & IDs
Public title
Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Patients With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
Scientific title
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
Secondary ID [1] 0 0
AG120-C-009
Universal Trial Number (UTN)
Trial acronym
AGILE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Newly Diagnosed Acute Myeloid Leukemia (AML) 0 0
Untreated AML 0 0
AML Arising From Myelodysplastic Syndrome (MDS) 0 0
Leukemia, Myeloid, Acute 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AG-120 (ivosidenib) with Azacitidine
Treatment: Drugs - Placebo with Azacitidine

Experimental: AG-120 (ivosidenib) with Azacitidine -

Placebo Comparator: Placebo with Azacitidine -


Treatment: Drugs: AG-120 (ivosidenib) with Azacitidine
Continuous 28-day cycles of AG-120 (ivosidenib) 500 mg orally (PO) once daily (QD) in combination with azacitidine 75 mg/m2/day SC or IV for the first week of each cycle

Treatment: Drugs: Placebo with Azacitidine
Continuous 28-day cycles of Placebo orally (PO) once daily (QD) in combination with azacitidine 75 mg/m2/day SC or IV for the first week of each cycle

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Event-Free Survival (EFS) - EFS is defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure is defined as failure to achieve complete remission (CR) by Week 24.
Timepoint [1] 0 0
Up to approximately 52 months
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
Up to approximately 52 months
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
Up to approximately 52 months
Secondary outcome [3] 0 0
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.
Timepoint [3] 0 0
Up to approximately 52 months
Secondary outcome [4] 0 0
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.
Timepoint [4] 0 0
Up to approximately 52 months
Secondary outcome [5] 0 0
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.
Timepoint [5] 0 0
Up to approximately 52 months
Secondary outcome [6] 0 0
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.
Timepoint [6] 0 0
Up to approximately 52 months
Secondary outcome [7] 0 0
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.
Timepoint [7] 0 0
Up to approximately 52 months
Secondary outcome [8] 0 0
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.
Timepoint [8] 0 0
Up to approximately 52 months
Secondary outcome [9] 0 0
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).
Timepoint [9] 0 0
Up to approximately 52 months
Secondary outcome [10] 0 0
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.
Timepoint [10] 0 0
Up to approximately 52 months
Secondary outcome [11] 0 0
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.
Timepoint [11] 0 0
Up to approximately 52 months
Secondary outcome [12] 0 0
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.
Timepoint [12] 0 0
Up to approximately 52 months
Secondary outcome [13] 0 0
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).
Timepoint [13] 0 0
Up to approximately 52 months
Secondary outcome [14] 0 0
Percentage of Participants with Abnormalities in Vital Sign Measurements - Vital signs will include body temperature, respiratory rate, blood pressure, and heart rate.
Timepoint [14] 0 0
From Baseline up to approximately 1 week after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [15] 0 0
Percentage of Participants with Abnormalities in Eastern Cooperative Oncology Group Performance Status (ECOG PS)
Timepoint [15] 0 0
From Baseline up to approximately 1 week after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [16] 0 0
Percentage of Participants with Abnormalities in 12-lead Electrocardiograms (ECGs)
Timepoint [16] 0 0
From Baseline up to approximately 1 week after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [17] 0 0
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.
Timepoint [17] 0 0
From Baseline up to approximately 1 week after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [18] 0 0
Percentage of Participants with Abnormalities in Clinical Laboratory Tests - Clinical laboratory assessments will include hematology, serum chemistry, coagulation.
Timepoint [18] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [19] 0 0
Percentage of Participants with Adverse Events (AEs) - An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a 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.
Timepoint [19] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [20] 0 0
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.
Timepoint [20] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [21] 0 0
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.
Timepoint [21] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [22] 0 0
Percentage of Participants Using Concomitant Medications - Participants receiving concomitant medications will be adequately monitored by ECG controls, drug concentration (where applicable), and serum electrolytes (ie, potassium and magnesium).
Timepoint [22] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [23] 0 0
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.
Timepoint [23] 0 0
Up to approximately 52 months
Secondary outcome [24] 0 0
Rate of Infection
Timepoint [24] 0 0
Up to approximately 52 months
Secondary outcome [25] 0 0
Days Spent Hospitalized
Timepoint [25] 0 0
Up to approximately 52 months
Secondary outcome [26] 0 0
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.
Timepoint [26] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [27] 0 0
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.
Timepoint [27] 0 0
From Baseline up to approximately 4 weeks after last dose of treatment (up to a maximum of 52 months)
Secondary outcome [28] 0 0
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.
Timepoint [28] 0 0
Up to approximately 52 months
Secondary outcome [29] 0 0
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.
Timepoint [29] 0 0
From Baseline up to the last dose of treatment (up to a maximum of 52 months)
Secondary outcome [30] 0 0
Circulating Plasma Concentration of AG-120 and 2-HG - Serial blood samples will be drawn before and after dosing of study treatment in order to determine circulating plasma concentrations.
Timepoint [30] 0 0
From Baseline up to the last dose of treatment (up to a maximum of 52 months)

Eligibility
Key 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 (eg, congestive heart failure requiring treatment, LVEF, =50%, or chronic
stable angina), severe pulmonary disorder (eg, 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 (ie, 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.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [3] 0 0
Flinders Medical Centre - Bedford park
Recruitment hospital [4] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
5042 - Bedford park
Recruitment postcode(s) [4] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Kentucky
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
New Jersey
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
North Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Oregon
Country [14] 0 0
United States of America
State/province [14] 0 0
South Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
Austria
State/province [16] 0 0
Salzburg
Country [17] 0 0
Austria
State/province [17] 0 0
Wien
Country [18] 0 0
Brazil
State/province [18] 0 0
Goias
Country [19] 0 0
Brazil
State/province [19] 0 0
Parana
Country [20] 0 0
Brazil
State/province [20] 0 0
Rio Grande Do Sul
Country [21] 0 0
Brazil
State/province [21] 0 0
Sao Paulo
Country [22] 0 0
Brazil
State/province [22] 0 0
Porto Alegre
Country [23] 0 0
Brazil
State/province [23] 0 0
Rio De Janeiro
Country [24] 0 0
Canada
State/province [24] 0 0
Manitoba
Country [25] 0 0
Canada
State/province [25] 0 0
Ontario
Country [26] 0 0
Canada
State/province [26] 0 0
Quebec
Country [27] 0 0
China
State/province [27] 0 0
Henan
Country [28] 0 0
China
State/province [28] 0 0
Jiangsu
Country [29] 0 0
China
State/province [29] 0 0
Shanghai
Country [30] 0 0
China
State/province [30] 0 0
Sichuan
Country [31] 0 0
China
State/province [31] 0 0
Beijing
Country [32] 0 0
China
State/province [32] 0 0
Changsha
Country [33] 0 0
China
State/province [33] 0 0
Fuzhou
Country [34] 0 0
China
State/province [34] 0 0
Guangzhou
Country [35] 0 0
China
State/province [35] 0 0
Hangzhou
Country [36] 0 0
China
State/province [36] 0 0
Suzhou
Country [37] 0 0
China
State/province [37] 0 0
Tianjin
Country [38] 0 0
Czechia
State/province [38] 0 0
Praha, Hlavni Mesto
Country [39] 0 0
Czechia
State/province [39] 0 0
Ostrava
Country [40] 0 0
Czechia
State/province [40] 0 0
Praha 2
Country [41] 0 0
France
State/province [41] 0 0
Gironde
Country [42] 0 0
France
State/province [42] 0 0
Haut-Rhin
Country [43] 0 0
France
State/province [43] 0 0
Indre-et-Loire
Country [44] 0 0
France
State/province [44] 0 0
Loire-Atlantique
Country [45] 0 0
France
State/province [45] 0 0
Rhone
Country [46] 0 0
France
State/province [46] 0 0
Sarthe
Country [47] 0 0
France
State/province [47] 0 0
Brest
Country [48] 0 0
France
State/province [48] 0 0
Caen
Country [49] 0 0
France
State/province [49] 0 0
Clamart
Country [50] 0 0
France
State/province [50] 0 0
Clermont-Ferrand
Country [51] 0 0
France
State/province [51] 0 0
Dunkerque
Country [52] 0 0
France
State/province [52] 0 0
Grenoble
Country [53] 0 0
France
State/province [53] 0 0
Le Chesnay
Country [54] 0 0
France
State/province [54] 0 0
Limoges
Country [55] 0 0
France
State/province [55] 0 0
Paris
Country [56] 0 0
France
State/province [56] 0 0
Perigueux
Country [57] 0 0
France
State/province [57] 0 0
Poitiers
Country [58] 0 0
France
State/province [58] 0 0
Rouen
Country [59] 0 0
France
State/province [59] 0 0
Strasbourg
Country [60] 0 0
France
State/province [60] 0 0
Toulouse
Country [61] 0 0
France
State/province [61] 0 0
Villejuif
Country [62] 0 0
Germany
State/province [62] 0 0
Baden-Wurttemberg
Country [63] 0 0
Germany
State/province [63] 0 0
Nordrhein-Westfalen
Country [64] 0 0
Germany
State/province [64] 0 0
Sachsen
Country [65] 0 0
Germany
State/province [65] 0 0
Thuringen
Country [66] 0 0
Germany
State/province [66] 0 0
Berlin
Country [67] 0 0
Germany
State/province [67] 0 0
Bonn
Country [68] 0 0
Germany
State/province [68] 0 0
Hamburg
Country [69] 0 0
Germany
State/province [69] 0 0
Hannover
Country [70] 0 0
Germany
State/province [70] 0 0
Kiel
Country [71] 0 0
Germany
State/province [71] 0 0
Leipzig
Country [72] 0 0
Germany
State/province [72] 0 0
Munchen
Country [73] 0 0
Germany
State/province [73] 0 0
Tubingen
Country [74] 0 0
Germany
State/province [74] 0 0
Ulm
Country [75] 0 0
Israel
State/province [75] 0 0
Beer Sheva
Country [76] 0 0
Israel
State/province [76] 0 0
Haifa
Country [77] 0 0
Israel
State/province [77] 0 0
Holon
Country [78] 0 0
Israel
State/province [78] 0 0
Jerusalem
Country [79] 0 0
Israel
State/province [79] 0 0
Petah Tikva
Country [80] 0 0
Israel
State/province [80] 0 0
Rehovot
Country [81] 0 0
Israel
State/province [81] 0 0
Tel Aviv
Country [82] 0 0
Israel
State/province [82] 0 0
Tzrifin
Country [83] 0 0
Italy
State/province [83] 0 0
Lombardia
Country [84] 0 0
Italy
State/province [84] 0 0
Milano
Country [85] 0 0
Italy
State/province [85] 0 0
Meldola
Country [86] 0 0
Italy
State/province [86] 0 0
Palermo
Country [87] 0 0
Italy
State/province [87] 0 0
Pavia
Country [88] 0 0
Italy
State/province [88] 0 0
Rimini
Country [89] 0 0
Italy
State/province [89] 0 0
Torino
Country [90] 0 0
Japan
State/province [90] 0 0
Ehime
Country [91] 0 0
Japan
State/province [91] 0 0
Gihu
Country [92] 0 0
Japan
State/province [92] 0 0
Kanagawa
Country [93] 0 0
Japan
State/province [93] 0 0
Miyagi
Country [94] 0 0
Japan
State/province [94] 0 0
Okayama
Country [95] 0 0
Japan
State/province [95] 0 0
Saitama
Country [96] 0 0
Japan
State/province [96] 0 0
Tokyo
Country [97] 0 0
Japan
State/province [97] 0 0
Fukui
Country [98] 0 0
Japan
State/province [98] 0 0
Himeji
Country [99] 0 0
Japan
State/province [99] 0 0
Kobe
Country [100] 0 0
Japan
State/province [100] 0 0
Nagasaki
Country [101] 0 0
Japan
State/province [101] 0 0
Osaka
Country [102] 0 0
Korea, Republic of
State/province [102] 0 0
Gyeonggido
Country [103] 0 0
Korea, Republic of
State/province [103] 0 0
Busan
Country [104] 0 0
Korea, Republic of
State/province [104] 0 0
Jeonnam
Country [105] 0 0
Korea, Republic of
State/province [105] 0 0
Seoul
Country [106] 0 0
Mexico
State/province [106] 0 0
Estado De Mexico
Country [107] 0 0
Mexico
State/province [107] 0 0
Michoacan
Country [108] 0 0
Mexico
State/province [108] 0 0
Nuevo Leon
Country [109] 0 0
Mexico
State/province [109] 0 0
Culiacan
Country [110] 0 0
Mexico
State/province [110] 0 0
Mexico
Country [111] 0 0
Mexico
State/province [111] 0 0
Oaxaca
Country [112] 0 0
Netherlands
State/province [112] 0 0
Noord-Holland
Country [113] 0 0
Netherlands
State/province [113] 0 0
Nijmegen
Country [114] 0 0
Netherlands
State/province [114] 0 0
Utrecht
Country [115] 0 0
Poland
State/province [115] 0 0
Dolnoslaskie
Country [116] 0 0
Poland
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Mazowieckie
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Poland
State/province [117] 0 0
Gdansk
Country [118] 0 0
Poland
State/province [118] 0 0
Opole
Country [119] 0 0
Poland
State/province [119] 0 0
Poznan
Country [120] 0 0
Russian Federation
State/province [120] 0 0
Kaluga
Country [121] 0 0
Russian Federation
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Krasnoyarsk
Country [122] 0 0
Russian Federation
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Moscow
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Petrozavodsk
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Ryazan
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Russian Federation
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Ufa
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Russian Federation
State/province [127] 0 0
Volgograd
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State/province [128] 0 0
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Country [129] 0 0
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State/province [129] 0 0
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Spain
State/province [130] 0 0
Barcelona
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Spain
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Cataluna
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Spain
State/province [132] 0 0
Las Palmas
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Spain
State/province [133] 0 0
Madrid, Communidad Delaware
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Spain
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Caceres
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Spain
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Madrid
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Sevilla
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State/province [138] 0 0
Valencia
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Zaragoza
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Taiwan
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Changhua City
Country [141] 0 0
Taiwan
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Kaohsiung
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Taiwan
State/province [142] 0 0
Taichung City
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Taiwan
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Tainan City
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Taiwan
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Taipei
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United Kingdom
State/province [145] 0 0
Kent
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United Kingdom
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Oxfordshire
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United Kingdom
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West Midlands
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United Kingdom
State/province [148] 0 0
Boston
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Dundee
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London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Agios Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/show/NCT03173248
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Affairs Agios Pharmaceuticals, Inc.
Address 0 0
Country 0 0
Phone 0 0
1.833.228.8474
Fax 0 0
Email 0 0
medinfo@agios.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03173248