TriangleNCT03173248 · STUDY IN CONTEXT
Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
Draft scope assessment: unclear. Attached because the study's own record names IDH1. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
Read the full study record and all posted results →
Linked outcomes
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.
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.
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.
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.
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.
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.
Reported safety
Quality of life and patient-reported measures
EORTC QLQ · Change From Baseline in the European Organisation for Research and Treatment of Cancer (EORTC) QLC-C30 Questionnaire
Score direction: Not stated by the study. Direction is never inferred from an instrument name.
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- qol key
- qol-0027
- source section id
- NCT03173248-om-0027
- source outcome index
- 27
- instrument
- EORTC QLQ
- instrument basis
- matched 'eortc' in the study's own wording
- scale title
- Change From Baseline in the European Organisation for Research and Treatment of Cancer (EORTC) QLC-C30 Questionnaire
- measure definition
- 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.
- time frame
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
EORTC QLQ · Change From Baseline in the EORTC EQ-5D-5L Questionnaire
Score direction: Not stated by the study. Direction is never inferred from an instrument name.
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- qol key
- qol-0028
- source section id
- NCT03173248-om-0028
- source outcome index
- 28
- instrument
- EORTC QLQ
- instrument basis
- matched 'eortc' in the study's own wording
- scale title
- Change From Baseline in the EORTC EQ-5D-5L Questionnaire
- measure definition
- 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.
- time frame
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- trial label
- Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation
- applicability
- unclear
- applicability note
- Attached because the study's own record names IDH1. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- provenance class
- regulator-cited
- discovery note
- Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
- outcome Count
- 6
- harms Count
- 2
- qol Count
- 2
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0000
- source section id
- NCT03173248-om-0000
- source outcome index
- 0
- endpoint
- Event-Free Survival (EFS)
- endpoint definition
- 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.
- time origin
- Up to Week 24
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- FAS included all participants who were randomized.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0001
- source section id
- NCT03173248-om-0001
- source outcome index
- 1
- endpoint
- Complete Remission Rate (CR Rate)
- endpoint definition
- 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.
- time origin
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0002
- source section id
- NCT03173248-om-0002
- source outcome index
- 2
- endpoint
- Overall Survival (OS)
- endpoint definition
- 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.
- time origin
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0003
- source section id
- NCT03173248-om-0003
- source outcome index
- 3
- endpoint
- CR + Complete Remission With Partial Hematologic (CRh) Rate
- endpoint definition
- 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.
- time origin
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0004
- source section id
- NCT03173248-om-0004
- source outcome index
- 4
- endpoint
- Objective Response Rate (ORR)
- endpoint definition
- 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.
- time origin
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0005
- source section id
- NCT03173248-om-0005
- source outcome index
- 5
- endpoint
- CR + CRi (Including CRp) Rate
- endpoint definition
- 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.
- time origin
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- data cutoff
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- arms
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0000
- arm key
- OG000
- arm label
- AG-120 + Azacitidine
- regimen
- Participants received AG-120 500 mg 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.
- analyzed count
- 72
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03173248-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- outcome key
- om-0000
- arm key
- OG001
- arm label
- Placebo + Azacitidine
- regimen
- 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.
- analyzed count
- 74
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03173248-om-0000#groups/OG001
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- harm key
- EG000
- source section id
- NCT03173248-ae
- event group key
- EG000
- event group label
- AG-120 + Azacitidine
- serious affected
- 49
- serious at risk
- 71
- other affected
- 68
- other at risk
- 71
- frequency threshold
- 5
- collection period
- 3 years
- event definition
- SAS included all participants who received at least 1 dose of the study treatment.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- harm key
- EG001
- source section id
- NCT03173248-ae
- event group key
- EG001
- event group label
- Placebo + Azacitidine
- serious affected
- 60
- serious at risk
- 73
- other affected
- 72
- other at risk
- 73
- frequency threshold
- 5
- collection period
- 3 years
- event definition
- SAS included all participants who received at least 1 dose of the study treatment.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- quality of life
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- qol key
- qol-0027
- source section id
- NCT03173248-om-0027
- source outcome index
- 27
- instrument
- EORTC QLQ
- instrument basis
- matched 'eortc' in the study's own wording
- scale title
- Change From Baseline in the European Organisation for Research and Treatment of Cancer (EORTC) QLC-C30 Questionnaire
- measure definition
- 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.
- time frame
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-acute-myeloid-leukemia-tibsovo-6eb8ff41
- nct id
- NCT03173248
- qol key
- qol-0028
- source section id
- NCT03173248-om-0028
- source outcome index
- 28
- instrument
- EORTC QLQ
- instrument basis
- matched 'eortc' in the study's own wording
- scale title
- Change From Baseline in the EORTC EQ-5D-5L Questionnaire
- measure definition
- 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.
- time frame
- Up to approximately 52 months
- analysis role
- secondary
- reporting status
- NOT_POSTED
- higher is better
- Source null
- direction quote
- Source null
- population note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- setting source links
Preserved source evidence · Not medical advice