Acute Myeloid LeukemiaPhase 1/2NCT02719574

Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation

A Phase 1/2 study testing FT-2102 (olutasidenib), Azacitidine and Cytarabine in people with Acute Myeloid Leukemia and Acute Myelogenous Leukemia, run by Forma Therapeutics, Inc. at 60 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Acute Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome
What is being tested?: FT-2102 (olutasidenib), Azacitidine and Cytarabine
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 336 participants
Where: 60 sitesin 9 countries
Study dates: 30 Apr 2016to 24 Jan 2024
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Pathologically proven acute myeloid leukemia (AML) (except acute promyelocytic leukemia [APL] with the t(15;17) translocation) or intermediate, high-risk, or very high risk Myelodysplastic Syndrome (MDS) as defined by the World Health Organization (WHO) criteria or Revised International Prognostic Scoring System (IPSS-R) which is relapsed or refractory (R/R) to standard therapy and/or for which standard therapy is contraindicated or which has not adequately responded to standard therapy.
Patients must have documented IDH1-R132 gene-mutated disease as evaluated by the site
Good performance status
Good kidney and liver function

Who cannot join

Patients with symptomatic central nervous system (CNS) metastases or other tumor location (such as spinal cord compression, other compressive mass, uncontrolled painful lesion, bone fracture, etc.) necessitating an urgent therapeutic intervention, palliative care, surgery or radiation therapy
Congestive heart failure (New York Heart Association Class III or IV) or unstable angina pectoris. Previous history of myocardial infarction within 1 year prior to study entry, uncontrolled hypertension or uncontrolled arrhythmias
Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy

Trial locations and contacts

60 sites in 9 countries.

Country

City, postcode or hospital

60 sites in 9 countries

United States19 sites

Emory Winship Cancer Institute
Atlanta, Georgia, 30322
University of Maryland Greenebaum Cancer Center
Baltimore, Maryland, 21201
Roswell Park Cancer Institute
Buffalo, New York, 14263
Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611
The Ohio State University
Columbus, Ohio, 43210
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Karmanos Cancer Institute
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27705
New York Medical College
Hawthorne, New York, 10532
MD Anderson Cancer Center
Houston, Texas, 77030
UCLA Medical Center
Los Angeles, California, 90024
University of Miami
Miami, Florida, 33136
Sarah Cannon Research Institute - Tennessee Oncology
Nashville, Tennessee, 37203
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Yale University
New Haven, Connecticut, 06510
Columbia University Medical Center
New York, New York, 10032
Cornell University Weill Medical College
New York, New York, 10065
Oregon Health & Science University
Portland, Oregon, 97229
UC Davis Comprehensive Cancer Center
Sacramento, California, 95817

Other trials in United States

France11 sites

Service d'Hématologie Clinique, Hôpital Avicenne-APHP-Université Paris
Bobigny, 93000
Assistance Publique Hopitaux de Marseille (AP-HM) - Hopital Nord
Marseille, 13005
Centre Hospitalier Universitaire Nantes
Nantes, 44093
Hôpital Saint-Louis
Paris, 75010
Hopitaux Universitaires Est Parisien Hopital Saint-Antoine
Paris, 75012
Centre Hospitalier Universitaire (CHU) Bordeaux - Hospitaux du Haut Leveque
Pessac, 33604
Centre Hospitalier Lyon Sud
Pierre-Bénite, 69495
University Hospital of Rennes
Rennes, 35033
Institut Universitaire du Cancer Toulouse - Oncopole
Toulouse, 31059
Centre Hospitalier Universitaire de Nancy - Hopital Brabois
Vandœuvre-lès-Nancy, 54511
Institut de Cancérologie Gustave Roussy
Villejuif, 94805

Other trials in France

Italy7 sites

Ospedale Mazzoni - UOC Ematologia Ascoli Piceno
Ascoli Piceno
Universita di Bologna
Bologna
Dipartimento di Oncologia Medica - IRST IRCC
Meldola
AOU S. Luigi Gonzaga - Orbassano
Orbassano, Turin
Università degli Studi di Parma
Parma
U.O. Ematologia Ravenna
Ravenna
Hospital Rimini Hematology, Department of Oncology and Hematoloy
Rimini

Other trials in Italy

Spain6 sites

Hospital Vall d'Hebron
Barcelona, 08035
Hospital Clinic de Barcelona
Barcelona, 8036
Institut Català d'Oncologia-Hospital Duran i Reynals
Barcelona, 8908
Hospital Universitario 12 De Octubre
Madrid, 28041
Hospital Clínico Universitario de Salamanca
Salamanca, 37007
Hospital La Fe
Valencia, 46026

Other trials in Spain

Australia5 sites

Royal Adelaide Hospital
Adelaide, South Australia, 5000
Box Hill Hospital, Monash University and Eastern Health Clinical School
Box Hill, 3128
The Alfred Hospital
Melbourne, Victoria, 3004
Sir Charles Gairdner Hospital
Nedlands, Western Australia, 6009
Victoria Cancer Care Center
Parkville, Victoria, 3000

Other trials in Australia

United Kingdom5 sites

University College London Hospitals NHS Foundation Trust
London, NW1 2PG
St. George's University Hospital
London
Churchill Hospital
Oxford
Southampton General Hospital
Southampton
Royal Marsden Hospital
Sutton, SM2 5PT

Other trials in United Kingdom

Germany4 sites

Staedtisches Klinikum Braunschweig gGmbH
Braunschweig
Universitaetsklinikum Giessen und Marburg GmbH - Klinik fuer Innere Medizin
Giessen
Landeszentrum fuer Zell- und Gentherapie
Halle
Universitätsklinikum Münster Medizinische Klinik A, Hämatologie, Hämostaseologi
Münster

Other trials in Germany

South Korea2 sites

Seoul National University Bundang Hospital
Gumi, 13620
Seoul National University Hospital
Seoul, 03080

Other trials in South Korea

Canada1 site

Princess Margaret Hospital
Toronto, Ontario, M5G 2M9

Other trials in Canada

About this study

Official title: A Phase 1/2, Multicenter, Open-label Study of FT-2102 as a Single Agent and in Combination With Azacitidine or Cytarabine in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome With an IDH1 Mutation

This Phase 1/2 study will evaluate the safety, efficacy, PK, and PD of FT-2102 (olutasidenib) as a single agent or in combination with azacitidine or cytarabine. The Phase 1 stage of the study is split into 2 distinct parts: a dose escalation part, which will utilize an open-label design of FT-2102 (olutasidenib) (single agent) and FT-2102 (olutasidenib) + azacitidine (combination agent) administered via one or more intermittent dosing schedules followed by a dose expansion part. The dose expansion part will enroll patients in up to 5 expansion cohorts, exploring single-agent FT-2102 (olutasidenib) activity as well as combination activity with azacitidine or cytarabine. Following the completion of the relevant Phase 1 cohorts, Phase 2 will begin enrollment. Patients will be enrolled across 8 different cohorts, examining the effect of FT-2102 (olutasidenib) (as a single agent) and FT-2102 (olutasidenib) + azacitidine (combination) on various AML/MDS disease states.

Conditions: Acute Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Being tested: FT-2102 (olutasidenib), Azacitidine, Cytarabine
Keywords: AML, MDS, IDH1, IDH
Medical subject headings: Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

FT-2102 (olutasidenib)Drug
FT-2102 (olutasidenib) will be supplied as 50 mg or 150 mg capsules and will be administered per the protocol defined frequency and dose level
AzacitidineDrug
Also called Vidaza
azacitidine will be administered per site's standard of care
CytarabineDrug
low-dose cytarabine will be administered per site's standard of care

Study groups in NCT02719574
Study group	Role	What they receive
PH1 Dose Escalation & Expansion FT-2102 (olutasidenib)	Experimental	Not described Receives: Drug: FT-2102 (olutasidenib)
PH1 Esc. and Exp. FT-2102 (olutasidenib)+Azacitidine	Experimental	Not described Receives: Drug: FT-2102 (olutasidenib); Drug: Azacitidine
PH1 Esc. and Exp. FT-2102 (olutasidenib)+Cytarabine	Experimental	Not described Receives: Drug: FT-2102 (olutasidenib); Drug: Cytarabine
PH2 Cohort 1 FT-2102 (olutasidenib) Single Agent	Experimental	Relapsed or Refractory (R/R) AML Receives: Drug: FT-2102 (olutasidenib)
PH2 Cohort 2 FT-2102 (olutasidenib) Single Agent	Experimental	AML in morphologic complete remission or complete remission with incomplete blood count recovery (CR/CRi) after prior therapy with residual IDH1-R132 mutation Receives: Drug: FT-2102 (olutasidenib)
PH2 Cohort 3 FT-2102 (olutasidenib) Single Agent	Experimental	R/R AML/MDS, previously treated with FT-2102 Receives: Drug: FT-2102 (olutasidenib)
PH2 Cohort 4 FT-2102 (olutasidenib)+Azacitidine	Experimental	R/R AML/MDS that are naïve to prior hypomethylating therapy and IDH1 inhibitor therapy Receives: Drug: FT-2102 (olutasidenib); Drug: Azacitidine
PH2 Cohort 5 FT-2102 (olutasidenib)+Azacitidine	Experimental	R/R AML/MDS that have inadequately responded to or have progressed on prior hypomethylating therapy Receives: Drug: FT-2102 (olutasidenib); Drug: Azacitidine
PH2 Cohort 6 FT-2102 (olutasidenib)+Azacitidine	Experimental	R/R AML/MDS that have been previously treated with single-agent FT-2102 as their last therapy prior to study enrollment Receives: Drug: FT-2102 (olutasidenib); Drug: Azacitidine
PH2 Cohort 7 FT-2102 (olutasidenib) Single Agent	Experimental	Treatment naïve AML for whom standard treatments are contraindicated Receives: Drug: FT-2102 (olutasidenib)
PH2 Cohort 8 FT-2102 (olutasidenib)+Azacitidine	Experimental	Treatment naïve AML who are candidates for azacitidine first line treatment Receives: Drug: FT-2102 (olutasidenib); Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
A TEAE was defined as an adverse event (AE) that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state. An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. A serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. Number of participants with TEAEs and SAEs is reported. Safety analysis set (SAS) included all the participants who have received at least one dose of study drug (FT-2102, azacitidine, or cytarabine).
Measured: Phase 1: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)
Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values
Number of participants with change from baseline in clinically significant abnormal laboratory values for hematology, chemistry and coagulation with Grade <1 to 4 is reported. National Cancer Institute - Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 toxicity grade used to determine severity of AE. Grade 1: mild;asymptomatic/mild symptoms; Grade 2: moderate; minimal; Grade 3: severe/medically significant; Grade 4: life-threatening consequences, Grade 5: death.
Measured: Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)
Number of participants with change from baseline in clinically significant abnormal ECG parameter (QTcF) is reported. QT interval was the time between the start of the Q wave and the end of the T wave in the cardiac electrical cycle. QTcF was the QT interval corrected for heart rate using Fridericia's formula: QTcF = QT divided by cube root of 60/heart rate.
Measured: Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Phase 2, Cohort 1: Percentage of Participants With Complete Remission (CR) Plus Complete Remission With Partial Hematological Recovery (CRh) for Acute Myeloid Leukemia Assessed by Investigator Based on International Working Group (IWG) Response Criteria
Percentage of participants with CR plus CRh (CR, Molecular CR [CRm], Cytogenetic CR [CRc], CRh) is re-ported. CR is defined as bone marrow blasts less than (<) 5 percent (%) (in aspirate with spicules and 200 nucleated cells), no blasts with auer rods, no extramedullary disease, absolute neutrophil count (ANC) greater than or equal to (>=) 1000/μL, platelet count >=100,000/μL and transfusion independ-ence. CRc is defined as CR with no residual cytogenetic abnormalities. CRm is defined as CR with unde-tectable IDH1m minimal residual disease (MRD) and CRh is defined as bone marrow blasts and partial recovery of peripheral blood counts (platelet count >50 × 10^9/L and ANC > 0.5 × 10^9/L).
Measured: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months)
Phase 2, Cohort 3, 4, 5, 6, 7, 8: Percentage of Participants With CR Plus CRh for Acute Myeloid Leukemia Assessed by Investigator Based on IWG Response Criteria
Percentage of participants with CR plus CRh (CR, Molecular CR [CRm]), Cytogenetic CR [CRc], CRh) is re-ported. CR is defined as bone marrow blasts <5 % (in aspirate with spicules and 200 nucleated cells), no blasts with auer rods, no extramedullary disease, ANC >=1000/μL, platelet count >=100,000/μL and transfusion independ-ence. CRc is defined as CR with no residual cytogenetic abnormalities. CRm is de-fined as CR with undetectable IDH1m MRD and CRh is defined as bone marrow blasts and partial recovery of peripheral blood counts (platelet count >50 × 10^9/L and ANC > 0.5 × 10^9/L).
Measured: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months)
Phase 2, Cohort 4 and 5: Percentage of Participants With CR for MDS Assessed by IWG Response Criteria
Percentage of participants with complete remission (CR) is reported. CR is defined as bone marrow blast ≤ 5% myeloblasts with normal maturation of all cell lines, peripheral blood: Hgb ≥11 grams per deciliter (g/dL), platelets ≥ 100 × 10^9/L, neutrophils ≥ 1.0 × 10^9/L and blasts 0%.
Measured: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months)
Phase 2, Cohort 2: Four-month Relapse Free Survival (RFS) Rate
RFS is defined as the time between the date of first dose until relapse or death from any cause, whichever occurs first. RFS is calculated for all participants in Phase 2 Cohort 2. 4-Month RFS rate is defined as the percentage of participants in Phase 2 Cohort 2 who have not relapsed or died on or before their 4-month response evaluation.
Measured: Phase 2: From the date of first dose until relapse or death from any cause, whichever occurs first (up to 4 months)

Secondary outcomes (20)

Phase 1: Area Under the Curve (AUClast) for FT-2102
Area under the plasma concentration-time curve from zero time until the last measurable concentration is presented.
Measured: Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 1: Maximum Plasma Concentration (Cmax) for FT-2102
Maximum plasma concentration (Cmax) for FT-2102 is presented.
Measured: Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 1: Time to Maximum Plasma Concentration (Tmax) for FT-2102
Time to reach the maximum plasma concentration (Tmax) is presented.
Measured: Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 1: Time to Response (TTR) for AML
TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.
Measured: Phase 1: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 1: Duration of Overall Response for AML
Duration of response is defined as the time from the date of the first response to the date of the relapse or death. The estimation of median duration of overall response was done by Kaplan-Meier method.
Measured: Phase 1: From date of the first response to the date of the relapse or death (up to 82 months)
Phase 1: Time to Response (TTR ) for MDS
TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.
Measured: Phase 1: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 2: Time to Response (TTR) for Acute Myeloid Leukemia (AML)
TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.
Measured: Phase 2: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 2: Duration of Overall Response for Acute Myeloid Leukemia (AML)
Duration of overall response is defined as the time from the date of the first response to the date of the relapse or death. The estimation of median duration of overall response was done by Kaplan-Meier method.
Measured: Phase 2: From the date of the first response to the date of the relapse or death (up to 82 months)
Phase 2, Cohort 4 and Cohort 5: Time to Response (TTR) for Myelodysplastic Syndrome (MDS)
TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.
Measured: Phase 2: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 2, Cohort 4 and Cohort 5: Duration of Overall Response for Myelodysplastic Syndrome (MDS)
Duration of overall response was calculated similarly to DCR but was assessed the time in months between documentation of the first response of PR or better until the date of relapse, death, whichever is earlier. The estimation of median duration of overall response was done by Kaplan-Meier method. The median duration of overall response is defined as the time at which 50% of the participants in a study have experienced the event of interest (relapse or death). Given the presence of only a single participant in the sample, there is an absence of variability in outcomes, and therefore, it was not possible to ascertain a time point at which half of the participants have experienced the event. Hence, median value could not be calculated.
Measured: Phase 2: From the documentation of the first response of PR or better until the date of relapse, death, whichever is earlier (up to 82 months)
Phase 2, Cohort 2: Time to Relapse-Free Survival (RFS)
RFS was calculated for all participants in Phase 2 Cohort 2. RFS is defined as the time (in months) between the date of first dose until relapse or death from any cause, whichever occurs first. Relapse free survival time to event is reported for all participants in Cohort 2.
Measured: Phase 2: From the date of first dose until relapse or death from any cause, whichever occurs first (up to 82 months)
Phase 2: Overall Survival (OS)
OS is defined as the time in months from the first dose of study drug until death from any cause. For the participants who are not known to have died by the end of study follow-up, OS will be censored on the date the participants was last known to be alive. OS is calculated using Kaplan-Meier method.
Measured: Phase 2: From first dose of study drug until death from any cause (up to 82 months)
Phase 2: Event-free Survival (EFS)
EFS is defined as the time in months between first dose of study drug and disease progression, relapse, death from any cause, treatment failure, or start of other (non-protocol study drug) new antileukemia therapy, whichever occurs first.
Measured: Phase 2: From first dose of study drug to disease progression, relapse, death from any cause, treatment failure, or start of other (non-protocol study drug) new antileukemia therapy (up to 82 months)
Phase 2: Transfusion Independence
Transfusion independence is defined as the number of participants who experience at least a 56-day period during any point on treatment without requiring a transfusion of RBC and/or platelet transfusion. Participants are classified as either "dependent" or "independent" at baseline based on their transfusion history. Those who received either platelets or pRBC or both within 8 weeks prior to the first dose of FT-2102 are considered "dependent". Number of participants with transfusion independent is reported here.
Measured: Phase 2: From baseline (8 weeks prior to first dose) up to treatment period (up to 82 months)
Phase 2: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
A TEAE was defined as an AE that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state. An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. A SAE is defined as any untoward medical occur-rence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. Number of participants with TEAEs and SAEs is reported.
Measured: Phase 2: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)
Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values
Number of participants with change from baseline in clinically significant abnormal laboratory values for hematology, chemistry and coagulation with Grade <1 to 4 is reported. National Cancer Institute - Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 toxicity grade used to determine severity of AE. Grade 1: mild;asymptomatic/mild symptoms; Grade 2: moderate; minimal; Grade 3: severe/medically significant; Grade 4: life-threatening consequences, Grade 5: death.
Measured: Phase 2: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)
Number of participants with change from baseline in clinically significant abnormal ECG parameter (QTcF) is reported. QT interval was the time between the start of the Q wave and the end of the T wave in the cardiac electrical cycle. QTcF was the QT interval corrected for heart rate using Fridericia's formula: QTcF = QT divided by cube root of 60/heart rate.
Measured: Phase 2: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Phase 2: Area Under the Curve (AUClast) for FT-2102
Area under the plasma concentration-time curve from zero time until the last measurable concentration is presented.
Measured: Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 2: Maximum Plasma Concentration (Cmax) for FT-2102
Maximum Plasma Concentartion (Cmax) for FT-2102 is presented.
Measured: Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 2: Time to Maximum Plasma Concentration (Tmax) for FT-2102
Time to reach the maximum plasma concentration (Tmax) for FT-2102 is presented.
Measured: Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

30 Apr 201624 Jan 2024

First posted25 Mar 2016 (estimated)
Study start30 Apr 2016
Primary completion28 Dec 2023
Study completion24 Jan 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 336 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02719574
Sponsor's protocol ID: 2102-HEM-101
Status last verified: Jun 2026
First submitted: 21 Mar 2016
Last updated: 29 Jun 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Cortes J, Curti A, Fenaux P, Jonas BA, Krauter J, Montesinos P, Recher C, Taussig DC, Wang ES, Watts J, Wei A, Yee KW, Tian H, Sheppard A, Marzac C, de Botton S. Olutasidenib for mutated IDH1 acute myeloid leukemia: final five-year results from the phase 2 pivotal cohort. J Hematol Oncol. 2025 Nov 14;18(1):102. doi: 10.1186/s13045-025-01751-w.
PubMed 41239466
Cortes JE, Yang J, Roboz GJ, Dinner SN, Wang ES, Wei AH, Tian H, di Trapani F, Baer MR, Donnellan W, Watts JM. Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome. Blood Adv. 2025 Oct 28;9(20):5293-5305. doi: 10.1182/bloodadvances.2025016718.
PubMed 40668616
Cortes JE, Roboz GJ, Baer MR, Jonas BA, Schiller GJ, Yee K, Ferrell PB, Yang J, Wang ES, Blum WG, Mims A, Tian H, Sheppard A, de Botton S, Montesinos P, Curti A, Watts JM; Olutasidenib Combination Therapy Study Group. Olutasidenib in combination with azacitidine induces durable complete remissions in patients with relapsed or refractory mIDH1 acute myeloid leukemia: a multicohort open-label phase 1/2 trial. J Hematol Oncol. 2025 Jan 16;18(1):7. doi: 10.1186/s13045-024-01657-z.
PubMed 39819505
de Botton S, Fenaux P, Yee K, Recher C, Wei AH, Montesinos P, Taussig DC, Pigneux A, Braun T, Curti A, Grove C, Jonas BA, Khwaja A, Legrand O, Peterlin P, Arnan M, Blum W, Cilloni D, Hiwase DK, Jurcic JG, Krauter J, Thomas X, Watts JM, Yang J, Polyanskaya O, Brevard J, Sweeney J, Barrett E, Cortes J. Olutasidenib (FT-2102) induces durable complete remissions in patients with relapsed or refractory IDH1-mutated AML. Blood Adv. 2023 Jul 11;7(13):3117-3127. doi: 10.1182/bloodadvances.2022009411.
PubMed 36724515
Watts JM, Baer MR, Yang J, Prebet T, Lee S, Schiller GJ, Dinner SN, Pigneux A, Montesinos P, Wang ES, Seiter KP, Wei AH, De Botton S, Arnan M, Donnellan W, Schwarer AP, Recher C, Jonas BA, Ferrell PB Jr, Marzac C, Kelly P, Sweeney J, Forsyth S, Guichard SM, Brevard J, Henrick P, Mohamed H, Cortes JE. Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial. Lancet Haematol. 2023 Jan;10(1):e46-e58. doi: 10.1016/S2352-3026(22)00292-7. Epub 2022 Nov 10.
PubMed 36370742

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All Acute Myeloid Leukemia trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2, Multicenter, Open-label Study of FT-2102 as a Single Agent and in Combination With Azacitidine or Cytarabine in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome With an IDH1 Mutation
ClinicalTrials.gov ID: NCT02719574
Acronym / sponsor ID: 2102-HEM-101
Conditions: Acute Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Keywords: AML, MDS, IDH1, IDH
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 336 participants (actual)
Lead sponsor: Forma Therapeutics, Inc.Industry (pharma, biotech, devices)
Study start: 30 Apr 2016 (actual)
Primary completion: 28 Dec 2023 (actual)
Study completion: 24 Jan 2024 (actual)
First posted: 25 Mar 2016 (estimated)
Results: Posted 26 Jun 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 11

Arm	Type	Receives
PH1 Dose Escalation & Expansion FT-2102 (olutasidenib)	Experimental	Drug: FT-2102 (olutasidenib)
PH1 Esc. and Exp. FT-2102 (olutasidenib)+Azacitidine	Experimental	Drug: FT-2102 (olutasidenib), Drug: Azacitidine
PH1 Esc. and Exp. FT-2102 (olutasidenib)+Cytarabine	Experimental	Drug: FT-2102 (olutasidenib), Drug: Cytarabine
PH2 Cohort 1 FT-2102 (olutasidenib) Single Agent	Experimental	Drug: FT-2102 (olutasidenib)Relapsed or Refractory (R/R) AML
PH2 Cohort 2 FT-2102 (olutasidenib) Single Agent	Experimental	Drug: FT-2102 (olutasidenib)AML in morphologic complete remission or complete remission with incomplete blood count recovery (CR/CRi) after prior therapy with residual IDH1-R132 mutation
PH2 Cohort 3 FT-2102 (olutasidenib) Single Agent	Experimental	Drug: FT-2102 (olutasidenib)R/R AML/MDS, previously treated with FT-2102
PH2 Cohort 4 FT-2102 (olutasidenib)+Azacitidine	Experimental	Drug: FT-2102 (olutasidenib), Drug: AzacitidineR/R AML/MDS that are naïve to prior hypomethylating therapy and IDH1 inhibitor therapy
PH2 Cohort 5 FT-2102 (olutasidenib)+Azacitidine	Experimental	Drug: FT-2102 (olutasidenib), Drug: AzacitidineR/R AML/MDS that have inadequately responded to or have progressed on prior hypomethylating therapy
PH2 Cohort 6 FT-2102 (olutasidenib)+Azacitidine	Experimental	Drug: FT-2102 (olutasidenib), Drug: AzacitidineR/R AML/MDS that have been previously treated with single-agent FT-2102 as their last therapy prior to study enrollment
PH2 Cohort 7 FT-2102 (olutasidenib) Single Agent	Experimental	Drug: FT-2102 (olutasidenib)Treatment naïve AML for whom standard treatments are contraindicated
PH2 Cohort 8 FT-2102 (olutasidenib)+Azacitidine	Experimental	Drug: FT-2102 (olutasidenib), Drug: AzacitidineTreatment naïve AML who are candidates for azacitidine first line treatment

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Pathologically proven acute myeloid leukemia (AML) (except acute promyelocytic leukemia [APL] with the t(15;17) translocation) or intermediate, high-risk, or very high risk Myelodysplastic Syndrome (MDS) as defined by the World Health Organization (WHO) criteria or Revised International Prognostic Scoring System (IPSS-R) which is relapsed or refractory (R/R) to standard therapy and/or for which standard therapy is contraindicated or which has not adequately responded to standard therapy.
Patients must have documented IDH1-R132 gene-mutated disease as evaluated by the site
Good performance status
Good kidney and liver function

Key exclusion criteria (3 listed)

Patients with symptomatic central nervous system (CNS) metastases or other tumor location (such as spinal cord compression, other compressive mass, uncontrolled painful lesion, bone fracture, etc.) necessitating an urgent therapeutic intervention, palliative care, surgery or radiation therapy
Congestive heart failure (New York Heart Association Class III or IV) or unstable angina pectoris. Previous history of myocardial infarction within 1 year prior to study entry, uncontrolled hypertension or uncontrolled arrhythmias
Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
FT-2102 (olutasidenib)FT-2102 (olutasidenib) will be supplied as 50 mg or 150 mg capsules and will be administered per the protocol defined frequency and dose level	Drug	PH1 Dose Escalation & Expansion FT-2102 (olutasidenib), PH1 Esc. and Exp. FT-2102 (olutasidenib)+Azacitidine, PH1 Esc. and Exp. FT-2102 (olutasidenib)+Cytarabine, PH2 Cohort 1 FT-2102 (olutasidenib) Single Agent, PH2 Cohort 2 FT-2102 (olutasidenib) Single Agent, PH2 Cohort 3 FT-2102 (olutasidenib) Single Agent, PH2 Cohort 4 FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 5 FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 6 FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 7 FT-2102 (olutasidenib) Single Agent, PH2 Cohort 8 FT-2102 (olutasidenib)+Azacitidine
AzacitidineAlso: Vidazaazacitidine will be administered per site's standard of care	Drug	PH1 Esc. and Exp. FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 4 FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 5 FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 6 FT-2102 (olutasidenib)+Azacitidine, PH2 Cohort 8 FT-2102 (olutasidenib)+Azacitidine
Cytarabinelow-dose cytarabine will be administered per site's standard of care	Drug	PH1 Esc. and Exp. FT-2102 (olutasidenib)+Cytarabine

Primary outcome

Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Phase 1: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)
A TEAE was defined as an adverse event (AE) that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state. An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. A serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. Number of participants with TEAEs and SAEs is reported. Safety analysis set (SAS) included all the participants who have received at least one dose of study drug (FT-2102, azacitidine, or cytarabine).
Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values
Time frame: Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Number of participants with change from baseline in clinically significant abnormal laboratory values for hematology, chemistry and coagulation with Grade <1 to 4 is reported. National Cancer Institute - Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 toxicity grade used to determine severity of AE. Grade 1: mild;asymptomatic/mild symptoms; Grade 2: moderate; minimal; Grade 3: severe/medically significant; Grade 4: life-threatening consequences, Grade 5: death.
Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)
Time frame: Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Number of participants with change from baseline in clinically significant abnormal ECG parameter (QTcF) is reported. QT interval was the time between the start of the Q wave and the end of the T wave in the cardiac electrical cycle. QTcF was the QT interval corrected for heart rate using Fridericia's formula: QTcF = QT divided by cube root of 60/heart rate.
Phase 2, Cohort 1: Percentage of Participants With Complete Remission (CR) Plus Complete Remission With Partial Hematological Recovery (CRh) for Acute Myeloid Leukemia Assessed by Investigator Based on International Working Group (IWG) Response Criteria
Time frame: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months)
Percentage of participants with CR plus CRh (CR, Molecular CR [CRm], Cytogenetic CR [CRc], CRh) is re-ported. CR is defined as bone marrow blasts less than (<) 5 percent (%) (in aspirate with spicules and 200 nucleated cells), no blasts with auer rods, no extramedullary disease, absolute neutrophil count (ANC) greater than or equal to (>=) 1000/μL, platelet count >=100,000/μL and transfusion independ-ence. CRc is defined as CR with no residual cytogenetic abnormalities. CRm is defined as CR with unde-tectable IDH1m minimal residual disease (MRD) and CRh is defined as bone marrow blasts and partial recovery of peripheral blood counts (platelet count >50 × 10^9/L and ANC > 0.5 × 10^9/L).
Phase 2, Cohort 3, 4, 5, 6, 7, 8: Percentage of Participants With CR Plus CRh for Acute Myeloid Leukemia Assessed by Investigator Based on IWG Response Criteria
Time frame: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months)
Percentage of participants with CR plus CRh (CR, Molecular CR [CRm]), Cytogenetic CR [CRc], CRh) is re-ported. CR is defined as bone marrow blasts <5 % (in aspirate with spicules and 200 nucleated cells), no blasts with auer rods, no extramedullary disease, ANC >=1000/μL, platelet count >=100,000/μL and transfusion independ-ence. CRc is defined as CR with no residual cytogenetic abnormalities. CRm is de-fined as CR with undetectable IDH1m MRD and CRh is defined as bone marrow blasts and partial recovery of peripheral blood counts (platelet count >50 × 10^9/L and ANC > 0.5 × 10^9/L).
Phase 2, Cohort 4 and 5: Percentage of Participants With CR for MDS Assessed by IWG Response Criteria
Time frame: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months)
Percentage of participants with complete remission (CR) is reported. CR is defined as bone marrow blast ≤ 5% myeloblasts with normal maturation of all cell lines, peripheral blood: Hgb ≥11 grams per deciliter (g/dL), platelets ≥ 100 × 10^9/L, neutrophils ≥ 1.0 × 10^9/L and blasts 0%.
Phase 2, Cohort 2: Four-month Relapse Free Survival (RFS) Rate
Time frame: Phase 2: From the date of first dose until relapse or death from any cause, whichever occurs first (up to 4 months)
RFS is defined as the time between the date of first dose until relapse or death from any cause, whichever occurs first. RFS is calculated for all participants in Phase 2 Cohort 2. 4-Month RFS rate is defined as the percentage of participants in Phase 2 Cohort 2 who have not relapsed or died on or before their 4-month response evaluation.

20 secondary outcomes

Phase 1: Area Under the Curve (AUClast) for FT-2102
Time frame: Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 1: Maximum Plasma Concentration (Cmax) for FT-2102
Time frame: Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 1: Time to Maximum Plasma Concentration (Tmax) for FT-2102
Time frame: Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 1: Time to Response (TTR) for AML
Time frame: Phase 1: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 1: Duration of Overall Response for AML
Time frame: Phase 1: From date of the first response to the date of the relapse or death (up to 82 months)
Phase 1: Time to Response (TTR ) for MDS
Time frame: Phase 1: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 2: Time to Response (TTR) for Acute Myeloid Leukemia (AML)
Time frame: Phase 2: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 2: Duration of Overall Response for Acute Myeloid Leukemia (AML)
Time frame: Phase 2: From the date of the first response to the date of the relapse or death (up to 82 months)
Phase 2, Cohort 4 and Cohort 5: Time to Response (TTR) for Myelodysplastic Syndrome (MDS)
Time frame: Phase 2: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Phase 2, Cohort 4 and Cohort 5: Duration of Overall Response for Myelodysplastic Syndrome (MDS)
Time frame: Phase 2: From the documentation of the first response of PR or better until the date of relapse, death, whichever is earlier (up to 82 months)
Phase 2, Cohort 2: Time to Relapse-Free Survival (RFS)
Time frame: Phase 2: From the date of first dose until relapse or death from any cause, whichever occurs first (up to 82 months)
Phase 2: Overall Survival (OS)
Time frame: Phase 2: From first dose of study drug until death from any cause (up to 82 months)
Phase 2: Event-free Survival (EFS)
Time frame: Phase 2: From first dose of study drug to disease progression, relapse, death from any cause, treatment failure, or start of other (non-protocol study drug) new antileukemia therapy (up to 82 months)
Phase 2: Transfusion Independence
Time frame: Phase 2: From baseline (8 weeks prior to first dose) up to treatment period (up to 82 months)
Phase 2: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Phase 2: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)
Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values
Time frame: Phase 2: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)
Time frame: Phase 2: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Phase 2: Area Under the Curve (AUClast) for FT-2102
Time frame: Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 2: Maximum Plasma Concentration (Cmax) for FT-2102
Time frame: Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Phase 2: Time to Maximum Plasma Concentration (Tmax) for FT-2102
Time frame: Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)

Results and safety

Results posted to the registry on 26 Jun 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Phase 1: FT-2102 100mg QD (Single Agent; Dose Escalation)	3	0
Phase 1: FT-2102 150mg QD (Single Agent; Dose Escalation)	8	0
Phase 1: FT-2102 150mg BID (Single Agent; Dose Escalation)	7	0
Phase 1: FT-2102 300mg QD (Single Agent; Dose Escalation)	4	0
Phase 1: FT-2102 150mg QD+Azacitidine (Combination Therapy; Dose Escalation)	7	0
Phase 1: FT-2102 150mg BID+Azacitidine (Combination Therapy; Dose Escalation)	8	0
Phase 1: FT-2102 150mg BID (Single Agent; Dose Expansion)	0	0
Phase 1: FT-2102 150mg BID+Azacitidine (Combination Therapy; Dose Expansion)	0	0
Phase 1: FT-2102 150mg BID+ Cytarabine (Combination Therapy; Dose Expansion)	0	0
Phase 2: Cohort 1; FT-2102 (Single Agent)	0	0
Phase 2: Cohort 2; FT-2102 (Single Agent)	0	0
Phase 2: Cohort 3; FT-2102 (Single Agent)	0	0
Phase 2: Cohort 4; FT-2102 + Azacitidine (Combination Therapy)	0	0
Phase 2: Cohort 5; FT-2102 + Azacitidine (Combination Therapy)	0	0
Phase 2: Cohort 6; FT-2102 + Azacitidine (Combination Therapy)	0	0
Phase 2: Cohort 7; FT-2102 (Single Agent)	0	0
Phase 2: Cohort 8; FT-2102 + Azacitidine (Combination Therapy)	0	0

Primary outcome: Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time frame: Phase 1: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months). Count of participants. Unit: Participants

Measure	Phase 1: FT-2102 (Single Agent) n = 16	Phase 1: Total FT-2102 (Single Agent) n = 31	Phase 1: FT-2102 + Azacitidine (Combination Therapy) n = 39	Phase 1: Total FT-2102; FT-2102 + Azacitidine (Combination Therapy) n = 46
TEAEs	16	31	39	46
SAEs	10	23	30	36

Population analysed: SAS analyzed. Since 150 mg BID has been established as MTD, the analysis was directed towards emphasizing findings that are most relevant for determining a safe and effective treatment strategy. Accordingly, data for the other dose levels (100 mg QD, 150 mg QD, and 300 mg QD) were combined as prespecified in statistical analysis plan (SAP).

Primary outcome: Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values

Time frame: Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months). Count of participants. Unit: Participants

Measure	Phase 1: FT-2102 (Single Agent) n = 16	Phase 1: Total FT-2102 (Single Agent) n = 31	Phase 1: FT-2102 + Azacitidine (Combination Therapy) n = 39	Phase 1: Total FT-2102; FT-2102 + Azacitidine (Combination Therapy) n = 46
Hematology: Hemoglobin; Increase(Grade <1)	15	30	38	45
Hematology: Hemoglobin; Increase (Grade 1)	0	0	0	0
Hematology: Hemoglobin; Increase (Grade 2)	0	1	1	1
Hematology: Hemoglobin; Increase (Grade 3)	0	0	0	0
Hematology: Hemoglobin; Increase (Grade 4)	0	0	0	0
Hematology: Hemoglobin; Increase (Grade 3 or 4)	0	0	0	0
Hematology: Hemoglobin; Decrease (Grade <1)	0	0	0	0
Hematology: Hemoglobin; Decrease (Grade 1)	3	3	0	0

Primary outcome: Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)

Time frame: Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months). Count of participants. Unit: Participants

Measure	Phase 1: FT-2102 (Single Agent) n = 15	Phase 1: Total FT-2102 (Single Agent) n = 30	Phase 1: FT-2102 + Azacitidine (Combination Therapy) n = 38	Phase 1: Total FT-2102; FT-2102 + Azacitidine (Combination Therapy) n = 45
<=450 msec	10	16	23	26
>450 to <=480 msec	4	11	10	13
>480 to <=500 msec	0	2	1	2
>500 msec	1	1	4	4
>30 to <=60 msec	2	8	13	14
>60 msec	1	2	5	6

Population analysed: SAS. Here, N=Number of participants with available data. Since 150mg BID has been established as MTD, analysis was directed towards emphasizing findings that are most relevant for determining safe and effective treatment strategy. Accordingly, data for other dose levels (100mg QD, 150mg QD, and 300mg QD) were combined as prespecified in SAP.

Primary outcome: Phase 2, Cohort 1: Percentage of Participants With Complete Remission (CR) Plus Complete Remission With Partial Hematological Recovery (CRh) for Acute Myeloid Leukemia Assessed by Investigator Based on International Working Group (IWG) Response Criteria

Time frame: Phase 2: up to 3 weeks post last dose of study drug or until the introduction of new anticancer therapy, whichever is earlier (up to 82 months). Number (95% Confidence Interval). Unit: Percentage of participants

Measure	Phase 2: Cohort 1; FT-2102 (Single Agent) n = 147
Value	35 (27.0 to 43.0)

Population analysed: Efficacy evaluable analysis set included all participants in Phase 2, Cohort 1 with confirmed IDH1-R132 who have received the first dose of FT-2102 180 days or more prior to the analysis cutoff date. This outcome measure is applicable only for Cohort 1.

20 secondary outcomes are reported in the full results.

Adverse events

Time frame: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase 1: FT-2102 (Single Agent)	10 of 16 (62.5%)	15 of 16 (93.8%)	4 of 16
Phase 1: Total FT-2102 (Single Agent)	23 of 31 (74.2%)	29 of 31 (93.5%)	11 of 31
Phase 1: FT-2102 + Azacitidine (Combination Therapy)	30 of 39 (76.9%)	39 of 39 (100%)	9 of 39
Phase 1: Total FT-2102; FT-2102 + Azacitidine (Combination Therapy)	36 of 46 (78.3%)	46 of 46 (100%)	10 of 46
Phase 1: Overall	60 of 78 (76.9%)	76 of 78 (97.4%)	21 of 78
Phase 2: Cohort 1; FT-2102 (Single Agent)	115 of 153 (75.2%)	152 of 153 (99.3%)	50 of 153
Phase 2: Cohort 2; FT-2102 (Single Agent)	6 of 18 (33.3%)	18 of 18 (100%)	0 of 18
Phase 2: Cohort 3; FT-2102 (Single Agent)	4 of 5 (80%)	5 of 5 (100%)	2 of 5
Phase 2: Cohort 4; FT-2102 + Azacitidine (Combination Therapy)	12 of 20 (60%)	18 of 20 (90%)	3 of 20
Phase 2: Cohort 5; FT-2102 + Azacitidine (Combination Therapy)	18 of 21 (85.7%)	20 of 21 (95.2%)	6 of 21
Phase 2: Cohort 6; FT-2102 + Azacitidine (Combination Therapy)	13 of 20 (65%)	19 of 20 (95%)	7 of 20
Phase 2: Cohort 7; FT-2102 (Single Agent)	8 of 10 (80%)	10 of 10 (100%)	4 of 10
Phase 2: Cohort 8; FT-2102 + Azacitidine (Combination Therapy)	9 of 11 (81.8%)	11 of 11 (100%)	2 of 11

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 41239466Cortes J, Curti A, Fenaux P, Jonas BA, Krauter J, Montesinos P, Recher C, Taussig DC, Wang ES, Watts J, Wei A, Yee KW, Tian H, Sheppard A, Marzac C, de Botton S. Olutasidenib for mutated IDH1 acute myeloid leukemia: final five-year results from the phase 2 pivotal cohort. J Hematol Oncol. 2025 Nov 14;18(1):102. doi: 10.1186/s13045-025-01751-w.
Derived from this study: PubMed 40668616Cortes JE, Yang J, Roboz GJ, Dinner SN, Wang ES, Wei AH, Tian H, di Trapani F, Baer MR, Donnellan W, Watts JM. Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome. Blood Adv. 2025 Oct 28;9(20):5293-5305. doi: 10.1182/bloodadvances.2025016718.
Derived from this study: PubMed 39819505Cortes JE, Roboz GJ, Baer MR, Jonas BA, Schiller GJ, Yee K, Ferrell PB, Yang J, Wang ES, Blum WG, Mims A, Tian H, Sheppard A, de Botton S, Montesinos P, Curti A, Watts JM; Olutasidenib Combination Therapy Study Group. Olutasidenib in combination with azacitidine induces durable complete remissions in patients with relapsed or refractory mIDH1 acute myeloid leukemia: a multicohort open-label phase 1/2 trial. J Hematol Oncol. 2025 Jan 16;18(1):7. doi: 10.1186/s13045-024-01657-z.
Derived from this study: PubMed 36724515de Botton S, Fenaux P, Yee K, Recher C, Wei AH, Montesinos P, Taussig DC, Pigneux A, Braun T, Curti A, Grove C, Jonas BA, Khwaja A, Legrand O, Peterlin P, Arnan M, Blum W, Cilloni D, Hiwase DK, Jurcic JG, Krauter J, Thomas X, Watts JM, Yang J, Polyanskaya O, Brevard J, Sweeney J, Barrett E, Cortes J. Olutasidenib (FT-2102) induces durable complete remissions in patients with relapsed or refractory IDH1-mutated AML. Blood Adv. 2023 Jul 11;7(13):3117-3127. doi: 10.1182/bloodadvances.2022009411.
Derived from this study: PubMed 36370742Watts JM, Baer MR, Yang J, Prebet T, Lee S, Schiller GJ, Dinner SN, Pigneux A, Montesinos P, Wang ES, Seiter KP, Wei AH, De Botton S, Arnan M, Donnellan W, Schwarer AP, Recher C, Jonas BA, Ferrell PB Jr, Marzac C, Kelly P, Sweeney J, Forsyth S, Guichard SM, Brevard J, Henrick P, Mohamed H, Cortes JE. Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial. Lancet Haematol. 2023 Jan;10(1):e46-e58. doi: 10.1016/S2352-3026(22)00292-7. Epub 2022 Nov 10.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Transparency (dept. 2834) study director

Novo Nordisk A/S

60 sites in 9 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 60 sites in United States, France, Italy, Spain, Australia and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Phase 1: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02719574, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jun 2026.