Phase 2
N=37
Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation
Acute Myeloid Leukemia · Acute Myelogenous Leukemia · Myelodysplastic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02719574 ↗Enrolled (actual)
37
Serious AEs
73.5%
Results posted
Jun 2025
Primary outcome: Primary: Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 16; 31; 39; 46 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- FT-2102 (olutasidenib) (Drug); Azacitidine (Drug); Cytarabine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Forma Therapeutics, Inc.
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
16; 31; 39; 46; 10; 23 | — |
| PRIMARY Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values |
15; 30; 38; 45; 0; 0 | — |
| PRIMARY Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG) |
10; 16; 23; 26; 4; 11 | — |
| PRIMARY 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 |
35 | — |
| PRIMARY 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 |
0; 47; 38; 30; 40; 45 | — |
| PRIMARY Phase 2, Cohort 4 and 5: Percentage of Participants With CR for MDS Assessed by IWG Response Criteria |
0; 13 | — |
| PRIMARY Phase 2, Cohort 2: Four-month Relapse Free Survival (RFS) Rate |
83 | — |
| SECONDARY Phase 1: Area Under the Curve (AUClast) for FT-2102 |
5505; 8955; 12100; 2538; 8529; 2288 | — |
| SECONDARY Phase 1: Maximum Plasma Concentration (Cmax) for FT-2102 |
571.5; 535.0; 708.0; 495.5; 543.3; 439.9 | — |
| SECONDARY Phase 1: Time to Maximum Plasma Concentration (Tmax) for FT-2102 |
4.05; 4.00; 23.83; 5.79; 4.02; 3.13 | — |
| SECONDARY Phase 1: Time to Response (TTR) for AML |
1.90; 1.90; 1.90; 1.90 | — |
| SECONDARY Phase 1: Duration of Overall Response for AML |
3.60; 2.90; 22.00; 22.00 | — |
| SECONDARY Phase 1: Time to Response (TTR ) for MDS |
4.65; 4.65; 2.20; 2.20 | — |
| SECONDARY Phase 2: Time to Response (TTR) for Acute Myeloid Leukemia (AML) |
1.90; 1.80; 2.50; 2.50; 1.90; 2.80 | — |
| SECONDARY Phase 2: Duration of Overall Response for Acute Myeloid Leukemia (AML) |
14.80; 20.00; 7.25; 4.65; 10.60; 20.90 | — |
| SECONDARY Phase 2, Cohort 4 and Cohort 5: Time to Response (TTR) for Myelodysplastic Syndrome (MDS) |
3.30; 1.50 | — |
| SECONDARY Phase 2, Cohort 4 and Cohort 5: Duration of Overall Response for Myelodysplastic Syndrome (MDS) |
NA; 11.90 | — |
| SECONDARY Phase 2, Cohort 2: Time to Relapse-Free Survival (RFS) |
18.40 | — |
| SECONDARY Phase 2: Overall Survival (OS) |
11.60; NA; 1.20; 25.20; 11.60; 10.20 | — |
| SECONDARY Phase 2: Event-free Survival (EFS) |
5.50; 18.40; 1.05; 5.70; 7.80; 4.80 | — |
| SECONDARY Phase 2: Transfusion Independence |
72; 15; 0; 12; 13; 14 | — |
| SECONDARY Phase 2: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
153; 18; 5; 19; 21; 19 | — |
| SECONDARY Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values |
146; 18; 5; 19; 20; 20 | — |
| SECONDARY Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG) |
90; 11; 1; 9; 9; 11 | — |
| SECONDARY Phase 2: Area Under the Curve (AUClast) for FT-2102 |
2836; 2657; 1880; 3051; 2031; 1701 | — |
| SECONDARY Phase 2: Maximum Plasma Concentration (Cmax) for FT-2102 |
534.5; 492.4; 388.8; 623.2; 404.1; 411.2 | — |
| SECONDARY Phase 2: Time to Maximum Plasma Concentration (Tmax) for FT-2102 |
4.00; 4.00; 5.00; 4.00; 4.00; 4.00 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT02719574). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.