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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02719574




Registration number
Help: Registration number
NCT02719574
Ethics application status
Help: Ethics application status
Date submitted
Help: Date submitted
21/03/2016
Date registered
Help: Date registered
25/03/2016
Date last updated
Help: Date last updated
29/06/2026

Titles & IDs
Public title
Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation
Scientific 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
Additional trial identifiers [1] 0 0
2102-HEM-101
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Acute Myeloid Leukemia 0 0
Acute Myelogenous Leukemia 0 0
Myelodysplastic Syndrome 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute 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 the intervention or exposure
Treatment: Drugs - FT-2102 (olutasidenib)
Treatment: Drugs - Azacitidine
Treatment: Drugs - Cytarabine

Experimental: PH1 Dose Escalation & Expansion FT-2102 (olutasidenib) -

Experimental: PH1 Esc. and Exp. FT-2102 (olutasidenib)+Azacitidine -

Experimental: PH1 Esc. and Exp. FT-2102 (olutasidenib)+Cytarabine -

Experimental: PH2 Cohort 1 FT-2102 (olutasidenib) Single Agent - Relapsed or Refractory (R/R) AML

Experimental: PH2 Cohort 2 FT-2102 (olutasidenib) Single Agent - AML in morphologic complete remission or complete remission with incomplete blood count recovery (CR/CRi) after prior therapy with residual IDH1-R132 mutation

Experimental: PH2 Cohort 3 FT-2102 (olutasidenib) Single Agent - R/R AML/MDS, previously treated with FT-2102

Experimental: PH2 Cohort 4 FT-2102 (olutasidenib)+Azacitidine - R/R AML/MDS that are naïve to prior hypomethylating therapy and IDH1 inhibitor therapy

Experimental: PH2 Cohort 5 FT-2102 (olutasidenib)+Azacitidine - R/R AML/MDS that have inadequately responded to or have progressed on prior hypomethylating therapy

Experimental: PH2 Cohort 6 FT-2102 (olutasidenib)+Azacitidine - R/R AML/MDS that have been previously treated with single-agent FT-2102 as their last therapy prior to study enrollment

Experimental: PH2 Cohort 7 FT-2102 (olutasidenib) Single Agent - Treatment naïve AML for whom standard treatments are contraindicated

Experimental: PH2 Cohort 8 FT-2102 (olutasidenib)+Azacitidine - Treatment naïve AML who are candidates for azacitidine first line treatment


Treatment: Drugs: 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

Treatment: Drugs: Azacitidine
azacitidine will be administered per site's standard of care

Treatment: Drugs: Cytarabine
low-dose cytarabine will be administered per site's standard of care
Intervention code [1] 0 0
Treatment: Drugs
Description of the comparator or control
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Phase 1: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)
Primary outcome [2] 0 0
Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values
Timepoint [2] 0 0
Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Primary outcome [3] 0 0
Phase 1: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)
Timepoint [3] 0 0
Phase 1: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Primary outcome [4] 0 0
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
Timepoint [4] 0 0
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)
Primary outcome [5] 0 0
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
Timepoint [5] 0 0
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)
Primary outcome [6] 0 0
Phase 2, Cohort 4 and 5: Percentage of Participants With CR for MDS Assessed by IWG Response Criteria
Timepoint [6] 0 0
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)
Primary outcome [7] 0 0
Phase 2, Cohort 2: Four-month Relapse Free Survival (RFS) Rate
Timepoint [7] 0 0
Phase 2: From the date of first dose until relapse or death from any cause, whichever occurs first (up to 4 months)
Secondary outcome [1] 0 0
Phase 1: Area Under the Curve (AUClast) for FT-2102
Timepoint [1] 0 0
Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Secondary outcome [2] 0 0
Phase 1: Maximum Plasma Concentration (Cmax) for FT-2102
Timepoint [2] 0 0
Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Secondary outcome [3] 0 0
Phase 1: Time to Maximum Plasma Concentration (Tmax) for FT-2102
Timepoint [3] 0 0
Phase 1: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Secondary outcome [4] 0 0
Phase 1: Time to Response (TTR) for AML
Timepoint [4] 0 0
Phase 1: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Secondary outcome [5] 0 0
Phase 1: Duration of Overall Response for AML
Timepoint [5] 0 0
Phase 1: From date of the first response to the date of the relapse or death (up to 82 months)
Secondary outcome [6] 0 0
Phase 1: Time to Response (TTR ) for MDS
Timepoint [6] 0 0
Phase 1: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Secondary outcome [7] 0 0
Phase 2: Time to Response (TTR) for Acute Myeloid Leukemia (AML)
Timepoint [7] 0 0
Phase 2: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Secondary outcome [8] 0 0
Phase 2: Duration of Overall Response for Acute Myeloid Leukemia (AML)
Timepoint [8] 0 0
Phase 2: From the date of the first response to the date of the relapse or death (up to 82 months)
Secondary outcome [9] 0 0
Phase 2, Cohort 4 and Cohort 5: Time to Response (TTR) for Myelodysplastic Syndrome (MDS)
Timepoint [9] 0 0
Phase 2: Time from first dose of study drug until documentation of the first overall response (up to 82 months)
Secondary outcome [10] 0 0
Phase 2, Cohort 4 and Cohort 5: Duration of Overall Response for Myelodysplastic Syndrome (MDS)
Timepoint [10] 0 0
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)
Secondary outcome [11] 0 0
Phase 2, Cohort 2: Time to Relapse-Free Survival (RFS)
Timepoint [11] 0 0
Phase 2: From the date of first dose until relapse or death from any cause, whichever occurs first (up to 82 months)
Secondary outcome [12] 0 0
Phase 2: Overall Survival (OS)
Timepoint [12] 0 0
Phase 2: From first dose of study drug until death from any cause (up to 82 months)
Secondary outcome [13] 0 0
Phase 2: Event-free Survival (EFS)
Timepoint [13] 0 0
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)
Secondary outcome [14] 0 0
Phase 2: Transfusion Independence
Timepoint [14] 0 0
Phase 2: From baseline (8 weeks prior to first dose) up to treatment period (up to 82 months)
Secondary outcome [15] 0 0
Phase 2: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [15] 0 0
Phase 2: From start of drug administration (Day 1) up to 28 days after last dose of study drug (up to 82 months)
Secondary outcome [16] 0 0
Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Laboratory Values
Timepoint [16] 0 0
Phase 2: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Secondary outcome [17] 0 0
Phase 2: Number of Participants With Change From Baseline in Clinically Significant Abnormal Electrocardiogram (ECG)
Timepoint [17] 0 0
Phase 2: From Baseline up to 28 days after last dose of study drug (up to 82 months)
Secondary outcome [18] 0 0
Phase 2: Area Under the Curve (AUClast) for FT-2102
Timepoint [18] 0 0
Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Secondary outcome [19] 0 0
Phase 2: Maximum Plasma Concentration (Cmax) for FT-2102
Timepoint [19] 0 0
Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)
Secondary outcome [20] 0 0
Phase 2: Time to Maximum Plasma Concentration (Tmax) for FT-2102
Timepoint [20] 0 0
Phase 2: Cycle 1 Day 1 and Cycle 2 Day 1 (Cycle length= 28 days)

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Healthy volunteers
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/04/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/01/2024
Sample size
Target
Accrual to date
Final
336
Recruitment in Australia
Recruitment state(s)
SA,VIC,WA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Victoria Cancer Care Center - Parkville
Recruitment hospital [4] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [5] 0 0
Box Hill Hospital, Monash University and Eastern Health Clinical School - Box Hill
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
3000 - Parkville
Recruitment postcode(s) [4] 0 0
6009 - Nedlands
Recruitment postcode(s) [5] 0 0
3128 - Box Hill
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
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Tennessee
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
France
State/province [15] 0 0
Bobigny
Country [16] 0 0
France
State/province [16] 0 0
Marseille
Country [17] 0 0
France
State/province [17] 0 0
Nantes
Country [18] 0 0
France
State/province [18] 0 0
Paris
Country [19] 0 0
France
State/province [19] 0 0
Pessac
Country [20] 0 0
France
State/province [20] 0 0
Pierre-Bénite
Country [21] 0 0
France
State/province [21] 0 0
Rennes
Country [22] 0 0
France
State/province [22] 0 0
Toulouse
Country [23] 0 0
France
State/province [23] 0 0
Vandœuvre-lès-Nancy
Country [24] 0 0
France
State/province [24] 0 0
Villejuif
Country [25] 0 0
Germany
State/province [25] 0 0
Braunschweig
Country [26] 0 0
Germany
State/province [26] 0 0
Giessen
Country [27] 0 0
Germany
State/province [27] 0 0
Halle
Country [28] 0 0
Germany
State/province [28] 0 0
Münster
Country [29] 0 0
Italy
State/province [29] 0 0
Turin
Country [30] 0 0
Italy
State/province [30] 0 0
Ascoli Piceno
Country [31] 0 0
Italy
State/province [31] 0 0
Bologna
Country [32] 0 0
Italy
State/province [32] 0 0
Meldola
Country [33] 0 0
Italy
State/province [33] 0 0
Parma
Country [34] 0 0
Italy
State/province [34] 0 0
Ravenna
Country [35] 0 0
Italy
State/province [35] 0 0
Rimini
Country [36] 0 0
South Korea
State/province [36] 0 0
Gumi
Country [37] 0 0
South Korea
State/province [37] 0 0
Seoul
Country [38] 0 0
Spain
State/province [38] 0 0
Barcelona
Country [39] 0 0
Spain
State/province [39] 0 0
Madrid
Country [40] 0 0
Spain
State/province [40] 0 0
Salamanca
Country [41] 0 0
Spain
State/province [41] 0 0
Valencia
Country [42] 0 0
United Kingdom
State/province [42] 0 0
London
Country [43] 0 0
United Kingdom
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Oxford
Country [44] 0 0
United Kingdom
State/province [44] 0 0
Southampton
Country [45] 0 0
United Kingdom
State/province [45] 0 0
Sutton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Forma Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Transparency (dept. 2834)
Address 0 0
Novo Nordisk A/S
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02719574