A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS)

NCT ID: NCT05568225

Last Updated: 2025-10-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-15
• Study Completion Date: 2024-07-15

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of etavopivat (FT-4202) for the treatment of anemia in adult patients with very low risk, low risk, or intermediate risk MDS.

Conditions

Very Low Risk, Low Risk, or Intermediate Risk MDS Per IPSS-R

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Etavopivat 400 mg QD daily

Non-transfusion dependent (NTD), Low transfusion burden (LTB) , and High transfusion burden (HTB) patients

Group Type: EXPERIMENTAL

Etavopivat

Intervention Type: DRUG

400 mg once daily

Interventions

Etavopivat

400 mg once daily

Intervention Type: DRUG

Other Intervention Names

FT-4202

Eligibility Criteria

Inclusion Criteria

1. Patient has provided documented informed consent; the informed consent form (ICF) must be reviewed and signed by each patient prior to any study-related assessments/procedures being conducted.

2. Age ≥ 18 years at time of first dose.

3. Patients, if female and of childbearing potential, must agree to use acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.

4. Documented diagnosis of idiopathic/de novo MDS according to World Health Organization (WHO) classification that meets the IPSS-R classification of very low, low, or intermediate risk disease, and:

• < 5% blasts in bone marrow based on local pathology review
• < Intermediate risk cytogenetic abnormalities per IPSS-R

5. Anemia defined as:

• Non-transfusion dependent (NTD): Subjects with mean Hb concentration < 10.0 g/dL of 2 measurements (1 performed within 3 days prior to Day 1 and the other performed 7 to 28 days prior to Day 1, not influenced by RBC transfusion within 7 days of measurement) and < 3 RBC transfusions for anemia in the prior 16 weeks before Day 1 of etavopivat dosing

OR

• Transfusion dependent (TD): Subjects having received ≥ 3 units of RBCs for the treatment of anemia within 16 weeks prior to Day 1

6. Serum erythropoietin level > 200 U/L, OR, if ≤ 200 U/L, subject is non-responsive, refractory, or intolerant to erythropoiesis-stimulating agents, or erythropoiesis-stimulating agents are contraindicated or unavailable.

7. ECOG performance status of ≤ 2

8. Subject is non-responsive, refractory, or intolerant to luspatercept, or luspatercept is contraindicated or not indicated.

9. No alternative treatment options are available and/or appropriate for the subject, at the discretion of the investigator.

10. Patient is willing and able to adhere to the study visit schedule and other protocol requirements

Exclusion Criteria

[MDS History]

1. MDS associated with del 5q cytogenetic abnormality and known TP53 abnormality

2. Therapy-associated MDS (eg. t-MDS) that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases

3. Known history of acute myeloid leukemia (AML)

[Medical Conditions]

4. Female who is breast feeding or pregnant

5. Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding

6. Absolute neutrophil count < 500/µL (0.5 x 10^9/L)

7. Platelet count < 50,000/µL (50 x 10^9/L) without transfusion support within 2 weeks

8. Hepatic dysfunction characterized by:

• Alanine aminotransferase (ALT) > 5.0 × upper limit of normal (ULN)
• Total bilirubin > 3.0 × ULN
• History of cirrhosis

9. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the local laboratory < 30 mL/min/1.73 m^2 ) or on chronic dialysis.

10. Patients with clinically significant and active bacterial, fungal, parasitic, or viral infection.

• Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay Screening/ enrollment until active therapy has been completed.
• Patients with acute viral infections without available therapies (eg, coronavirus disease 2019 [COVID-19]) should delay Screening/ enrollment until the acute infection has resolved.

Note: Infection prophylaxis is allowed.

11. Known human immunodeficiency virus (HIV) positivity

12. Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive)

13. Active hepatitis C infection

14. History of malignancy, other than MDS, within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation.

• Patients with malignancy considered surgically cured are eligible (eg, non-melanoma skin cancer, carcinoma in situ of the cervix, or carcinoma in situ of the breast)
• Patients with incidental histologic findings of prostate cancer (T1a or T1b) are eligible

15. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:

• Unstable angina pectoris or myocardial infarction or elective coronary intervention
• Heart disease, heart failure as classified by the New York Heart Association classification 3 or higher, or significant arrhythmia requiring treatment,
• Pulmonary fibrosis or pulmonary hypertension which are clinically significant ie, ≥ Grade 3 National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (or higher)

16. Uncontrolled hypertension, defined as repeated elevation of diastolic blood pressure ≥ 100 mmHg despite adequate treatment

17. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable).

[Prior/Concomitant Therapy]

18. Prior treatment with azacitidine (injectable or oral) or decitabine

19. Use of erythropoietin, other hematopoietic growth factor treatment or lenalidomide within 30 days of starting study treatment or anticipated need for such agents during the study.

20. Prior use of luspatercept:

• NTD patients must not have received luspatercept within 30 days prior to Day 1 treatment
• TD patients must not have received luspatercept within 16 weeks prior to Day 1 treatment

21. Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP)3A4/5 (see Appendix F) within 2 weeks of starting study treatment or anticipated need for such agents during the study.

22. Prior allogeneic or autologous stem cell transplant

23. Initiation of a new chelation therapy within 3 months before the first dose of study treatment.

[Prior/Concurrent Clinical Study Experience]

24. Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device).

[Other Exclusions]

25. Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may preclude safe participation, confound study interpretation, interfere with compliance, or preclude informed consent.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Forma Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Transparency (dept. 2834), MD

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

University of Miami Hospital and Clinics

Miami, Florida, United States

Ocala Oncology

Ocala, Florida, United States

Cedars-Sinai Medical Center

Plainsboro, New Jersey, United States

Northwell Health

Plainsboro, New Jersey, United States

Northwestern Memorial Hospital

Plainsboro, New Jersey, United States

The Ohio State University Medical Center

Plainsboro, New Jersey, United States

NYU Langone Health

New York, New York, United States

Columbia University Irving Medical Center

New York, New York, United States

University of British Columbia - St. Paul's Hospital

Vancouver, British Columbia, Canada

Nice University Hospital - Hôpital de l'Archet

Route de Saint-Antoine, Nice, France

Hopital Saint Louis

Paris, , France

Master centre for France

Paris La Défense, , France

Centre Hospitalier Universitaire de Bordeaux-Hopital Haut Leveque-1

Pessac, , France

Universitoetsklinikum Heidelberg

Heidelberg, , Germany

Charite Universitätsmedizin Berlin

Mainz, , Germany

Universitätsklinikum Leipzig, Klinik und Poliklinik

Mainz, , Germany

Universitaetsklinikum Muenster

Münster, , Germany

Universitoetsklinikum Halle (Saale)

Münster, , Germany

Countries

United States; Canada; France; Germany

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

4202-ONC-203

Identifier Type: -

Identifier Source: org_study_id