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

NCT05568225 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: 4202-ONC-203
• Study Type: interventional
• Target: 17
• Locations: 4 countries
• Sites: 18

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

Keywords

Anemia; Myelodysplastic Syndromes; MDS; Pyruvate Kinase; Activator; Transfusion dependent; FT-4202; Etavopivat; IPSS-R very low risk; IPSS-R low risk; IPSS-R intermediate risk; ESA refractory; ESA intolerant; Luspatercept refractory; Luspatercept intolerant

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment

  This outcome measure reported on the combined incidence of NTD, LTB and HTB in terms of (HI-E) response for \>=8 weeks duration in participants with myelodysplastic syndromes (MDS) within 24 weeks. The participants were allocated to the following arms which were defined as: 1) NTD: \>=1.5 grams per deciliter (g/dL) increase in haemoglobin (Hb) from baseline maintained \>=8 consecutive weeks and no transfusion of RBC units for anemia over a continuous 8-week treatment period; 2) LTB: absence of any transfusion for \>=8 consecutive weeks; and 3) HTB: reduction by \>=50 percent (%) of RBC units for \>=8 consecutive weeks.

  From Baseline to Week 24

Secondary Outcomes (14)

• Percentage of Participants With Hematologic Improvement-Erythroid (HI-E) Response for =>8 Weeks Within 16 and 48 Weeks of Etavopivat

  48 weeks

• Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for =>16 Weeks Within 24 and 48 Weeks of Etavopivat

  48 weeks

• Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat

  From baseline up to 48 weeks

• Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions

  Within 48 weeks

• Overall Response Rate

  Up to 48 weeks

Study Arms (1)

Etavopivat 400 mg QD daily

EXPERIMENTAL

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

Drug: Etavopivat

Interventions

Etavopivat DRUG

400 mg once daily

Also known as: FT-4202

Etavopivat 400 mg QD daily

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Age ≥ 18 years at time of first dose.
• 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.
• 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
• 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
• Transfusion dependent (TD): Subjects having received ≥ 3 units of RBCs for the treatment of anemia within 16 weeks prior to Day 1
• 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.
• ECOG performance status of ≤ 2
• Subject is non-responsive, refractory, or intolerant to luspatercept, or luspatercept is contraindicated or not indicated.
• No alternative treatment options are available and/or appropriate for the subject, at the discretion of the investigator.
• Patient is willing and able to adhere to the study visit schedule and other protocol requirements

You may not qualify if:

• \[MDS History\]
• MDS associated with del 5q cytogenetic abnormality and known TP53 abnormality
• 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
• Known history of acute myeloid leukemia (AML)
• \[Medical Conditions\]
• Female who is breast feeding or pregnant
• Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
• Absolute neutrophil count \< 500/µL (0.5 x 10\^9/L)
• Platelet count \< 50,000/µL (50 x 10\^9/L) without transfusion support within 2 weeks
• Hepatic dysfunction characterized by:
• Alanine aminotransferase (ALT) \> 5.0 × upper limit of normal (ULN)
• Total bilirubin \> 3.0 × ULN
• History of cirrhosis
• 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.
• Patients with clinically significant and active bacterial, fungal, parasitic, or viral infection.

Sponsors & Collaborators

• Forma Therapeutics, Inc.: lead

Study Sites (18)

University of Miami Hospital and Clinics

Miami, Florida, 33136, United States

Location

Ocala Oncology

Ocala, Florida, 34474, United States

Location

Cedars-Sinai Medical Center

Plainsboro, New Jersey, 08536, United States

Location

Northwell Health

Plainsboro, New Jersey, 08536, United States

Location

Northwestern Memorial Hospital

Plainsboro, New Jersey, 08536, United States

Location

The Ohio State University Medical Center

Plainsboro, New Jersey, 08536, United States

Location

NYU Langone Health

New York, New York, 10016, United States

Location

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

University of British Columbia - St. Paul's Hospital

Vancouver, British Columbia, V6Z 2K5, Canada

Location

Nice University Hospital - Hôpital de l'Archet

Route de Saint-Antoine, Nice, 06200, France

Location

Hopital Saint Louis

Paris, France

Location

Master centre for France

Paris La Défense, 92936, France

Location

Centre Hospitalier Universitaire de Bordeaux-Hopital Haut Leveque-1

Pessac, 33600, France

Location

Universitoetsklinikum Heidelberg

Heidelberg, Germany

Location

Charite Universitätsmedizin Berlin

Mainz, 55124, Germany

Location

Universitätsklinikum Leipzig, Klinik und Poliklinik

Mainz, 55124, Germany

Location

Universitaetsklinikum Muenster

Münster, Germany

Location

Universitoetsklinikum Halle (Saale)

Münster, Germany

Location

MeSH Terms

Conditions

Anemia; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Limitations and Caveats

The futility assessment concluded low likelihood of the trial showing meaningful participant benefit. The decision was based on a predefined QTL that required 5 or more participants to complete 16 weeks of treatment or not having 8 or more consecutive weeks of primary endpoint data. However 7 participants did not fulfill this requirement, leading to the early termination of the study.

Results Point of Contact

• Title: Clinical Reporting Office (2834)
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

(+1) 866-867-7178

Study Officials

• Clinical Transparency (dept. 2834), MD

  Novo Nordisk A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 15, 2022
• First Posted: October 5, 2022
• Study Start: November 15, 2022
• Primary Completion: July 15, 2024
• Study Completion: July 15, 2024
• Last Updated: October 22, 2025
• Results First Posted: October 22, 2025

Record last verified: 2025-10

Locations

US (8); DE (5); CA (1); FR (4)