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

Stopped early · Phase 2

Conditions studied: Very Low Risk, Low Risk, or Intermediate Risk MDS Per IPSS-R

In brief

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.

Key facts

Study ID

NCT05568225

Run by

Forma Therapeutics, Inc.

People needed

17

Starts

2022-11-15

Expected to finish

2024-07-15

Last updated by the study team

2025-10-22

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

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
• OR
• 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.
• 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.
• Known human immunodeficiency virus (HIV) positivity
• Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive)
• Active hepatitis C infection
• 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
• History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:

Where it is running

• 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

Full record on ClinicalTrials.gov

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