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

Phase 2

Terminated

• Conditions: Very Low Risk, Low Risk, or Intermediate Risk MDS Per IPSS-R
• Interventions: Drug: Etavopivat

• Registration Number: NCT05568225
• Lead Sponsor: Forma Therapeutics, Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-09-15
• Study First Submitted QC: 2022-09-30
• Study First Posted: 2022-10-05
• Study Start: 2022-11-22
• Primary Completion: 2024-07-15
• Study Completion: 2024-07-15
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-12
• Last Update Posted: 2024-12-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Etavopivat 400 mg QD daily	Etavopivat	Non-transfusion dependent (NTD), Low transfusion burden (LTB) , and High transfusion burden (HTB) patients

Primary Outcome Measures

Name	Time	Method
The hematologic improvement based on an erythroid response (HI -E) ≥ 8 weeks duration in patients with MDS after 16 weeks of etavopivat treatment	16 weeks	Measure in number of patient incidence; This endpoint will be based on the combined incidence of: * Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in hemoglobin (Hb) from baseline maintained ≥ 8 consecutive weeks; * Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 8 consecutive weeks; * High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 8 consecutive weeks

Secondary Outcome Measures

Name	Time	Method
The HI-E ≥ 16 weeks duration in this population of patients after 24 and 48 weeks of etavopivat treatment	24 and 48 weeks	Measure in number of patient incidence; This endpoint will be based on the combined incidence of: * Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in hemoglobin (Hb) from baseline maintained ≥ 16 consecutive weeks; * Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 16 consecutive weeks; * High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 16 consecutive weeks
Incidence of AEs, serious adverse events (SAEs), and AEs related to etavopivat	4(first 6 participants), 16, 24, and 48 weeks	Measure in number of patient incidences
Participants who achieved a hematologic improvement in platelet response	16, 24, and 48 weeks.	Measure in number of patient incidence
Change from baseline in mean serum ferritin	16, 24, and 48 weeks.	Measure in ng/mL
Change from baseline in mean daily dose of iron chelation therapy	16, 24, and 48 weeks.	Measure in dose unit
The HI-E ≥ 8 weeks duration in this population of patients after 24 and 48 weeks of etavopivat treatment	24 and 48 weeks	Measure in number of patient incidence; This endpoint will be based on the combined incidence of: * Non-transfusion dependent (NTD) patients: ≥ 1.5 g/dL increase in hemoglobin (Hb) from baseline maintained ≥ 8 consecutive weeks; * Low transfusion burden (LTB) patients: absence of any transfusion for ≥ 8 consecutive weeks; * High transfusion burden (HTB) patients: reduction by ≥ 50% of red blood cell (RBC) units for ≥ 8 consecutive weeks
Overall response rate for MDS	16, 24, and 48 weeks.	Measure in number of patient incidence, per Chelson, 2006 International Working Group \[IWG\] Criteria
Number of premature discontinuations, dose interruptions, and dose reductions	4(first 6 participants), 16, 24, and 48 weeks	Measure in number of patient incidences
Duration of response	16, 24, and 48 weeks.	Measure in number of days, per 2006 IWG Criteria
Percentage of participants who achieved RBC transfusion independence in participants with LTB or HTB at study entry	16, 24, and 48 weeks.	Measure in percentage
Change from baseline in RBC units transfused in patients with NTD, LTB or HTB at study entry	16, 24, and 48 weeks.	Measure in RBC units
Participants who achieved a hematologic improvement in neutrophil	16, 24, and 48 weeks.	Measure in number of patient incidence
Overall survival	16, 24, and 48 weeks.	Measure in number of patient incidence

Trial Locations

Locations (18)

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

Master centre for France; 🇫🇷 Paris La Défense, France

Centre Hospitalier Universitaire de Bordeaux-Hopital Haut Leveque-1; 🇫🇷 Pessac, France

Charite Universitätsmedizin Berlin; 🇩🇪 Mainz, Germany

Universitätsklinikum Leipzig, Klinik und Poliklinik; 🇩🇪 Mainz, Germany

University of Miami Hospital and Clinics; 🇺🇸 Miami, Florida, United States

Ocala Oncology; 🇺🇸 Ocala, Florida, 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

Universitoetsklinikum Heidelberg; 🇩🇪 Heidelberg, Germany

Universitaetsklinikum Muenster; 🇩🇪 Münster, Germany

Universitoetsklinikum Halle (Saale); 🇩🇪 Münster, Germany