A Phase 2 interventional study of Etavopivat in Very Low Risk, Low Risk, or Intermediate Risk MDS Per IPSS-R, sponsored by Forma Therapeutics, Inc.. Terminated at 10 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-03.
Sponsored by Forma Therapeutics, Inc. · Phase 2, Interventional, and Treatment
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.
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's enrollment of 17 is below the median of 94 across 1,291 interventional studies indexed under Anemia.
Browse Anemia studies →Forma Therapeutics, Inc. is the lead sponsor of 10 studies on the registry; 2 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
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:
Anemia defined as:
OR
EXCLUSION CRITERIA:
[MDS History]
Known history of acute myeloid leukemia (AML)
[Medical Conditions]
Hepatic dysfunction characterized by:
Patients with clinically significant and active bacterial, fungal, parasitic, or viral infection.
Note: Infection prophylaxis is allowed.
History of malignancy, other than MDS, within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation.
History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable).
[Prior/Concomitant Therapy]
Prior use of luspatercept:
Initiation of a new chelation therapy within 3 months before the first dose of study treatment.
[Prior/Concurrent Clinical Study Experience]
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]
Non-transfusion dependent (NTD), Low transfusion burden (LTB) , and High transfusion burden (HTB) patients
Drug: Etavopivat
400 mg once daily
Also known as: FT-4202
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.
Time frame: From Baseline to Week 24
Percentage of Participants With Hematologic Improvement-Erythroid (HI-E) Response for =>8 Weeks Within 16 and 48 Weeks of Etavopivat
This outcome measure focused on the combined incidence of NTD, LTB and HTB participants, evaluating HI-E lasting =\>8 weeks in individuals with MDS within 16 weeks and 48 weeks. Participant's response were defined as follows: 1) NTD: an increase of =\>1.5 g/dL in Hb maintained for =\>8 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =\>8 consecutive weeks; and 3) HTB: a reduction of =\>50% in RBC units for =\>8 consecutive weeks.
Time frame: 48 weeks
Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for =>16 Weeks Within 24 and 48 Weeks of Etavopivat
This outcome measure focused on the combined incidence of NDT, LTB and HTB participants, evaluating HI-E response lasting =\>16 weeks in individuals with MDS within 24 weeks and 48 weeks. Participant's response were defined as follows: 1) NTD: an increase of =\>1.5 g/dL in Hb maintained for =\>16 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =\>16 consecutive weeks; and 3) HTB: a reduction of =\>50% in RBC units for =\>16 consecutive weeks.
Time frame: 48 weeks
Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat
This outcome measures the total number of AEs and SAEs in participants, including AEs related to etavopivat. AEs are any unfavorable medical occurrences in participants taking the medicinal product, regardless of causality. They include new or worsening symptoms, abnormal lab findings, and exacerbations of existing conditions, documented from the first dose through 28 days after the last dose. SAEs are severe AEs that result in death, are life-threatening, require hospitalization, lead to significant disability, or involve congenital anomalies. Important medical events posing risks to the participants are also classified as SAEs. Treatment Emergent Adverse Events (TEAEs) are AEs occurring after treatment initiation, aiding in the safety assessment of etavopivat. TEAEs will be considered drug-related if assessed by the Investigator as possibly related or related, or if relationship is missing.
Time frame: From baseline up to 48 weeks
Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions
The outcome measures the total number of premature discontinuations, dose interruptions and dose reductions. Premature discontinuation was defined as any discontinuation prior to week 48. A dose interruption is a temporary halt in treatment due to an AE, while a dose reduction involves lowering the dosage of the drug when AEs occur. If a participant tolerates a reduced dose for 14 days, a rechallenge with the original dose may occur after a clinic visit, but any new Grade 3 or higher AEs require treatment discontinuation and consultation with the Global Medical Monitor before resuming.
Time frame: Within 48 weeks
Overall Response Rate
The Overall Response Rate (ORR) is defined as the percentage of participants who achieve a predefined level of response according to the 2006 International Working Group (IWG) criteria, assessed at each scheduled evaluation.
Time frame: Up to 48 weeks
Duration of Response
This outcome measure reported duration of response which was defined as duration of response will be summarized with quantiles based on product limit estimates (ie, Kaplan-Meier). Duration of response is defined as the time from date of first known incidence of a 2006 IWG criteria response to the most recent date that the 2006 IWG (International Working Group) criteria is not met following the initial response. If the participant has met IWG criteria throughout the period following the initial response, the participant will be censored at the latest date of end of study, loss to follow-up, or death.
Time frame: Up to 48 weeks
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry
This outcome measure reports reduction in RBC transfusion by 8-week interval in participants with LTB and HTB.
Time frame: Up to 48 weeks
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry
This outcome measure is percent change in RBC transfusion by 8-week interval in participants with LTB and HTB. Percent reduction from baseline in RBC transfusion burden is defined as -100×(Total RBC Units Transfused During Interval)/(Baseline RBC Units Transfused Per 8 Weeks).
Time frame: Up to 48 weeks
Change From Baseline in Neutrophils and/or Platelets Counts
This outcome measure reports the change from baseline in neutrophils and/or platelets counts from baseline to week 48
Time frame: Baseline (week 0), week 48
Decrease in Ferritin and Transferrin Saturation (TSAT)
This outcome measure were to report decrease in ferritin and TSAT from baseline to Week 48.
Time frame: Up to 48 weeks
Decrease in Iron Chelation Therapy
This outcome measure were to report decrease of Iron chelation therapy and will be recorded at Screening and on an ongoing basis throughout the study.
Time frame: up to 48 weeks
Overall Survival
Overall survival is defined as the time from first dose to date of death. If the participant was alive at last contact, the end date will be censored at the latest of either end of study, loss to follow-up, or study discontinuation.
Time frame: Within 48 weeks
Etavopivat Plasma Concentrations
This outcome measure reported Etavopivat plasma concentrations in order to assess the PK properties of etavopivat in participants with MDS. PK parameters included but not limited to were: Time to maximum observed plasma concentration, area under the plasma concentration-time curve from time zero until the last quantifiable time point (AUC0-last), from time zero to infinity (AUC0-inf), for a dosing interval (AUCtau/AUC0-24).) However due to early termination of the study, the PK parameters were not assessed and only the plasma concentrations could be assessed.
Time frame: Weeks 1 and 4 pre-dose, post-dose 1, 2, 4, 6 hours.Week 2 and EOT (week 48):pre-dose, post-dose 1, 2 hours
RBC 2,3-diphosphoglycerate (2,3-DPG) and Adenosine Triphosphate (ATP) Levels Over Time
Etavopivat plasma concentrations were collected in order to assess the pharmacodynamic (PD) properties of etavopivat in participants with MDS.
Time frame: Within 48 weeks
The trial was conducted at 10 sites in 4 countries.
| Milestone | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Started | 3 | 5 | 9 |
| Efficacy evaluable set (ees) | 1 | 2 | 4 |
| Safety population | 3 | 5 | 9 |
| Full analysis set (fas) | 3 | 5 | 9 |
| Pharmacokinetic (pk) set | 3 | 5 | 9 |
| Completed | 1 | 2 | 4 |
| Not completed | 2 | 3 | 5 |
| Withdrew: Termination of the study by the sponsor | 0 | 0 | 1 |
| Withdrew: Physician decision | 0 | 1 | 0 |
| Withdrew: Disease progression | 0 | 1 | 0 |
| Withdrew: Lack of efficacy | 0 | 1 | 0 |
| Withdrew: Withdrawal of conset | 1 | 0 | 4 |
| Withdrew: Adverse event | 1 | 0 | 0 |
| Milestone | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Started | 1 | 2 | 4 |
| Completed | 1 | 2 | 1 |
| Not completed | 0 | 0 | 3 |
| Withdrew: Termination of the study by the sponsor | 0 | 0 | 1 |
| Withdrew: Protocol violation | 0 | 0 | 1 |
| Withdrew: Lack of efficacy | 0 | 0 | 1 |
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.
| Percentage of participants | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment | 0 | 50 | 25 |
This outcome measure focused on the combined incidence of NTD, LTB and HTB participants, evaluating HI-E lasting =\>8 weeks in individuals with MDS within 16 weeks and 48 weeks. Participant's response were defined as follows: 1) NTD: an increase of =\>1.5 g/dL in Hb maintained for =\>8 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =\>8 consecutive weeks; and 3) HTB: a reduction of =\>50% in RBC units for =\>8 consecutive weeks.
No measurements were reported for this outcome.
This outcome measure focused on the combined incidence of NDT, LTB and HTB participants, evaluating HI-E response lasting =\>16 weeks in individuals with MDS within 24 weeks and 48 weeks. Participant's response were defined as follows: 1) NTD: an increase of =\>1.5 g/dL in Hb maintained for =\>16 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =\>16 consecutive weeks; and 3) HTB: a reduction of =\>50% in RBC units for =\>16 consecutive weeks.
No measurements were reported for this outcome.
This outcome measures the total number of AEs and SAEs in participants, including AEs related to etavopivat. AEs are any unfavorable medical occurrences in participants taking the medicinal product, regardless of causality. They include new or worsening symptoms, abnormal lab findings, and exacerbations of existing conditions, documented from the first dose through 28 days after the last dose. SAEs are severe AEs that result in death, are life-threatening, require hospitalization, lead to significant disability, or involve congenital anomalies. Important medical events posing risks to the participants are also classified as SAEs. Treatment Emergent Adverse Events (TEAEs) are AEs occurring after treatment initiation, aiding in the safety assessment of etavopivat. TEAEs will be considered drug-related if assessed by the Investigator as possibly related or related, or if relationship is missing.
| Events | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| AE | 33 | 33 | 54 |
| SAE | 1 | 0 | 6 |
| AEs related to etavopivat | 0 | 0 | 1 |
| AEs possibly related etavopivat | 8 | 3 | 6 |
The outcome measures the total number of premature discontinuations, dose interruptions and dose reductions. Premature discontinuation was defined as any discontinuation prior to week 48. A dose interruption is a temporary halt in treatment due to an AE, while a dose reduction involves lowering the dosage of the drug when AEs occur. If a participant tolerates a reduced dose for 14 days, a rechallenge with the original dose may occur after a clinic visit, but any new Grade 3 or higher AEs require treatment discontinuation and consultation with the Global Medical Monitor before resuming.
| Events | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Premature discontinuations | 1 | 0 | 0 |
| Dose interruptions | 0 | 0 | 2 |
| Dose reductions | 0 | 0 | 0 |
The Overall Response Rate (ORR) is defined as the percentage of participants who achieve a predefined level of response according to the 2006 International Working Group (IWG) criteria, assessed at each scheduled evaluation.
No measurements were reported for this outcome.
This outcome measure reported duration of response which was defined as duration of response will be summarized with quantiles based on product limit estimates (ie, Kaplan-Meier). Duration of response is defined as the time from date of first known incidence of a 2006 IWG criteria response to the most recent date that the 2006 IWG (International Working Group) criteria is not met following the initial response. If the participant has met IWG criteria throughout the period following the initial response, the participant will be censored at the latest date of end of study, loss to follow-up, or death.
No measurements were reported for this outcome.
This outcome measure reports reduction in RBC transfusion by 8-week interval in participants with LTB and HTB.
| Total RBS units | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|
| Week 1-8 | 3.0 ± 2.83 | 5.8 ± 2.63 |
| Week 9-16 | 2.5 ± 2.12 | 6.3 ± 1.71 |
| Week 17-24 | 3.5 ± 2.12 | 7.5 ± 3.00 |
| Week 25-32 | 5.5 ± 0.71 | 7.3 ± 1.15 |
| Week 33-40 | 5.0 ± 4.24 | 6.7 ± 1.15 |
| Week 41-48 | 4.5 ± 2.12 | 7.5 ± 2.12 |
This outcome measure is percent change in RBC transfusion by 8-week interval in participants with LTB and HTB. Percent reduction from baseline in RBC transfusion burden is defined as -100×(Total RBC Units Transfused During Interval)/(Baseline RBC Units Transfused Per 8 Weeks).
| Percent change of total RBC units | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|
| Week 1-8 | -20.83 ± 64.818 | 68.75 ± 156.994 |
| Week 9-16 | -33.33 ± 47.140 | 68.75 ± 98.219 |
| Week 17-24 | -4.17 ± 41.248 | 120.83 ± 191.183 |
| Week 25-32 | 58.33 ± 11.785 | 127.78 ± 149.381 |
| Week 33-40 | 33.33 ± 94.281 | 94.44 ± 91.793 |
| Week 41-48 | 25.00 ± 35.355 | 50.00 ± 0 |
This outcome measure reports the change from baseline in neutrophils and/or platelets counts from baseline to week 48
| 10^9 cells per liter | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Neutrophils | -3.200 ± NA | -0.950 ± 0.0707 | -0.100 ± NA |
| Platelets counts | 43.0 ± NA | -51.0 ± 205.06 | -8.0 ± NA |
This outcome measure were to report decrease in ferritin and TSAT from baseline to Week 48.
No measurements were reported for this outcome.
This outcome measure were to report decrease of Iron chelation therapy and will be recorded at Screening and on an ongoing basis throughout the study.
No measurements were reported for this outcome.
Overall survival is defined as the time from first dose to date of death. If the participant was alive at last contact, the end date will be censored at the latest of either end of study, loss to follow-up, or study discontinuation.
No measurements were reported for this outcome.
This outcome measure reported Etavopivat plasma concentrations in order to assess the PK properties of etavopivat in participants with MDS. PK parameters included but not limited to were: Time to maximum observed plasma concentration, area under the plasma concentration-time curve from time zero until the last quantifiable time point (AUC0-last), from time zero to infinity (AUC0-inf), for a dosing interval (AUCtau/AUC0-24).) However due to early termination of the study, the PK parameters were not assessed and only the plasma concentrations could be assessed.
| ng/mL | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Week 1 predose | 0 ± 0 | 0 ± 0 | 0 ± 0 |
| Week 1 post-dose 1 hour | 379.77 ± 292.748 | 904.80 ± 521.000 | 1016.78 ± 794.503 |
| Week 1 post-dose 2 hour | 751.67 ± 393.465 | 579.60 ± 403.593 | 519.11 ± 273.776 |
| Week 1 post-dose 4 hour | 274.50 ± 86.974 | 203.80 ± 134.884 | 165.16 ± 64.895 |
| Week 1 post-dose 6 hour | 63.50 ± NA | 101.02 ± 67.693 | 83.74 ± 40.170 |
| Week 2 pre-dose | 27.20 ± 17.843 | 19.08 ± 9.037 | 19.20 ± 12.240 |
| Week 2 post-dose 1 hour | 1156.00 ± 371.198 | 899.40 ± 442.555 | 1076.44 ± 693.907 |
| Week 2 post-dose 2 hour | 927.00 ± 310.140 | 1024.00 ± 530.102 | 661.78 ± 392.238 |
| Week 4 predose | 17.04 ± 11.540 | 24.34 ± 23.333 | 20.26 ± 9.289 |
| Week 4 post-dose 1 hour | 1050.00 ± 28.284 | 386.90 ± 214.249 | 913.20 ± 1013.207 |
| Week 4 post-dose 2 hour | 526.00 ± 134.350 | 453.40 ± 179.140 | 678.49 ± 379.918 |
| Week 4 post-dose 4 hour | 151.50 ± 12.021 | 383.73 ± 261.103 | 297.90 ± 282.646 |
| Week 4 post-dose 6 hour | 60.75 ± 11.526 | 148.13 ± 113.650 | 121.34 ± 115.306 |
| End of treatment pre-dose | — | 21.60 ± 5.798 | 24.20 ± NA |
| End of treatment post-dose 1 hours | — | 1620.00 ± NA | 1680.00 ± NA |
| End of treatment post-dose 2 hours | — | 571.00 ± NA | 815.00 ± NA |
Etavopivat plasma concentrations were collected in order to assess the pharmacodynamic (PD) properties of etavopivat in participants with MDS.
No measurements were reported for this outcome.
Collected over Up to 48 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Non-transfusion Dependent | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Low Transfusion Burden | 0/5 (0%) | 0/5 (0%) | 5/5 (100%) |
| High Transfusion Burden | 0/9 (0%) | 3/9 (33.3%) | 8/9 (88.9%) |
| Event | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| Cardiac failureCardiac disorders | 1/3 | 0/5 | 0/9 |
| Angina pectorisCardiac disorders | 0/3 | 0/5 | 1/9 |
| Arterial ruptureVascular disorders | 0/3 | 0/5 | 1/9 |
| Hip fractureInjury, poisoning and procedural complications | 0/3 | 0/5 | 1/9 |
| Peripheral arterial occlusive diseaseVascular disorders | 0/3 | 0/5 | 1/9 |
| Post procedural haemorrhageInjury, poisoning and procedural complications | 0/3 | 0/5 | 1/9 |
| Staphylococcal infectionInfections and infestations | 0/3 | 0/5 | 1/9 |
| Event | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden |
|---|---|---|---|
| AnxietyPsychiatric disorders | 2/3 | 1/5 | 0/9 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 2/3 | 0/5 | 1/9 |
| Lymphocyte count decreasedInvestigations | 2/3 | 0/5 | 0/9 |
| ThrombocytopeniaBlood and lymphatic system disorders | 2/3 | 0/5 | 0/9 |
| PneumoniaInfections and infestations | 0/3 | 2/5 | 0/9 |
| AnaemiaBlood and lymphatic system disorders | 1/3 | 0/5 | 0/9 |
| Atrioventricular block second degreeCardiac disorders | 1/3 | 0/5 | 0/9 |
| Cardiac failureCardiac disorders | 1/3 | 1/5 | 0/9 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/3 | 0/5 | 1/9 |
| FatigueGeneral disorders | 1/3 | 0/5 | 1/9 |
FAS included all participants who signed the informed consent and received at least 1 dose of etavopivat.
| Age, Continuous(Years) | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden | Total |
|---|---|---|---|---|
| Mean | 77.3 ± 5.69 | 80.2 ± 3.49 | 71.2 ± 8.11 | 74.9 ± 7.58 |
| Sex: Female, Male(Participants) | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden | Total |
|---|---|---|---|---|
| Female | 0 | 1 | 3 | 4 |
| Male | 3 | 4 | 6 | 13 |
| Race/Ethnicity, Customized(Participants) | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden | Total |
|---|---|---|---|---|
| Race: White | 3 | 4 | 7 | 14 |
| Race: Not reported | 0 | 1 | 2 | 3 |
| Race/Ethnicity, Customized(Participants) | Non-transfusion Dependent | Low Transfusion Burden | High Transfusion Burden | Total |
|---|---|---|---|---|
| Ethnicity: Not Hispanic or Latino | 3 | 4 | 6 | 13 |
| Ethnicity: Hispanic or Latino | 0 | 1 | 1 | 2 |
| Ethnicity: Not Reported | 0 | 0 | 2 | 2 |
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Forma Therapeutics, Inc.