Trial · NCT05568225
A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS)
NCT05568225, Phase 2, Terminated, Forma Therapeutics, Inc.; results first posted 2025-10-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS). Trials, record NCT05568225. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05568225
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS)
- NCT number
- NCT05568225
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Forma Therapeutics, Inc.
- Conditions
- Very Low Risk, Low Risk, or Intermediate Risk MDS Per IPSS-R
- Interventions
- Etavopivat
- First posted
- 2022-10-05
- Last updated
- 2026-09-03
- Results first posted
- 2025-10-22
- 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.
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment | From Baseline to Week 24 | 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. |
| Secondary | Percentage of Participants With Hematologic Improvement-Erythroid (HI-E) Response for =>8 Weeks Within 16 and 48 Weeks of Etavopivat | 48 weeks | 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. |
| Secondary | Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for =>16 Weeks Within 24 and 48 Weeks of Etavopivat | 48 weeks | 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. |
| Secondary | Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | From baseline up to 48 weeks | 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. |
| Secondary | Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions | Within 48 weeks | 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. |
| Secondary | Overall Response Rate | Up to 48 weeks | 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. |
| Secondary | Duration of Response | Up to 48 weeks | 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. |
| Secondary | Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry | Up to 48 weeks | This outcome measure reports reduction in RBC transfusion by 8-week interval in participants with LTB and HTB. |
| Secondary | Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry | Up to 48 weeks | 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). |
| Secondary | Change From Baseline in Neutrophils and/or Platelets Counts | Baseline (week 0), week 48 | This outcome measure reports the change from baseline in neutrophils and/or platelets counts from baseline to week 48 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment | From Baseline to Week 24 | EES: All participants in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Hematologic Improvement-Erythroid (HI-E) Response for =>8 Weeks Within 16 and 48 Weeks of Etavopivat | 48 weeks | FAS: All participants who signed the informed consent and received at least 1 dose of etavopivat. Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in statistical analysis plan (SAP). | ||
| SECONDARY | Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for =>16 Weeks Within 24 and 48 Weeks of Etavopivat | 48 weeks | FAS: All participants who signed the informed consent and received at least 1 dose of etavopivat. Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP. | ||
| SECONDARY | Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | From baseline up to 48 weeks | Safety population includes all participants who received at least one dose of etavopivat (including partial dosing). | Events | NUMBER |
| SECONDARY | Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions | Within 48 weeks | Safety population included all participants who receive at least one dose of etavopivat (including partial dosing). | Events | NUMBER |
| SECONDARY | Overall Response Rate | Up to 48 weeks | Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP. | ||
| SECONDARY | Duration of Response | Up to 48 weeks | Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP. | ||
| SECONDARY | Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry | Up to 48 weeks | EES: All participants in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. n (number analysed) = Participants with available data for a specified category. | Total RBS units | MEAN |
| SECONDARY | Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry | Up to 48 weeks | EES: All participants in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. n (number analysed) = participants with available data for a specified category. | Percent change of total RBC units | MEAN |
| SECONDARY | Change From Baseline in Neutrophils and/or Platelets Counts | Baseline (week 0), week 48 | The Safety Set includes all participants who receive at least one dose of etavopivat (including partial dosing). Here, n (number analysed) = Participants with available data for a specified category. | 10^9 cells per liter | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group99
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment | Non-transfusion Dependent | 0 | Percentage of participants | ||
| Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment | Low Transfusion Burden | 50 | Percentage of participants | ||
| Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment | High Transfusion Burden | 25 | Percentage of participants | ||
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | Non-transfusion Dependent | AE | 33 | Events | |
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | Low Transfusion Burden | AE | 33 | Events | |
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | High Transfusion Burden | AE | 54 | Events | |
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | Non-transfusion Dependent | SAE | 1 | Events | |
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | Low Transfusion Burden | SAE | 0 | Events | |
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | High Transfusion Burden | SAE | 6 | Events | |
| Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat | Non-transfusion Dependent | AEs related to etavopivat | 0 | Events |
1 to 10 of 99Source ClinicalTrials.gov
See all 99 results by groupParticipant flow30
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Treatment Period | STARTED | Non-transfusion Dependent | 3 | |
| Primary Treatment Period | STARTED | Low Transfusion Burden | 5 | |
| Primary Treatment Period | STARTED | High Transfusion Burden | 9 | |
| Primary Treatment Period | Efficacy Evaluable Set (EES) | Non-transfusion Dependent | 1 | |
| Primary Treatment Period | Efficacy Evaluable Set (EES) | Low Transfusion Burden | 2 | |
| Primary Treatment Period | Efficacy Evaluable Set (EES) | High Transfusion Burden | 4 | |
| Primary Treatment Period | Safety Population | Non-transfusion Dependent | 3 | |
| Primary Treatment Period | Safety Population | Low Transfusion Burden | 5 | |
| Primary Treatment Period | Safety Population | High Transfusion Burden | 9 | |
| Primary Treatment Period | Full Analysis Set (FAS) | Non-transfusion Dependent | 3 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 participant flowAdverse events213
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal distension | Gastrointestinal disorders | Non-transfusion Dependent | 0 | 3 | 0 |
| other | Abdominal distension | Gastrointestinal disorders | Low Transfusion Burden | 0 | 5 | 0 |
| other | Abdominal distension | Gastrointestinal disorders | High Transfusion Burden | 1 | 9 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Non-transfusion Dependent | 0 | 3 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Low Transfusion Burden | 1 | 5 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | High Transfusion Burden | 0 | 9 | 0 |
| other | Alanine aminotransferase increased | Investigations | Non-transfusion Dependent | 0 | 3 | 0 |
| other | Alanine aminotransferase increased | Investigations | Low Transfusion Burden | 0 | 5 | 0 |
| other | Alanine aminotransferase increased | Investigations | High Transfusion Burden | 1 | 9 | 3 |
| other | Anaemia | Blood and lymphatic system disorders | Non-transfusion Dependent | 1 | 3 | 3 |
1 to 10 of 213Source ClinicalTrials.gov
See all 213 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Non-transfusion Dependent | 0 | 3 |
| Total, all-cause mortality | Low Transfusion Burden | 0 | 5 |
| Total, all-cause mortality | High Transfusion Burden | 0 | 9 |
| Total, other adverse events | Non-transfusion Dependent | 3 | 3 |
| Total, other adverse events | Low Transfusion Burden | 5 | 5 |
| Total, other adverse events | High Transfusion Burden | 8 | 9 |
| Total, serious adverse events | Non-transfusion Dependent | 1 | 3 |
| Total, serious adverse events | Low Transfusion Burden | 0 | 5 |
| Total, serious adverse events | High Transfusion Burden | 3 | 9 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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