Trial · NCT04770753
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
NCT04770753, Phase 3, Active not recruiting, Agios Pharmaceuticals, Inc.; results first posted 2025-01-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT). Trials, record NCT04770753. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04770753
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
- NCT number
- NCT04770753
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 194 participants, actual
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
- Interventions
- Placebo Matching Mitapivat, Mitapivat
- First posted
- 2021-02-25
- Last updated
- 2026-10-30
- Other registries' IDs
- eudract:2021-000211-23
- Results first posted
- 2025-01-24
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline | Double-Blind Period: Baseline up to Week 12 through Week 24 | Hb response is defined as ≥10 grams/ liter (g/L) (1.0 gram per deciliter) (g/dL) increase in average Hb concentration from Week 12 through Week 24 compared with baseline. Hb response was tested using the Mantel-Haenszel stratum weighted method adjusting for randomization stratification factors. Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24 | Double-Blind Period: Baseline, Week 12 through Week 24 | The FACIT-Fatigue subscale includes a 13-item self-reported fatigue subscale, which assesses the severity and impact of fatigue (including the impact on daily activities and functioning).The FACIT-Fatigue subscale is scored on a 5-point Likert scale: 0 (not at all) to 4 (very much). The total FACIT-Fatigue subscale score ranges from 0 to 52, with a higher score indicating better health-related quality of life (HRQOL). Baseline is defined as the last assessment before randomization for subjects randomized and not dosed or the last assessment before start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Average Hb Concentration From Week 12 Through Week 24 | Double-Blind Period: Baseline, Week 12 through Week 24 | Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Percentage of Participants Who Achieved Hb 1.5+ Response From Week 12 Through Week 24 Compared With Baseline | Double-Blind Period: Baseline up to Week 12 through Week 24 | Hb 1.5+ response is defined as a ≥1.5 g/dL increase in average Hb concentration from Week 12 through Week 24 compared with baseline. Hb 1.5+ response will be summarized using the Mantel-Haenszel stratum weighted method adjusting for randomization stratification factors. Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Indirect Bilirubin at Week 24 | Double-Blind Period: Baseline, Week 24 | Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24 | Double-Blind Period: Baseline, Week 24 | Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Haptoglobin at Week 24 | Double-Blind Period: Baseline, Week 24 | Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Reticulocytes at Week 24 | Double-Blind Period: Baseline, Week 24 | Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Change From Baseline in Erythropoietin at Week 24 | Double-Blind Period: Baseline, Week 24 | Baseline is defined as the average of all assessments within 42 days before randomization for subjects randomized and not dosed or within 42 days before the start of study treatment for subjects randomized and dosed. |
| Secondary | Double-Blind Period: Percentage of Participants Who Achieved Patient Global Impression of Severity (PGIS)- Fatigue Response at Weeks 12, 16, 20, and 24 | Double-Blind Period: At Weeks 12, 16, 20, and 24 | PGIS-Fatigue measured participants' perception of their fatigue severity (7-day recall) on a 4-point scale ranging from '1=none' to '4=severe'. A participant was considered to have achieved the PGIS-Fatigue response at Weeks 12, 16, 20, or 24, if their baseline to postbaseline score met one of the following conditions: 'none' at baseline to 'none' postbaseline; 'mild' to 'mild' or 'none'; 'moderate' to 'mild' or 'none'; or 'severe' to 'moderate', 'mild', or 'none'. |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes24
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline | Double-Blind Period: Baseline up to Week 12 through Week 24 | FAS included all participants who were randomized. | Percentage of participants | NUMBER |
| SECONDARY | Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24 | Double-Blind Period: Baseline, Week 12 through Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Change From Baseline in Average Hb Concentration From Week 12 Through Week 24 | Double-Blind Period: Baseline, Week 12 through Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | Gram per Liter (g/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Percentage of Participants Who Achieved Hb 1.5+ Response From Week 12 Through Week 24 Compared With Baseline | Double-Blind Period: Baseline up to Week 12 through Week 24 | FAS included all participants who were randomized. | Percentage of participants | NUMBER |
| SECONDARY | Double-Blind Period: Change From Baseline in Indirect Bilirubin at Week 24 | Double-Blind Period: Baseline, Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | Micromole per Liter (umol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24 | Double-Blind Period: Baseline, Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | Units per Liter (U/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Change From Baseline in Haptoglobin at Week 24 | Double-Blind Period: Baseline, Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | Gram per Liter (g/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Change From Baseline in Reticulocytes at Week 24 | Double-Blind Period: Baseline, Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | 10^9 cells per liter (10^9 cells/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Change From Baseline in Erythropoietin at Week 24 | Double-Blind Period: Baseline, Week 24 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | International units per Liter (IU/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-Blind Period: Percentage of Participants Who Achieved Patient Global Impression of Severity (PGIS)- Fatigue Response at Weeks 12, 16, 20, and 24 | Double-Blind Period: At Weeks 12, 16, 20, and 24 | FAS included all participants who were randomized. | percentage of participants | NUMBER |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 outcomesResults by group92
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline | Mitapivat | 42.3 | Percentage of participants | ||
| Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline | Placebo | 1.6 | Percentage of participants | ||
| Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24 | Mitapivat | 4.85 | Standard Error 0.732 | score on a scale | |
| Double-Blind Period: Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score From Week 12 Through Week 24 | Placebo | 1.46 | Standard Error 0.955 | score on a scale | |
| Double-Blind Period: Change From Baseline in Average Hb Concentration From Week 12 Through Week 24 | Mitapivat | 8.57 | Standard Error 0.666 | Gram per Liter (g/L) | |
| Double-Blind Period: Change From Baseline in Average Hb Concentration From Week 12 Through Week 24 | Placebo | -1.06 | Standard Error 0.867 | Gram per Liter (g/L) | |
| Double-Blind Period: Percentage of Participants Who Achieved Hb 1.5+ Response From Week 12 Through Week 24 Compared With Baseline | Mitapivat | 24.6 | Percentage of participants | ||
| Double-Blind Period: Percentage of Participants Who Achieved Hb 1.5+ Response From Week 12 Through Week 24 Compared With Baseline | Placebo | 0 | Percentage of participants | ||
| Double-Blind Period: Change From Baseline in Indirect Bilirubin at Week 24 | Mitapivat | -10.65 | Standard Error 1.047 | Micromole per Liter (umol/L) | |
| Double-Blind Period: Change From Baseline in Indirect Bilirubin at Week 24 | Placebo | -0.03 | Standard Error 1.403 | Micromole per Liter (umol/L) |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Mitapivat | 130 | |
| Overall Study | STARTED | Placebo | 64 | |
| Overall Study | Treated | Mitapivat | 129 | |
| Overall Study | Treated | Placebo | 63 | |
| Overall Study | COMPLETED | Mitapivat | 122 | |
| Overall Study | COMPLETED | Placebo | 62 | |
| Overall Study | NOT COMPLETED | Mitapivat | 8 | |
| Overall Study | NOT COMPLETED | Placebo | 2 |
Source ClinicalTrials.gov
Adverse events48
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 | Upper respiratory tract infection | Infections and infestations | Mitapivat | 13 | 129 | |
| other | Upper respiratory tract infection | Infections and infestations | Placebo | 4 | 63 | |
| other | COVID-19 | Infections and infestations | Mitapivat | 8 | 129 | |
| other | COVID-19 | Infections and infestations | Placebo | 5 | 63 | |
| other | Influenza | Infections and infestations | Mitapivat | 5 | 129 | |
| other | Influenza | Infections and infestations | Placebo | 6 | 63 | |
| other | Nausea | Gastrointestinal disorders | Mitapivat | 15 | 129 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 5 | 63 | |
| other | Diarrhoea | Gastrointestinal disorders | Mitapivat | 11 | 129 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 6 | 63 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Mitapivat | 0 | 130 |
| Total, all-cause mortality | Placebo | 0 | 64 |
| Total, other adverse events | Mitapivat | 80 | 129 |
| Total, other adverse events | Placebo | 33 | 63 |
| Total, serious adverse events | Mitapivat | 8 | 129 |
| Total, serious adverse events | Placebo | 0 | 63 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40544857 | DERIVED |
Source PubMed
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Mitapivat vs Placebo: deaths from any cause | Trial comparisons |
| Mitapivat vs Placebo: serious adverse events | Trial comparisons |
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04770753
GET https://conductscience.org/api/trials/v1/NCT04770753/registered?page=2