Trial · NCT07506863
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
NCT07506863, Phase 3, Recruiting, Agios Pharmaceuticals, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT). Trials, record NCT07506863. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07506863
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
- NCT number
- NCT07506863
- Status
- Recruiting
- Phases
- Phase 3
- Enrollment
- 54 participants, estimated
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
- Interventions
- Mitapivat Matched Placebo, Mitapivat
- First posted
- 2026-04-02
- Last updated
- 2026-10-29
Source ClinicalTrials.gov
Registered outcomes27
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48 | Baseline, through Week 48 | TRR is defined as ≥50 percent (%) reduction in transfused red blood cells (RBC) volume (normalized by weight) in any consecutive 12-week period through Week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks. |
| Secondary | Percentage of Participants Who Achieved TRR2 From Week 13 to Week 24 | Baseline, Week 13 through Week 24 | TRR2 is defined as ≥50% reduction in transfused RBC volume (normalized by weight) from week 13 to week 24 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks. |
| Secondary | Percentage of Participants Who Achieved TRR3 From Week 25 to Week 36 | Baseline, Week 25 through Week 36 | TRR3 is defined as ≥50% reduction in transfused RBC volume (normalized by weight) from week 25 to week 36 compared with historical RBC transfusion volume (Normalized by Weight) and standardized to 12 weeks. |
| Secondary | Percentage of Participants Who Achieved TRR4 From Week 37 to Week 48 | Baseline, Week 37 through Week 48 | TRR4 is defined as ≥50% reduction in transfused RBC volume (normalized by weight) from week 37 to week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks. |
| Secondary | Percentage of Participants Who Achieved TRR5 From Week 25 to Week 48 | Baseline, Week 25 through Week 48 | TRR5 is defined as ≥50% reduction in transfused RBC volume (normalized by weight) from week 25 to week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 24 weeks. |
| Secondary | Percent Change in Transfused RBC Volume (Normalized by Weight) From Week 13 Through Week 48 Compared With Historical Transfusion Volume (Normalized by Weight) and Standardized to 36 Weeks | Baseline, Week 13 through Week 48 | |
| Secondary | Percentage of Participants Who Achieved Transfused Independence Through Week 48 | Baseline, through Week 48 | Transfusion independence is defined as transfusion-free for greater than or equal to 8 consecutive weeks through Week 48. |
| Secondary | Change in the Number of Transfusion Episodes From Week 13 Through Week 48 Compared With Historical Number of Transfusion Episodes Standardized to 36 Weeks | Baseline, Week 13 through Week 48 | |
| Secondary | Change From Baseline in Serum Iron at Week 48 | Baseline, Week 48 | |
| Secondary | Change From Baseline in Total Iron-Binding Capacity at Week 48 | Baseline, Week 48 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
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.
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/NCT07506863
GET https://conductscience.org/api/trials/v1/NCT07506863/registered?page=2