Trial · NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
NCT07517133, 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 α- or β-Non-Transfusion-Dependent Thalassemia. Trials, record NCT07517133. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07517133
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 α- or β-Non-Transfusion-Dependent Thalassemia
- NCT number
- NCT07517133
- Status
- Recruiting
- Phases
- Phase 3
- Enrollment
- 45 participants, estimated
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
- Interventions
- Mitapivat, Placebo Matching Mitapivat
- First posted
- 2026-04-08
- Last updated
- 2026-10-28
Source ClinicalTrials.gov
Registered outcomes29
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Achieved a Hemoglobin (Hb) Response | Baseline, Week 12 through Week 24 | Hb response is defined as a ≥1.0 grams per deciliter (g/dL) increase in average Hb concentration from Week 12 through Week 24 compared with baseline. |
| Secondary | Change From Baseline in Average Hb Concentration From Week 12 Through Week 24 | Baseline, Week 12 through Week 24 | |
| Secondary | Percentage of Participants Who Achieved a Hb 1.5+ Response | Baseline, 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. |
| Secondary | Change From Baseline in Indirect Bilirubin at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Haptoglobin at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Reticulocytes at Week 24 | Baseline, Week 24 | |
| Secondary | Change From Baseline in Erythropoietin at Week 24 | Baseline, Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Estradiol Through Week 24 | Baseline, up to Week 24 | |
| Secondary | Cohort 1: Change From Baseline in Estrone Through Week 24 | Baseline, up to Week 24 |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 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/NCT07517133
GET https://conductscience.org/api/trials/v1/NCT07517133/registered?page=2