Trial · NCT05144256
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
NCT05144256, Phase 3, Active not recruiting, Agios Pharmaceuticals, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period. Trials, record NCT05144256. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05144256
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
- NCT number
- NCT05144256
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 49 participants, actual
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
- Interventions
- Mitapivat, Mitapivat-matching placebo
- First posted
- 2021-12-03
- Last updated
- 2026-10-28
- Other registries' IDs
- eudract:2021-003265-36
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 Achieving Transfusion Reduction Response (TRR) | Week 9 to Week 32 | TRR is defined as ≥33% reduction in total red blood cell (RBC) transfusion volume from Week 9 through Week 32 of the double-blind period, normalized by weight and actual study drug duration compared with the historical transfusion volume, standardized by weight, and to 24 weeks. |
| Secondary | Percentage of Participants With Transfusion-free Response | Week 9 to Week 32 | Transfusion-free response is defined as achievement of 0 transfusions administered from Week 9 through Week 32 of the double-blind period. |
| Secondary | Change in the Number of Transfusion Episodes | Week 9 to Week 32 | The change in the number of transfusion episodes from Week 9 through Week 32 of the double-blind period compared with the historical number of transfusion episodes standardized to 24 weeks. |
| Secondary | Percentage Change in Weight-normalized and Study Treatment Duration-normalized Total Transfusion Volume | Week 9 to Week 32 | The percentage change in weight-normalized and study treatment duration-normalized total transfusion volume from Week 9 through Week 32 of the double-blind period will be compared with the historical transfusion volume standardized by weight and to 24 weeks. |
| Secondary | Percentage of Participants With Normal Hemoglobin (Hb) Response | Week 9 to Week 32 | Normal Hb response is defined as achievement of Hb concentrations in the normal range at least once, 8 weeks or more after a transfusion during Week 9 through Week 32 of the double-blind period. |
| Secondary | Change From Baseline in Estradiol Concentration | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Estrone Concentration | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Total Testosterone Concentration | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Free Testosterone Concentration in Participants ≥7 Years of Age or Tanner Stage ≥2 (Whichever Occurs First) | Baseline up to Week 298 | |
| Secondary | Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age | Baseline up to Week 298 |
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/NCT05144256
GET https://conductscience.org/api/trials/v1/NCT05144256/registered?page=2