Trial · NCT05175105
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
NCT05175105, 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 Not Regularly Transfused, Followed by a 5-Year Extension Period. Trials, record NCT05175105. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05175105
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 Not Regularly Transfused, Followed by a 5-Year Extension Period
- NCT number
- NCT05175105
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 30 participants, actual
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
- Interventions
- Mitapivat, Mitapivat-matching placebo
- First posted
- 2022-01-03
- Last updated
- 2026-10-28
- Other registries' IDs
- eudract:2021-003333-11
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 Achieving a Hemoglobin (Hb) Response | Baseline up to Week 20 | Hb response is defined as a ≥1.5 grams per deciliter (g/dL) (0.93 millimoles per liter \[mmol/L\]) increase in Hb concentration from baseline that is sustained at 2 or more scheduled assessments at Weeks 12, 16, and 20 during the double-blind period. The individual participant's baseline Hb concentration is defined as the average of all available Hb concentrations collected for that participant during the screening period up to the first dose of study drug. |
| Secondary | Average Change From Baseline in Hb Concentration at Weeks 12, 16, and 20 | Baseline, Weeks 12, 16, and 20 | |
| Secondary | Maximal Change in Hb Concentration From Baseline During the Double-blind Period | Baseline up to Week 20 | |
| Secondary | Change From Baseline in Estradiol Concentration | Baseline up to Week 286 | |
| Secondary | Change From Baseline in Estrone Concentration | Baseline up to Week 286 | |
| Secondary | Change From Baseline in Total Testosterone Concentration | Baseline up to Week 286 | |
| 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 286 | |
| Secondary | Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age | Baseline up to Week 286 | |
| Secondary | Change From Baseline in Sexual Maturity Rating with Tanner Stage | Baseline up to Week 286 | Tanner Stage 1 corresponds to the prepubertal form, with progression to Tanner Stage 5, the final adult form. |
| Secondary | Number of Female Participants With Development of Ovarian Cysts | Baseline up to Week 286 |
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/NCT05175105
GET https://conductscience.org/api/trials/v1/NCT05175105/registered?page=2