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
Public ClinicalTrials.gov record NCT05144256. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period
Study identification
- NCT ID
- NCT05144256
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Enrollment
- 49 participants
Conditions and interventions
Interventions
- Mitapivat Drug
- Mitapivat-matching placebo Drug
Drug
Eligibility (public fields only)
- Age range
- 1 Year to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 7, 2022
- Primary completion
- May 2, 2024
- Completion
- May 31, 2029
- Last update posted
- Jun 22, 2026
2022 – 2029
United States locations
- U.S. sites
- 9
- U.S. states
- 8
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children's Hospital | Phoenix | Arizona | 85016 | — |
| Stanford Medicine | Palo Alto | California | 94304 | — |
| Children's Healthcare of Atlanta - Emory | Atlanta | Georgia | 30322 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences | Las Vegas | Nevada | 89135 | — |
| Weill Cornell Medical College | New York | New York | 10065 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| UT Southwestern Medical Center | Dallas | Texas | 75390 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 13 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05144256, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 22, 2026 · Synced Jul 22, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05144256 live on ClinicalTrials.gov.