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
Public ClinicalTrials.gov record NCT05175105. 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 Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period
Study identification
- NCT ID
- NCT05175105
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Enrollment
- 30 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 5, 2022
- Primary completion
- Dec 12, 2024
- Completion
- Dec 31, 2029
- Last update posted
- Apr 23, 2026
2022 – 2030
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford Medicine | Palo Alto | California | 94304 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Children's Healthcare of Atlanta - Emory | Atlanta | Georgia | 30322 | — |
| UChicago Medicine | Chicago | Illinois | 60637 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Children's Hospital of Michigan | Detroit | Michigan | 48304 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| St Jude's Children's Research Hospital | Memphis | Tennessee | 38105 | — |
| 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 8 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05175105, 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 Apr 23, 2026 · Synced Apr 26, 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 NCT05175105 live on ClinicalTrials.gov.