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NCT05144256 · ClinicalTrials.gov registry record · Phase 3
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
A Phase 3 study of Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia, sponsored by Agios Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 49
- Enrollment target
- 20
- Study locations
NCT05144256: Active Phase 3 study of Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia, sponsored by Agios Pharmaceuticals.
NCT05144256 is a Phase 3 study of Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia that is active but no longer recruiting, run by Agios Pharmaceuticals. The registered enrollment target is 49 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (94% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05144256, a Phase 3 study of Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia, is active but no longer recruiting, sponsored by Agios Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 49 participants
- Enrollment target
- 20
- Study locations
Study Summary
ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either mitapivat or matching placebo. Randomization will be stratified by age (1 to \< 6 years, 6 to \< 12 years, 12 to \< 18 years) and splenectomy status. Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 24-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat in the open-label extension (OLE) period.
Primary Outcome
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.
Conditions Studied
Interventions
- DRUG Mitapivat
- DRUG Mitapivat-matching placebo
Study Locations (20)
Other
- Fakultní Nemocnice Olomouc - Olomouc
- Rigshospitalet - Copenhagen
- Charite - UB - CVK - Medizinische Klinik - Berlin
- Universitair Medisch Centrum Utrecht - Utrecht
- Hospital Universitario Vall d'Hebron - Barcelona
- Hospital Clinico Universitario Virgen de la Arrixaca - Madrid
- Hacettepe University - Ankara
Texas
- UT Southwestern Medical Center - Dallas
- Texas Children's Hospital - Houston
Arizona
- Phoenix Children's Hospital - Phoenix
California
- Stanford Medicine - Palo Alto
Georgia
- Children's Healthcare of Atlanta - Emory - Atlanta
Massachusetts
- Boston Children's Hospital - Boston
Nevada
- Cure 4 the Kids Foundation, A Division of Roseman University of Health Sciences - Las Vegas
New York
- Weill Cornell Medical College - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 49 participants |
| Start Date | 2022-06-08 |
| Est. Completion | 2029-06 |
| Phase | Phase 3 |
What the registry record for NCT05144256 still lists
NCT05144256 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 49 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (94% lower).
The record links to 2 conditions, with Pediatric Pyruvate Kinase Deficiency appearing as the primary indexed condition, and to 2 interventions - of which Mitapivat is the first listed.
NCT05144256 names 20 study sites across 13 states, led by Other, Texas, Arizona.
Frequently Asked Questions
What is clinical trial NCT05144256 about?
NCT05144256 is a clinical study titled "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". ACTIVATE-KidsT (AG348-C-022) is a multicenter study designed to evaluate the efficacy and safety of treatment with mitapivat compared with placebo in pediatric participants with pyruvate kinase deficiency (PK deficiency) who are regularly receiving blood transfusions. Participants will be randomized...
What is the current status of trial NCT05144256?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 49 participants. The study started on 2022-06-08. Estimated completion is 2029-06.
What conditions does trial NCT05144256 study?
This clinical trial studies the following conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia.
What interventions are being tested in trial NCT05144256?
The interventions under investigation include: Mitapivat (DRUG), Mitapivat-matching placebo (DRUG).
Who is sponsoring clinical trial NCT05144256?
This trial is sponsored by Agios Pharmaceuticals, which has 44 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05144256 being conducted?
This trial has 20 study locations across Arizona, California, Georgia, Massachusetts, Nevada. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05144256's enrollment target sits among peer trials
49 1861st of 2000 higher than 139 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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