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NCT05175105 · 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 Not 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
- 30
- Enrollment target
- 19
- Study locations
NCT05175105 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 30 participants. The trial reports 19 study locations across 15 states.
The verdict
NCT05175105, 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
- 30 participants
- Enrollment target
- 19
- Study locations
Study Summary
Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not 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). Participants will be dosed by age and weight during a double-blind period consisting of an 8-week dose titration period followed by a 12-week fixed-dose period. Participants who complete the double-blind period will be eligible to receive mitapivat for up to 5 years in the open-label extension (OLE) period.
Conditions Studied
Interventions
- DRUG Mitapivat
- DRUG Mitapivat-matching placebo
Study Locations (19)
Other
- Charite - UB - CVK - Medizinische Klinik - Berlin
- Universitair Medisch Centrum Utrecht - Utrecht
- Hospital Universitario Vall d'Hebron - Barcelona
- Hospital Universitario Infantil Nino Jesus - Madrid
Texas
- UT Southwestern Medical Center - Dallas
- Texas Children's Hospital - Houston
California
- Stanford Medicine - Palo Alto
Colorado
- Children's Hospital Colorado - Aurora
Georgia
- Children's Healthcare of Atlanta - Emory - Atlanta
Illinois
- UChicago Medicine - Chicago
Massachusetts
- Boston Children's Hospital - Boston
Michigan
- Children's Hospital of Michigan - Detroit
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2022-06-06 |
| Est. Completion | 2030-01 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05175105
The ClinicalTrials.gov registry entry for NCT05175105 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Agios Pharmaceuticals, which has 44 total studies on file at ClinicalTrials.gov.
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.
NCT05175105 reports 19 study locations spanning 15 distinct geographic areas - top geographies include Other, Texas, California.
Frequently Asked Questions
What is clinical trial NCT05175105 about?
NCT05175105 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 Not Regularly Transfused, Followed by a 5-Year Extension Period". Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving blood transfusions. Participants will be randomized 2:1 to receive either ...
What is the current status of trial NCT05175105?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 30 participants. The study started on 2022-06-06. Estimated completion is 2030-01.
What conditions does trial NCT05175105 study?
This clinical trial studies the following conditions: Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia.
What interventions are being tested in trial NCT05175105?
The interventions under investigation include: Mitapivat (DRUG), Mitapivat-matching placebo (DRUG).
Who is sponsoring clinical trial NCT05175105?
This trial is sponsored by Agios Pharmaceuticals, which has 44 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05175105 being conducted?
This trial has 19 study locations across California, Colorado, Georgia, Illinois, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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