Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Active Phase 3 study of Pediatric Pyruvate Kinase Deficiency and Pediatric Hemolytic Anemia, sponsored by Agios Pharmaceuticals.
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, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower). The trial reports 19 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
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.
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 |
What the registry record for NCT05175105 still lists
NCT05175105 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% 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.
NCT05175105 lists 19 locations in 15 states (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.
Learn More About Clinical Trials
Similar trials for Pediatric Pyruvate Kinase Deficiency
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05175105's enrollment target sits among peer trials
30 1918th of 2000 higher than 68 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00977977 · 30 participants · Phase 2
Rituximab Plus Cyclosporine in Idiopathic Membranous Nephropathy
- NCT01459107 · 30 participants · Phase 2
Human Upper Extremity Allotransplantation
- NCT01666080 · 30 participants · NA
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
- NCT01720836 · 30 participants · Phase 1
Study of the Immune Response of MUC1 (Mucin1) Peptide Vaccine for Non-small Cell Lung Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI