# 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

> **NCT05175105** · PHASE3 · ACTIVE_NOT_RECRUITING · sponsor: **Agios Pharmaceuticals, Inc.** · enrollment: 30 (actual)

## Conditions studied

- Pediatric Pyruvate Kinase Deficiency
- Pediatric Hemolytic Anemia

## Interventions

- **DRUG:** Mitapivat
- **DRUG:** Mitapivat-matching placebo

## Key facts

- **NCT ID:** NCT05175105
- **Lead sponsor:** Agios Pharmaceuticals, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2022-06-06
- **Primary completion:** 2024-12-13
- **Final completion:** 2030-01
- **Target enrollment:** 30 (ACTUAL)
- **Last updated:** 2026-04-24


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05175105

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05175105, "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". Retrieved via AI Analytics 2026-06-09 from https://api.ai-analytics.org/clinical/NCT05175105. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*