9 completed, 3 active not recruiting, 1 enrolling by invitation, 1 not yet recruiting. 5 Phase 3, 2 Phase 1, 2 Phase 2, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 5 | Completed 3, Active not recruiting 2 |
| Phase 1 | 2 | Completed 1, Not yet recruiting 1 |
| Phase 2 | 2 | Completed 1, Suspended 1 |
| Phase 4 | 1 | Enrolling by invitation 1 |
Follow Pyruvate Kinase Deficiency of Red Cells: new trials by email Literature Review of Pyruvate Kinase Deficiency of Red Cells trials Research Report on Pyruvate Kinase Deficiency of Red Cells
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study Vitamin C - Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency | Phase 1 | Not yet recruiting | University of Utah | 3 | 2026-07-15 | 2026-05-28 |
| Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency RP-L301 - Pyruvate Kinase Deficiency | Phase 2 | Suspended | Rocket Pharmaceuticals Inc. | 10 | 2026-04-01 | 2026-04-16 |
| A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study Mitapivat - Pyruvate Kinase Deficiency, Anemia, Hemolytic | Phase 4 | Enrolling by invitation | Agios Pharmaceuticals, Inc. | 59 | 2023-07-06 | 2026-09-29 |
| 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 Mitapivat, Mitapivat-matching placebo - Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 49 | 2022-06-08 | 2026-09-28 |
| 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 Mitapivat, Mitapivat-matching placebo - Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | Phase 3 | Active not recruiting | Agios Pharmaceuticals, Inc. | 30 | 2022-06-06 | 2026-08-27 |
| Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms Blood Draw - Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia | Completed | Massachusetts General Hospital | 18 | 2022-02-01 | 2026-05-19 | |
| RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) Data collection from EHR. - Sickle Cell Disease, Thalassemia | Recruiting | Hospital Universitari Vall d'Hebron Research Institute | 32564 | 2021-11-30 | 2024-01-19 | |
| Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO) Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia | Terminated | Agios Pharmaceuticals, Inc. | 15 | 2021-07-02 | 2023-09-07 | |
| Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008 Pyruvate Kinase Deficiency | Completed | Agios Pharmaceuticals, Inc. | 13 | 2021-07-02 | 2023-10-16 | |
| Gene Therapy for Pyruvate Kinase Deficiency (PKD) RP-L301 - Pyruvate Kinase Deficiency | Phase 1 | Completed | Rocket Pharmaceuticals Inc. | 4 | 2020-07-06 | 2025-09-05 |
| Pyruvate Kinase Deficiency Epidemiological Study (PIECE) Pyruvate Kinase Deficiency | Completed | CENTOGENE GmbH Rostock | 75 | 2020-01-13 | 2023-02-09 | |
| Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 Mitapivat - Pyruvate Kinase Deficiency | Phase 3 | Completed | Agios Pharmaceuticals, Inc. | 90 | 2019-03-21 | 2025-11-18 |
| A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Placebo, AG-348 - Pyruvate Kinase Deficiency, Anemia, Hemolytic | Phase 3 | Completed | Agios Pharmaceuticals, Inc. | 80 | 2018-08-09 | 2022-05-24 |
| A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) AG-348 - Pyruvate Kinase Deficiency, Anemia, Hemolytic | Phase 3 | Completed | Agios Pharmaceuticals, Inc. | 27 | 2018-06-26 | 2022-01-04 |
| Pyruvate Kinase Deficiency Global Longitudinal Registry Pyruvate Kinase Deficiency | Active not recruiting | Agios Pharmaceuticals, Inc. | 500 | 2018-04-23 | 2026-09-28 | |
| A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency AG-348 - Pyruvate Kinase Deficiency | Phase 2 | Completed | Agios Pharmaceuticals, Inc. | 52 | 2015-06-26 | 2026-05-01 |
| Pyruvate Kinase Deficiency Natural History Study Pyruvate Kinase Deficiency, Congenital Non-Spherocytic Hemolytic Anemia | Completed | Boston Children's Hospital | 254 | 2013-12-01 | 2020-05-22 |
Anemia, Hemolytic (8) · Anemia (6) · Anemia, Hemolytic, Congenital Nonspherocytic (3) · Hematologic Diseases (3) · Anemia, Sickle Cell (2) · Metabolic Diseases (2) · Thalassemia (2) · Anemia, Hemolytic, Congenital (1) · Anemia, Sideroblastic (1) · Cystinosis (1) · Glucosephosphate Dehydrogenase Deficiency (1) · Hemoglobinopathies (1) · Hemolysis (1) · Iron Metabolism Disorders (1)
Hemic and Lymphatic Diseases (14) · Hematologic Diseases (11) · Anemia (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Anemia, Hemolytic, Congenital (6) · Anemia, Hemolytic (4) · Nutritional and Metabolic Diseases (4) · Bone Marrow Diseases (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Carbohydrate Metabolism, Inborn Errors (1) · Hemoglobinopathies (1)
mitapivat (7) · Ascorbic Acid (1) · Blood Specimen Collection (1) · Methods (1) · Therapeutics (1)
Agios Pharmaceuticals, Inc. (10) · Rocket Pharmaceuticals Inc. (2) · Boston Children's Hospital (1) · CENTOGENE GmbH Rostock (1) · Hospital Universitari Vall d'Hebron Research Institute (1) · Massachusetts General Hospital (1) · University of Utah (1)
United States (15) · Netherlands (10) · Canada (9) · Spain (9) · Italy (8) · United Kingdom (8) · France (7) · Germany (6) · Denmark (5) · Switzerland (5) · Turkey (Türkiye) (5) · Czechia (4) · Thailand (4) · Brazil (3) · Ireland (3)
Ami Shah, MD (2) · Eva Gallagher, VP, Medical Affairs (1) · Hanny Al-Samkari, MD (1) · Julian Sevilla Navarro, MD, PhD (1) · Maria Chitty-Lopez, MD (1) · Peter Bauer, M.D. (1) · Petros Kountouris, PhD (1)
PMID 31974203, PMID 36594181, PMID 38330179, PMID 31483964, PMID 34470054, PMID 35417638, PMID 35988546, PMID 36958777, PMID 37943362
2015 (1), 2017 (1), 2018 (1)