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Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Pyruvate Kinase Deficiency Clear all
Completed Phase 2 Interventional Results available

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

NCT02476916
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
AG-348
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Palo Alto, California • Boston, Massachusetts • Detroit, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Jul 22, 2026, 10:43 AM EDT
Completed Phase 1 Interventional

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

NCT04105166
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 50 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Jul 22, 2026, 10:43 AM EDT
Completed No phase listed Observational

Pyruvate Kinase Deficiency Natural History Study

NCT02053480
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Congenital Non-Spherocytic Hemolytic Anemia
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
254 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
19
States / cities
Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 16 more
Source: ClinicalTrials.gov public record
Updated May 21, 2020 · Synced Jul 22, 2026, 10:43 AM EDT
Completed No phase listed Observational

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

NCT04995315
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
Not listed
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 15, 2023 · Synced Jul 22, 2026, 10:43 AM EDT
Active, not recruiting No phase listed Observational

Pyruvate Kinase Deficiency Global Longitudinal Registry

NCT03481738
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
Not listed
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
15
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Orange, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 22, 2026, 10:43 AM EDT
Completed No phase listed Observational

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

NCT04902833
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Hereditary Hemolytic Anemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasm, Clonal Myeloid Neoplasm, Myeloproliferative Neoplasm, Acute Myeloid Leukemia, Clonal Cytopenia of Undetermined Significance, Other Clonal Myeloid Neoplasm, Unexplained Coombs-negative Non-immune Hemolytic Anemia
Interventions
Blood Draw
Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Jul 22, 2026, 10:43 AM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

NCT03548220
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Anemia, Hemolytic
Interventions
Placebo, AG-348
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
12
States / cities
Little Rock, Arkansas • Atlanta, Georgia • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated May 23, 2022 · Synced Jul 22, 2026, 10:43 AM EDT
Suspended Phase 2 Interventional

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

NCT06422351
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 22, 2026, 10:43 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

NCT03559699
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Anemia, Hemolytic
Interventions
AG-348
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
4
States / cities
Oakland, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 3, 2022 · Synced Jul 22, 2026, 10:43 AM EDT
Completed No phase listed Observational

Pyruvate Kinase Deficiency Epidemiological Study (PIECE)

NCT03866590
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
Not listed
Lead sponsor
CENTOGENE GmbH Rostock
Industry
Eligibility
5 Years to 30 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Pembroke Pines, Florida
Source: ClinicalTrials.gov public record
Updated Feb 8, 2023 · Synced Jul 22, 2026, 10:43 AM EDT
Terminated No phase listed Observational

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

NCT04964323
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic
Interventions
Not listed
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 6, 2023 · Synced Jul 22, 2026, 10:43 AM EDT
Active, not recruiting Phase 3 Interventional

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

NCT05144256
View directory record Official record
Conditions
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Interventions
Mitapivat, Mitapivat-matching placebo
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
9
States / cities
Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 22, 2026, 10:43 AM EDT
Completed Phase 3 Interventional Results available

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

NCT03853798
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
12
States / cities
Phoenix, Arizona • Oakland, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Jul 22, 2026, 10:43 AM EDT
Active, not recruiting Phase 3 Interventional

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
View directory record Official record
Conditions
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Interventions
Mitapivat, Mitapivat-matching placebo
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
11
States / cities
Palo Alto, California • Aurora, Colorado • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 22, 2026, 10:43 AM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Jul 22, 2026, 10:43 AM EDT