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ClinicalTrials.gov public records Last synced May 21, 2026, 11:54 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–14 of 14 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Pyruvate Kinase Deficiency Clear all
Completed Phase 3 Interventional Results available
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 May 21, 2026, 11:54 PM EDT
Active, not recruiting No phase listed Observational
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 Apr 22, 2026 · Synced May 21, 2026, 11:54 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 11:54 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 11:54 PM EDT
Completed Phase 3 Interventional Results available
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 May 21, 2026, 11:54 PM EDT
Completed Phase 1 Interventional
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 May 21, 2026, 11:54 PM EDT
Completed Phase 3 Interventional Results available
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 May 21, 2026, 11:54 PM EDT
Active, not recruiting Phase 3 Interventional
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 Apr 23, 2026 · Synced May 21, 2026, 11:54 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 11:54 PM EDT
Completed No phase listed Observational
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 May 21, 2026, 11:54 PM EDT
Suspended Phase 2 Interventional
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 May 21, 2026, 11:54 PM EDT
Terminated No phase listed Observational
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 May 21, 2026, 11:54 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 11:54 PM EDT
Active, not recruiting Phase 3 Interventional
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 Apr 23, 2026 · Synced May 21, 2026, 11:54 PM EDT