Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Jul 22, 2026, 2:08 PM EDT

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Showing 1–3 of 3 matching trials from the live ClinicalTrials.gov search.
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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
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
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