At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 80 enrolled
Drug / intervention
Placebo +1 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Subjects With Pyruvate Kinase Deficiency
In Brief
A Phase 3 clinical trial evaluating Placebo and AG-348 for Pyruvate Kinase Deficiency and Anemia, Hemolytic. Completed, enrolled 80 participants across 46 sites in 16 countries.
Detailed Summary
Study AG348-C-006 evaluated the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase (PK) deficiency, who were not regularly receiving blood transfusions. Participants were randomized 1:1 to receive either AG-348 or a matching placebo.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPyruvate Kinase Deficiency, Anemia, Hemolytic
CountriesBrazil, Canada, Czechia, Denmark, France, Germany, Italy, Japan, Netherlands, South Korea, Spain, Switzerland, Thailand, Turkey (Türkiye), United Kingdom, United States
Collaborators--
Timeline
Phase 3CompletedFinished
20192020202120222023202420252026
First PostedJun 2018
Enrollment StartAug 2018
Primary CompletionOct 2020
TodayJul 2026
First PostedJun 7, 2018
Enrollment StartAug 9, 2018
Primary CompletionOct 9, 2020
TodayJul 2, 2026
Enrollment to primary: 2.2 yearsPosted 8.1 years ago
Interventions
Placebodrug
Placebo matching AG-348 tablets, administered to maintain the blind.
AG-348drug
AG-348 tablets.