Trials / Completed
CompletedNCT03548220
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
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
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 80 (actual)
- Sponsor
- Agios Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Placebo | Placebo matching AG-348 tablets, administered to maintain the blind. |
| DRUG | AG-348 | AG-348 tablets. |
Timeline
- Start date
- 2018-08-09
- Primary completion
- 2020-10-09
- Completion
- 2020-10-09
- First posted
- 2018-06-07
- Last updated
- 2022-05-24
- Results posted
- 2022-05-24
Locations
46 sites across 16 countries: United States, Brazil, Canada, Czechia, Denmark, France, Germany, Italy, Japan, Netherlands, South Korea, Spain, Switzerland, Thailand, Turkey (Türkiye), United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03548220. Inclusion in this directory is not an endorsement.