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NCT03548220 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
A Phase 3 study, sponsored by Agios Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 80
- Enrollment target
NCT03548220: Completed Phase 3 study, sponsored by Agios Pharmaceuticals.
NCT03548220 is a Phase 3 study that has completed, run by Agios Pharmaceuticals. The registered enrollment target is 80 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03548220, a Phase 3 study, has completed, sponsored by Agios Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 80 participants
- Enrollment target
Study 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.
Primary Outcome
Hemoglobin response (HR) is defined as a ≥1.5 g/dL (0.93 mmol/L) increase in Hb concentration from baseline that is sustained at 2 or more scheduled assessments at Weeks 16, 20, and 24. The baseline Hb concentration is the average of all available Hb concentrations for a participant during the Screening Period up to the first dose of study treatment. As pre-specified in the protocol, the data for this outcome measure is summarized between the active arm vs the placebo arm (AG-348 5 mg, 20 mg and
Interventions
- DRUG Placebo
- DRUG AG-348
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 80 participants |
| Start Date | 2018-08-09 |
| Est. Completion | 2020-10-09 |
| Phase | Phase 3 |
What the finished NCT03548220 record still lists
NCT03548220 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 80 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT03548220 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03548220 about?
NCT03548220 is a clinical study titled "A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)". 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.
What is the current status of trial NCT03548220?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 80 participants. The study started on 2018-08-09. Estimated completion is 2020-10-09.
What interventions are being tested in trial NCT03548220?
The interventions under investigation include: Placebo (DRUG), AG-348 (DRUG).
Who is sponsoring clinical trial NCT03548220?
This trial is sponsored by Agios Pharmaceuticals, which has 44 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03548220's enrollment target sits among peer trials
80 1755th of 2000 higher than 233 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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