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NCT03559699 · ClinicalTrials.gov registry record · Phase 3
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
A Phase 3 study, sponsored by Agios Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 27
- Enrollment target
NCT03559699: Completed Phase 3 study, sponsored by Agios Pharmaceuticals.
NCT03559699 is a Phase 3 study that has completed, run by Agios Pharmaceuticals. The registered enrollment target is 27 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03559699, a Phase 3 study, has completed, sponsored by Agios Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 27 participants
- Enrollment target
Study Summary
Study AG348-C-007 was a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, participants with pyruvate kinase (PK) deficiency, who were regularly receiving blood transfusions. The study was composed of two parts. During Part 1, Dose Optimization Period, participants started on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose of AG-348 was sequentially increased to 20 mg twice a day, followed by 50 mg twice a day depending on their tolerance. During Part 2, Fixed-Dose Period, participants received AG-348 at their optimized dose from Part 1.
Primary Outcome
Reduction in transfusion burden is defined as a ≥33% reduction in the number of RBC units transfused during the Fixed Dose Period standardized to 24 weeks compared with the historical transfusion burden standardized to 24 weeks (Standardized Control Period). The on-study (Fixed Dose Period) transfusion burden was calculated as the total number of transfused RBC units received in the Fixed Dose Period standardized to 24 weeks.
Interventions
- DRUG AG-348
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 27 participants |
| Start Date | 2018-06-26 |
| Est. Completion | 2020-11-12 |
| Phase | Phase 3 |
What the finished NCT03559699 record still lists
NCT03559699 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 27 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which AG-348 is the first listed.
NCT03559699 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03559699 about?
NCT03559699 is a clinical study titled "A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)". Study AG348-C-007 was a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, participants with pyruvate kinase (PK) deficiency, who were regularly receiving blood transfusions. The study was composed of two parts. During Part 1, D...
What is the current status of trial NCT03559699?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 27 participants. The study started on 2018-06-26. Estimated completion is 2020-11-12.
What interventions are being tested in trial NCT03559699?
The interventions under investigation include: AG-348 (DRUG).
Who is sponsoring clinical trial NCT03559699?
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 NCT03559699's enrollment target sits among peer trials
27 1936th of 2000 higher than 64 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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