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NCT04000165 · ClinicalTrials.gov registry record · Early Phase 1
A Dose-Finding Study of AG-348 in Sickle Cell Disease
A Early Phase 1 study, sponsored by National Institutes of Health Clinical Center (CC).
- Completed
- Registry status
- Early Phase 1
- Development phase
- 17
- Enrollment target
NCT04000165: Completed Early Phase 1 study, sponsored by National Institutes of Health Clinical Center (CC).
NCT04000165 is a Early Phase 1 study that has completed, run by National Institutes of Health Clinical Center (CC). The registered enrollment target is 17 participants, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (68% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04000165, a Early Phase 1 study, has completed, sponsored by National Institutes of Health Clinical Center (CC).
- COMPLETED
- Registry status
- Early Phase 1
- Development phase
- 17 participants
- Enrollment target
Study Summary
Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells. Researchers are investigating the safety and efficacy of an investigational medicine called AG-348 (mitapivat sulfate) to determine if it will help people with SCD. Objective: To test the tolerability and safety of AG-348 in people with SCD. Eligibility: People ages 18 and older with SCD. Design: Participants will have 8 visits over approximately 14 weeks. At the first visit participants will be screened with a medical history, a physical exam, blood and urine testing, and an EKG. During the following 5 visits, participants will stay at the clinic for 1 night each. Participants will take study drug in increasing doses up to visit 6, after which the drug will be tapered off. All visits will include physical exam, blood, and urine tests. The last visit will occur 4 weeks after stopping the drug. Participants will provide DNA from the blood samples they provide. The DNA will be tested for an inherited gene that can cause differences in response to the study drug. Researchers may also test other genes to see if they can find any genes that interact with SCD.
Primary Outcome
To assess the clinical safety and tolerability of multiple escalating doses of AG-348, an allosteric activator of the enzyme pyruvate kinase, in subjects with stable sickle cell disease (SCD). Safety and tolerability were assessed by frequency and severity of adverse events (AEs) using Common Terminology Criteria for Adverse Events (CTCAE) 5.0.
Interventions
- DRUG AG-348
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2019-07-11 |
| Est. Completion | 2021-06-21 |
| Phase | Early Phase 1 |
What the finished NCT04000165 record still lists
NCT04000165 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 17 participants, a relatively small participant target, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (68% lower).
The record links to 0 conditions, and to 1 intervention - of which AG-348 is the first listed.
NCT04000165 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04000165 about?
NCT04000165 is a clinical study titled "A Dose-Finding Study of AG-348 in Sickle Cell Disease". Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells. Researchers are investigating the safety and efficacy of an investigational medicine called AG-348 (mitapivat sulfate) to determine if it will help people with SC...
What is the current status of trial NCT04000165?
This trial is currently completed. It is a Early Phase 1 study. The enrollment target is 17 participants. The study started on 2019-07-11. Estimated completion is 2021-06-21.
What interventions are being tested in trial NCT04000165?
The interventions under investigation include: AG-348 (DRUG).
Who is sponsoring clinical trial NCT04000165?
This trial is sponsored by National Institutes of Health Clinical Center (CC), which has 338 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04000165's enrollment target sits among peer trials
17 1350th of 2000 higher than 632 of 2,000 other Early Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Early Phase 1 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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