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NCT04610866 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
A Phase 1 study of Sickle Cell Disease and Hemolytic Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Active
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 1
- Study location
NCT04610866: Active Phase 1 study of Sickle Cell Disease and Hemolytic Anemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT04610866 is a Phase 1 study of Sickle Cell Disease and Hemolytic Anemia that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 15 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04610866, a Phase 1 study of Sickle Cell Disease and Hemolytic Anemia, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work. Researchers want to see if a drug called mitapivat can help people with SCD. Objective: To test the long-term tolerability and safety of mitapivat (or AG-348) in people with SCD. Eligibility: Adults age 18-70 with SCD who took part in and benefited from NIH study #19H0097. Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. They will have an electrocardiogram to test heart function. Participants will repeat some of the screening tests during the study. Participants will complete 6-minute walk tests to measure mobility and function. They will have transthoracic echocardiograms to measure heart and lung function. They will have dual-energy X-ray absorptiometry scans to measure bone health. They will complete online questionnaires that measure their overall health and well-being. Participants will take the study drug in the form of a tablet twice a day. Participants will keep a study diary. They will record any symptoms they may have. Participation will last for about 54 weeks. After 48 weeks, participants can either keep taking the study drug for 48 more weeks or be tapered off of the study drug to complete the study. Those who are on the study for 1 year will have 10 study visits. Those who are on the study for 2 years will have 14 study visits.
Primary Outcome
Frequency and severity of AEs and changes in clinical and laboratory parameters over 48 weeks of maintenance therapy with mitapivat.
Conditions Studied
Interventions
- DRUG Mitapivat
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2020-12-09 |
| Est. Completion | 2028-02-28 |
| Phase | Phase 1 |
What the registry record for NCT04610866 still lists
NCT04610866 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (95% lower).
The record links to 2 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which Mitapivat is the first listed.
NCT04610866 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT04610866 about?
NCT04610866 is a clinical study titled "Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat". Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work. Researchers want to see if a drug called mitapivat can help people with SCD. Objective: To test the long-term...
What is the current status of trial NCT04610866?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2020-12-09. Estimated completion is 2028-02-28.
What conditions does trial NCT04610866 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Hemolytic Anemia.
What interventions are being tested in trial NCT04610866?
The interventions under investigation include: Mitapivat (DRUG).
Who is sponsoring clinical trial NCT04610866?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04610866 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04610866's enrollment target sits among peer trials
15 185th of 213 higher than 24 of 213 other Sickle Cell Disease trials
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Each bar is a band; taller bars hold more other Sickle Cell Disease 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.
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