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NCT05828108 · ClinicalTrials.gov registry record · Phase 1
Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia
A Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
NCT05828108 is a Phase 1 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 15 participants.
The verdict
NCT05828108, a Phase 1 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
Study Summary
Background: Diamond-Blackfan anemia (DBA) is an inherited disease that affects the bone marrow. People with DBA have chronic anemia that can be severe. Many must have frequent transfusions of red blood cells. Current treatments for DBA all have risks of serious side effects. Better treatments are needed. Objective: To test a new drug (bitopertin) in people with DBA. Eligibility: People aged 18 or older with DBA. Design: Participants will be screened. They will have a physical exam; they will have blood tests and a test of their heart function. They will have a bone marrow biopsy: An area of their hip will be numbed, and a needle will be inserted to remove a sample of tissue from inside the bone. Bitopertin is a pill taken by mouth. Participants will take the drug once a day every day for 8 months. They will start with a low dose of the drug; the dosage may increase gradually over time. They will keep a diary to record each dose. Participants will have blood tests every 4 weeks. This may be done in the clinic. Participants may also have telehealth visits; they can have blood drawn at a local lab and sent to the researchers. The bone marrow biopsy and other tests will be repeated after 8 months. Participants who have a positive response to bitopertin will be invited to enter an extended phase of the trial. They may continue to take the drug for 3 more years. Those who choose not to continue in the extended phase may have a follow-up visit 6 months after they stop taking the drug.
Interventions
- DRUG Bitopertin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2023-07-25 |
| Est. Completion | 2025-08-28 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05828108
The ClinicalTrials.gov registry entry for NCT05828108 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Heart, Lung, and Blood Institute (NHLBI), which has 501 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Bitopertin is the first listed.
NCT05828108 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT05828108 about?
NCT05828108 is a clinical study titled "Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia". Background: Diamond-Blackfan anemia (DBA) is an inherited disease that affects the bone marrow. People with DBA have chronic anemia that can be severe. Many must have frequent transfusions of red blood cells. Current treatments for DBA all have risks of serious side effects. Better treatments are n...
What is the current status of trial NCT05828108?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2023-07-25. Estimated completion is 2025-08-28.
What interventions are being tested in trial NCT05828108?
The interventions under investigation include: Bitopertin (DRUG).
Who is sponsoring clinical trial NCT05828108?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
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