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Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir

Testing Pociredir: Checking safety, side effects, and body response.

Recruiting
18-65 years
All
Phase 1

Pociredir Study for Sickle Cell Disease

This study checks if a new drug called Pociredir is safe and works well for people with sickle cell disease (SCD). Sickle cell disease is a condition where red blood cells become shaped like a sickle, causing pain and other problems. We're looking at how the body processes the drug (pharmacokinetics) and how it affects the body (pharmacodynamics). Participants must be between 18 and 65 years old. They will take Pociredir for 12 weeks, then have 4 weeks of follow-up. Different groups will get different doses, starting at 6 mg per day, either 2 mg or 12 mg, and possibly higher. The study aims to see how safe the drug is and if it helps increase fetal hemoglobin, which might reduce sickle cell problems.

  • The study lasts for 16 weeks, with 12 weeks of taking the drug and 4 weeks of follow-up.
  • Participants should not have had certain treatments like Hydroxyurea or other SCD drugs recently.
  • Eligibility requires certain health conditions related to SCD and specific lab results.
Study details
    Sickle Cell Disease
    Sickle Cell Anemia

NCT05169580

Fulcrum Therapeutics

14 June 2025

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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