NCT07431398

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

A Phase 1, Open-Label Study to Assess Pharmacokinetics After Single Doses of Pociredir in Participants With Sickle Cell Disease

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Fulcrum Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).

Detailed description

The study is designed to assess the pharmacokinetics of the tablet formulation of pociredir under fasted conditions in up to approximately 12 study participants with SCD (Fasted Cohort). An additional cohort of up to approximately 12 study participants with SCD may be enrolled to evaluate the potential effect of food on the PK of the tablet formulation (Fed Cohort). The fasted cohort will be conducted first. The study will include screening period: Day -28 to Day -2; In-patient confinement period: Days -1 to 3; check-in on day -1; single dose of pociredir on day 1; discharge on Day 3; outpatient visit: Day 4; end of study (EOS) visit: Days 8-11 Eligible participants who meet all inclusion and none of the exclusion criteria will be admitted to the clinical site on Day -1, the day before dosing. Participants may be discharged on Day 3 following completion of 48-hour PK sampling or may remain in-clinic through Day 4 if needed. Fasted Cohort: Participants will fast for at least 10 hours prior to dosing and remain fasted for 4 hours post-dose. Water intake will be restricted for 1 hour before and after administration of the investigational medicinal product (IMP). Fed Cohort (if conducted): Participants will fast for at least 10 hours prior to breakfast and will receive a standard high-fat breakfast starting 30 minutes before dosing. Pharmacokinetic samples will be collected up to 72 hours post-dose. Participants are required to remain in-clinic for the first 48 hours post-dose and may return to the site for subsequent Outpatient and EOS visits.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Pociredir	Pociredir tablet formulation

Timeline

• Start date: 2025-12-13
• Primary completion: 2026-05-31
• Completion: 2026-06-01
• First posted: 2026-02-24
• Last updated: 2026-03-30

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study