NCT07401823

Open-Label Extension Study to Pioneer Study 6058-SCD-101

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of Pociredir in Participants With Sickle Cell Disease (SCD) Who Have Participated in a Pociredir Study

• Status: Enrolling By Invitation
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Fulcrum Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder study 6058-SCD-101 (NCT05169580). Participants in this study will receive once daily doses of pociredir for up to 48 months.

Detailed description

The first dose of study drug will be administered on Day 1 in the clinic and participants will continue at home dosing once daily (QD). Dosing will occur in the clinic on days where there are clinic visits. Treatment Period clinic visits are planned every other week through Week 12 (Weeks 2, 4, 6, 8, 10, and 12), monthly through Week 24 (Weeks 16, 20, and 24), and then every 12 weeks from Week 24 through Week 192. A final follow-up visit (Week 196) will occur 4 weeks after the final dose of study drug at Week 192. Participants will receive pociredir at the dose level they received in Study 6058-SCD-101 through Week 192, unless data from that study indicates a change to a different optimized dose.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Pociredir	Pociredir Oral Capsules will be administered

Timeline

• Start date: 2026-03-30
• Primary completion: 2030-02-04
• Completion: 2030-07-05
• First posted: 2026-02-11
• Last updated: 2026-04-14

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study