By InvitationPhase 2

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

United States50 participantsStarted 2026-03-30

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Exclusion criteria

. Absolute neutrophil count ≥ 1.5 × 10\^9/liter,
. Platelets ≥ 80 × 10\^9/liter

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of participants reporting Treatment Emergent Adverse Events (TEAEs)

Timeframe: Up to Week 196

Number of participants with clinically significant changes in 12-lead Electrocardiogram (ECGs)

Timeframe: Up to Week 196

Number of participants with clinically significant changes in Vital signs

Timeframe: Up to Week 196

Number of participants with clinically significant changes in Clinical laboratory tests

Timeframe: Up to Week 196

Trial details

NCT IDNCT07401823

SponsorFulcrum Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2030-02-04

View on ClinicalTrials.gov More Sickle Cell Disease trials

Plain-language summary

What they're measuring

Number of participants reporting Treatment Emergent Adverse Events (TEAEs)

Timeframe: Up to Week 196

Number of participants with clinically significant changes in 12-lead Electrocardiogram (ECGs)

Timeframe: Up to Week 196

Number of participants with clinically significant changes in Vital signs

Timeframe: Up to Week 196

Number of participants with clinically significant changes in Clinical laboratory tests

Timeframe: Up to Week 196