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

Enrolling by invitation · Phase 2

Conditions studied: Sickle Cell Disease

In brief

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.

Key facts

Study ID

NCT07401823

Run by

Fulcrum Therapeutics

People needed

50

Starts

2026-03-30

Expected to finish

2030-07-05

Last updated by the study team

2026-04-14

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants aged ≥18 years and older must have previously participated in and successfully completed Study 6058-SCD-101.
• Participant has signed and dated the informed consent form (ICF) before any study-specific procedures are performed and is willing and able to comply with the study procedures and restrictions.
• Participants who meet all other inclusion and exclusion criteria for this study, and per Investigator's recommendation may continue standard of care as indicated with the exception of hydroxyurea (HU). Participants may continue crizanlizumab, and/or L-glutamine, but must be on a stable dose for at least 6 months.
• Participants, who if female and of childbearing potential, agree to use 2 effective methods of contraception, 1 of which must be highly effective, or practice abstinence starting at the time of the ICF signing to 90 days after the last dose of study drug, and, who if male, should use condoms or practice abstinence from the time of ICF signing to 90 days after the last dose of study drug.
• Documented HbF benefit, as judged by the Investigator, from prior study.
• Participant must meet both of the following laboratory values during Screening:
• Absolute neutrophil count ≥ 1.5 × 10\^9/liter,
• Platelets ≥ 80 × 10\^9/liter
• Absolute reticulocyte count during Screening > 100 × 10\^9/liter.

You may not qualify if…

• Major surgery, serious illness (acute or chronic), infection (clinically significant bacterial, fungal, parasitic or viral infection which requires therapy), fever not resolved within 3 days of onset and requiring treatment, significant bleeding, cerebrovascular accident, or seizure within 14 days prior to signing the ICF and 14 days prior to starting study drug.
• Sickle cell complication requiring more than 24-hour care from a medical provider in hospital or emergency care setting in the 14 days prior to starting study drug.
• Use of medications that are moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A4 or CYP2C8, inhibit P-glycoprotein, breast cancer resistance protein, or multidrug and toxin extrusion protein 2-K, or are substrates of CYP2B6 within 14 days prior to first dose of study drug or anticipated need for any of these medications during the study.
• Participation in any other study with an investigational agent other than pociredir within the past 60 days prior to the first dose of study drug.
• History of bone marrow transplant or hematopoietic stem cell transplant or gene therapies.
• Vaccination in the previous 7 days prior to the first dose of study drug.
• Note: Other protocol specified criteria may apply

Where it is running

• University of Arkansas for Medical Sciences — Little Rock, Arkansas, United States
• University of California, Los Angeles — Los Angeles, California, United States
• Our Lady of the Lake Hospital — Baton Rouge, Louisiana, United States
• Boston Medical Center — Boston, Massachusetts, United States
• Queens Hospital Cancer Center — Jamaica, New York, United States
• Jacobi Medical Center — The Bronx, New York, United States
• University of Texas Houston — Houston, Texas, United States
• Inova Schar Cancer Institute — Fairfax, Virginia, United States

Full record on ClinicalTrials.gov

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