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

NCT07401823 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: 6058-SCD-102
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 8

Brief 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.

Conditions

Sickle Cell Disease

Keywords

FTX-6058; Pociredir; Fetal hemoglobin; Sickle Cell Disease; Open-label; Anemia, Sickle Cell; Hematologic diseases

Outcome Measures

Primary Outcomes (4)

• Number of participants reporting Treatment Emergent Adverse Events (TEAEs)

  Up to Week 196

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

  Up to Week 196

• Number of participants with clinically significant changes in Vital signs

  Up to Week 196

• Number of participants with clinically significant changes in Clinical laboratory tests

  Laboratory assessments including hematology, coagulation, serum chemistry and electrolytes, lipid panel, SCD characterization, serology, urinalysis and pregnancy tests will be performed.

  Up to Week 196

Secondary Outcomes (9)

• Change from Baseline in percent Fetal hemoglobin (HbF)

  Baseline (Day 1), and Up to Week 192

• Change from Baseline in percent Reticulocytes

  Baseline (Day 1), and Up to Week 192

• Change from Baseline in Red cell distribution width

  Baseline (Day 1), and Up to Week 192

• Change from Baseline in Unconjugated bilirubin

  Baseline (Day 1), and Up to Week 192

• Change from Baseline in Lactate dehydrogenase (LDH)

  Baseline (Day 1), and Up to Week 192

Study Arms (1)

Pociredir

EXPERIMENTAL

Participants will receive Pociredir orally once daily (QD)

Drug: Pociredir

Interventions

Pociredir DRUG

Pociredir Oral Capsules will be administered

Also known as: FTX-6058

Pociredir

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• Fulcrum Therapeutics: lead

Study Sites (8)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

University of California, Los Angeles

Los Angeles, California, 90095, United States

Location

Our Lady of the Lake Hospital

Baton Rouge, Louisiana, 70808, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Queens Hospital Cancer Center

Jamaica, New York, 11432, United States

Location

Jacobi Medical Center

The Bronx, New York, 10461, United States

Location

University of Texas Houston

Houston, Texas, 77030, United States

Location

Inova Schar Cancer Institute

Fairfax, Virginia, 22031, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; beta-Thalassemia; Hematologic Diseases

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Thalassemia

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2026
• First Posted: February 11, 2026
• Study Start: March 30, 2026
• Primary Completion (Estimated): February 4, 2030
• Study Completion (Estimated): July 5, 2030
• Last Updated: April 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)