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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Pociredir Oral Capsules will be administered
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
Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to Week 196
Number of participants with clinically significant changes in 12-lead Electrocardiogram (ECGs)
Time frame: Up to Week 196
Number of participants with clinically significant changes in Vital signs
Time frame: 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.
Time frame: Up to Week 196
Change from Baseline in percent Fetal hemoglobin (HbF)
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in percent Reticulocytes
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Red cell distribution width
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Unconjugated bilirubin
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Lactate dehydrogenase (LDH)
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Haptoglobin
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Reticulocyte count
Time frame: Baseline (Day 1), and Up to Week 192
Number of participants reporting SCD-related complications
Time frame: Up to Week 192
Annualized rate of Vaso-occlusive episode (VOE)
Time frame: through end of month 48