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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pociredir | Experimental | Participants will receive Pociredir orally once daily (QD) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pociredir | Drug | Pociredir Oral Capsules will be administered |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
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 reticulocyte count during Screening > 100 × 10^9/liter.
Exclusion Criteria:
Note: Other protocol specified criteria may apply
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | United States | ||
| University of California, Los Angeles |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D017086 | beta-Thalassemia |
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006425 | Hemic and Lymphatic Diseases |
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| 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 |
| Change from Baseline in Haptoglobin | Baseline (Day 1), and Up to Week 192 |
| Change from Baseline in Reticulocyte count | Baseline (Day 1), and Up to Week 192 |
| Number of participants reporting SCD-related complications | Up to Week 192 |
| Annualized rate of Vaso-occlusive episode (VOE) | through end of month 48 |
| Los Angeles |
| California |
| 90095 |
| United States |
| Our Lady of the Lake Hospital | Baton Rouge | Louisiana | 70808 | United States |
| Boston Medical Center | Boston | Massachusetts | 02118 | United States |
| Queens Hospital Cancer Center | Jamaica | New York | 11432 | United States |
| Jacobi Medical Center | The Bronx | New York | 10461 | United States |
| University of Texas Houston | Houston | Texas | 77030 | United States |
| Inova Schar Cancer Institute | Fairfax | Virginia | 22031 | United States |
| D006453 |
| Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013789 | Thalassemia |