Sickle Cell Disease
Conditions
Keywords
FTX-6058, Pociredir, Fetal hemoglobin, Sickle Cell Disease, Open-label, Anemia, Sickle Cell, Hematologic diseases
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.
Detailed description
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.
Interventions
DRUGPociredir
Pociredir Oral Capsules will be administered
Sponsors
Fulcrum Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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: 1. Absolute neutrophil count ≥ 1.5 × 10\^9/liter, 2. Platelets ≥ 80 × 10\^9/liter * Absolute reticulocyte count during Screening \> 100 × 10\^9/liter.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Up to Week 196 | Laboratory assessments including hematology, coagulation, serum chemistry and electrolytes, lipid panel, SCD characterization, serology, urinalysis and pregnancy tests will be performed. |
Secondary
| Measure | 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 |
| 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 |
Countries
United States
Outcome results
None listed