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Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of FTX-6058
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Brief Summary
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of FTX-6058 in participants with sickle cell disease.
Primary Purpose
Study Type
Phase
Eligibility
Gender
Healthy Volunteers
Minimum Age
Maximum Age
Key Inclusion Criteria:
- Participant is 18 to 65 years of age, inclusive at the time informed consent is obtained.
- Participants who meet at least one the following criteria:
- ≥4 episodes of SCD pain crisis over 12 months, or ≥2 over 6 months prior to screening
- ≥2 episodes of SCD pain crisis plus at least one of the following over previous 12 months: i. Acute chest syndrome (ACS) ii. Hepatic or splenic sequestration iii. Priapism c. ≥2 of the following events over the previous 12 months: i. ACS ii. Hepatic or splenic sequestration iii. Priapism d. SCD-related pulmonary arterial hypertension e. SCD-related chronic kidney disease (CKD) f. Meet medical criteria to receive (e.g., post-cerebrovascular accident) but are contraindicated for chronic transfusions (e.g., alloimmunization, transfusion reactions)
- Previous experience with Hydroxyurea (HU) use for at least 6 months at the maximum tolerated dose but have shown to be unresponsive and/or intolerant or ineligible AND
- Previous experience with a stable dose of voxelotor, crizanlizumab, or L-glutamine for at least 6 months but have shown to be unresponsive and/or intolerant or ineligible
- Documented SCD at the time of screening (S/S, S/β0 and S/β+ genotypes only).
- Documented HbF ≤ 20% of total Hb.
- Total Hb ≥ 5.5 g/dL and ≤ 12 g/dL (males) or ≤ 10.6 g/dL (females) at screening.
- Participant must meet both of the following laboratory values at screening:
- Absolute neutrophil count ≥ 1.5 × 10^9 per liter (/L)
- Platelets ≥ 80 × 10^9/L
- Absolute reticulocyte count at screening ≥ 100 x 10^9/L.
Key Exclusion Criteria:
- Sickle cell complication requiring care from a medical provider in the 14 days prior to starting study drug.
- History of bone marrow transplant or human stem cell transplant or gene therapies.
- Participants with a history of severe renal disease defined as estimated glomerular filtration rate < 30 mL/min/1.73m^2. Participants on dialysis of any kind are excluded.
- Participants receiving regularly scheduled transfusions or any participant who has been transfused within 60 days prior to initiating study drug.
- Participant with active malignancy, or history of cancer (except for squamous cell skin cancer, basal cell skin cancer, and stage 0 cervical carcinoma in situ, with no recurrence for the last 5 years), or with an immediate family member with known or suspected familial cancer syndrome. Known presence of a chromosomal abnormality or genetic mutation that may put the participant at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).
- Participant currently on HU, voxelotor, crizanlizumab, and/ or L-glutamine or have received HU, voxelotor, crizanlizumab, and/ or L-glutamine within 60 days prior to initiating study drug.
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Study Stats
Protocol No.
24-5149
Category
Blood Disorders
Genetic and Rare Diseases
Principal Investigator
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