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FTX-6058- Two Dosing Periods · 1 trial · 2 indications
To evaluate the safety and tolerability of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease based on the frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and clinically significant laboratory test results, electrocardiograms (ECGs), and vital signs.
| Arm | Type | Description |
|---|---|---|
| Single Ascending Dose (SAD) cohorts in Healthy Subjects (Part A) | EXPERIMENTAL | Subjects will be randomized to receive a single dose of FTX-6058 or placebo. Cohorts 1 and 2 will enroll 5 subjects per cohort randomized 3:2. Cohorts 3-8 will enroll 7 subjects per cohort randomized 5:2. Planned doses are 2 mg (Cohort 1), 4 mg (Cohort 2), 10 mg (Cohort 3), 20 mg (Cohort 4), 30 mg (Cohort 5), 40 mg (Cohort 6), 60 mg (Cohort 7), and 90 mg (Cohort 8). |
| Multiple Ascending Dose (MAD) cohorts in Healthy Subjects (Part B) | EXPERIMENTAL | Subjects will be randomized 3:1 to receive once daily FTX-6058 or placebo by mouth for 14 days. Up to 6 cohorts of 8 subjects per cohort will be enrolled. Planned doses are 2 mg (Cohort 1), 6 mg (Cohort 2), 10 mg (Cohort 3), 20 mg (Cohort 4), 30 mg (Cohort 5), and 40 mg (Cohort 6). |
| Pilot Food Effect Cohort in Healthy Subjects (Part C) | EXPERIMENTAL | Ten subjects will be randomized to receive a single 20 mg dose of FTX-6058 with and without a high-fat meal with a washout period of 4 days. |
| Potential for CYP3A Induction in Healthy Subjects (Part D) | EXPERIMENTAL | Sixteen subjects will receive 3 mg Midazolam once by mouth on Day 1. On Days 3-12, subjects will receive FTX-6058 by mouth once daily. On Day 12, a second dose of 3 mg Midazolam will be given once by mouth. The dose of FTX-6058 will be the highest tolerated dose from Part B. |
| Multiple Dose Cohort in Sickle Cell Disease Subjects (Part E) | EXPERIMENTAL | Subjects will be randomized 3:1 to receive FTX-6058 or placebo once daily by mouth for 14 days. Up to 8 subjects will be enrolled. The planned dose is 6mg. |
| Name | Type | Description |
|---|---|---|
| FTX-6058/placebo oral capsule(s) | DRUG | Subjects will receive FTX-6058 or matching placebo. |
| FTX-6058 - Two Dosing Periods | DRUG | Subjects will receive FTX-6058 |
| FTX-6058 / Midazolam Syrup | DRUG | Subjects will receive FTX-6058 and midazolam syrup |
Inclusion Criteria: Healthy Subjects 1. Healthy male / female subjects, 18 to 55 years of age, inclusive at screening. 2. Good health, based upon the opinion of the investigator and the results of medical history, physical examination, vital signs, ECG, and laboratory profiles of both blood and uri...
FTX-6058 is an investigational small molecule being studied for use in healthy adult subjects and in patients with sickle cell disease. It is in Phase 1 clinical development and has received Fast Track designation from the FDA. The drug is being developed by Fulcrum Therapeutics.
FTX-6058 is a small molecule developed by Fulcrum Therapeutics. Its specific molecular target has not been disclosed in the available information. The drug is being investigated for its safety, tolerability, and pharmacokinetics in healthy subjects and in patients with sickle cell disease.
FTX-6058 is being developed by Fulcrum Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol FULC. The company is conducting clinical research on this investigational small molecule for potential use in sickle cell disease.
FTX-6058 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA. The drug has received Fast Track designation, which is intended to facilitate development and expedite review for serious conditions.
FTX-6058 has one completed Phase 1 clinical trial, identified as NCT04586985, titled "Safety, Tolerability and Pharmacokinetics of FTX-6058." This trial enrolled 109 participants, including healthy adult subjects and patients with sickle cell disease, and was conducted in the United States.
FTX-6058 is the primary name for this investigational drug. No alternative names have been reported in the available information. It is a small molecule being studied by Fulcrum Therapeutics for potential use in sickle cell disease.