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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...
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FTX-6058 - Two Dosing Periods is an investigational small molecule developed by Fulcrum Therapeutics, Inc. (ticker FULC) for sickle cell disease. It was studied in a Phase 1 clinical trial that evaluated safety, tolerability, and pharmacokinetics in healthy adult subjects and people with sickle cell disease. The program has received FDA Fast Track designation.
FTX-6058 - Two Dosing Periods is being developed for the treatment of sickle cell disease, a hematologic disorder. The Phase 1 study enrolled healthy adult subjects as well as individuals with sickle cell disease to assess the drug's safety, tolerability, and pharmacokinetic profile.
FTX-6058 - Two Dosing Periods is developed by Fulcrum Therapeutics, Inc., which trades under the ticker symbol FULC. Fulcrum is the sponsor of the clinical program evaluating this small molecule in sickle cell disease.
FTX-6058 - Two Dosing Periods is in Phase 1 development. The single Phase 1 trial, NCT04586985, has been completed. The drug is investigational and has not been approved by the FDA; it holds Fast Track designation for sickle cell disease.
FTX-6058 - Two Dosing Periods was evaluated in one Phase 1 trial, NCT04586985, titled "Safety, Tolerability and Pharmacokinetics of FTX-6058." The trial was completed, enrolled 109 participants, and was conducted in the United States. It was randomized, double-blind, and controlled, including healthy adult volunteers and subjects with sickle cell disease.
Yes, FTX-6058 - Two Dosing Periods is also known as FTX-6058 - Two Dosing Periods. The name refers to the same investigational small molecule developed by Fulcrum Therapeutics for sickle cell disease, studied in the completed Phase 1 trial NCT04586985.