NCT04586985

Safety, Tolerability and Pharmacokinetics of FTX-6058

A Phase 1 Randomized, Double-Blind, Placebo-Controlled, Single-and Multiple-Dose Study Evaluating Safety, Tolerability, and Pharmacokinetics, With an Open-Label Initial Food Effect and CYP3A Drug-Drug Interaction Study, of FTX-6058 in Healthy Adult Subjects, and a Randomized, Double-Blind, Placebo-Controlled Multiple-Dose Study Evaluating, Safety, Tolerability and Pharmacokinetics in Subjects With Sickle Cell Disease (SCD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 109 (actual)
• Sponsor: Fulcrum Therapeutics · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

This is a study to evaluate the safety, tolerability and pharmacokinetics of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease (SCD).

Detailed description

This study is a Phase 1 randomized, double-blind, placebo-controlled, single- and multiple-dose study evaluating safety, tolerability, and pharmacokinetics, with an open-label initial food effect and CYP3A drug-drug interaction study, of FTX-6058 in healthy adult subjects and a randomized, double-blind, placebo-controlled, multiple-dose study in adult subjects with sickle cell disease (SCD). This study will comprise 5 parts and will be conducted in healthy adult subjects and adult subjects with sickle cell disease (SCD) (Part E only). Part A will be a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study in up to 8 cohorts of healthy adult subjects. Part B will be a randomized, double-blind, placebo-controlled, multiple ascending dose (MAD) study in up to 6 cohorts of healthy adult subjects dosed once daily for 14 days. Part C will be an open-label pilot food effect study in healthy adult subjects randomized to take FTX-6058 with and without a high-fat meal, and Part D will be an open-label study to evaluate the potential of FTX-6058 to induce CYP3A (using midazolam) in healthy adult subjects. Part E will be a randomized, double-blind, placebo-controlled, multiple dose study in adult subjects with sickle cell disease (SCD). Subjects in Part E may enroll into Study 6058-SCD-101 (NCT 05169580) Part A (6 mg cohort) at the Day 15 visit; subjects that enroll into 6058-SCD-101 will not need to undergo the Safety Follow-up visit. The primary endpoint of the study is to evaluate the safety and tolerability of FTX-6058 in healthy adult subjects and adult subjects with sickle cell disease as measured by the frequency of adverse events. Secondary endpoints include evaluation of the pharmacokinetics of single dose and multiple dose FTX-6058 in healthy adult subjects, pharmacokinetics of multiple dose FTX-6058 in adult subjects with sickle cell disease, evaluation of the potential effect of food on FTX-6058 and evaluation of the potential for CYP3A induction by FTX-6058 in healthy adult subjects.

Conditions

• Healthy Adult Subjects
• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	FTX-6058/placebo oral capsule(s)	Subjects will receive FTX-6058 or matching placebo.
DRUG	FTX-6058/placebo oral capsule(s)	Subjects will receive FTX-6058 or matching placebo.
DRUG	FTX-6058 - Two Dosing Periods	Subjects will receive FTX-6058
DRUG	FTX-6058 / Midazolam Syrup	Subjects will receive FTX-6058 and midazolam syrup
DRUG	FTX-6058/placebo oral capsule(s)	Subjects will receive FTX-6058 or matching placebo.

Timeline

• Start date: 2020-10-26
• Primary completion: 2022-11-15
• Completion: 2022-11-15
• First posted: 2020-10-14
• Last updated: 2022-12-12

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study