Safety, Tolerability and Pharmacokinetics of FTX-6058
Condition: Healthy Adult Subjects; Sickle Cell Disease
MeSH terms: Anemia, Sickle Cell
Study type: Interventional
Phase: Phase 1

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.
    

Intervention name(s): FTX-6058/placebo oral capsule(s); FTX-6058/placebo oral capsule(s); FTX-6058 - Two Dosing Periods; FTX-6058 / Midazolam Syrup; FTX-6058/placebo oral capsule(s)
Intervention type(s): Drug; Drug; Drug; Drug; Drug
Intervention description(s): Subjects will receive FTX-6058 or matching placebo.; Subjects will receive FTX-6058 or matching placebo.; Subjects will receive FTX-6058; Subjects will receive FTX-6058 and midazolam syrup; Subjects will receive FTX-6058 or matching placebo.
Intervention MeSH terms: Midazolam
Allocation: Randomized
Intervention model: Sequential Assignment
Intervention model description: Single ascending dose escalation and multiple ascending dose escalation study followed by an evaluation of food effects on absorption and CYP3A induction
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Healthy Adult Subjects; Sickle Cell Disease; Sickle Cell Anemia; Sickle Cell Disorder