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FTX-6058 - Two Dosing Periods for Sickle Cell Disease

Phase 1

Waitlist Available

Research Sponsored by Fulcrum Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up study part e : days -1, 7,14 and and 7-10 days after last dose of study drug

Summary

This trial tests a new drug called FTX-6058 to see if it is safe and how it behaves in healthy adults and adults with sickle cell disease. The study aims to understand its safety, tolerability, and interactions in the body.

Eligible Conditions

Healthy Adult Subjects
Sickle Cell Disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ study part e : days -1, 7,14 and and 7-10 days after last dose of study drug

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~study part e : days -1, 7,14 and and 7-10 days after last dose of study drug

This trial's timeline: 3 weeks for screening, Varies for treatment, and study part e : days -1, 7,14 and and 7-10 days after last dose of study drug for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Treatment-Emergent Adverse Events

Secondary study objectives

Plasma Concentrations of FTX-6058

Plasma Concentrations of Midazolam

Plasma Concentrations of for 1-OH-Midazolam

Other study objectives

Predictive Model of the Relationship between FTX-6058 Concentration and QTc Intervals

Target Engagement of FTX-6058

Trial Design

5Treatment groups

Experimental Treatment

Group I: Single Ascending Dose (SAD) cohorts in Healthy Subjects (Part A)Experimental Treatment1 Intervention

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).

Group II: Potential for CYP3A Induction in Healthy Subjects (Part D)Experimental Treatment1 Intervention

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.

Group III: Pilot Food Effect Cohort in Healthy Subjects (Part C)Experimental Treatment1 Intervention

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.

Group IV: Multiple Dose Cohort in Sickle Cell Disease Subjects (Part E)Experimental Treatment1 Intervention

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.

Group V: Multiple Ascending Dose (MAD) cohorts in Healthy Subjects (Part B)Experimental Treatment1 Intervention

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).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

FTX-6058/placebo oral capsule(s)

2020

Completed Phase 1

~110

FTX-6058 - Two Dosing Periods

2020

Completed Phase 1

~110