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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
Awards & highlights

Study Summary

This trial is testing a new drug to see if it is safe and works well in people with sickle cell disease.

Eligible Conditions
  • Healthy Adult Subjects
  • Sickle Cell Disease

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
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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Treatment-Emergent Adverse Events
Secondary outcome measures
Plasma Concentrations of FTX-6058
Plasma Concentrations of Midazolam
Plasma Concentrations of for 1-OH-Midazolam
Other outcome measures
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
FTX-6058 / Midazolam Syrup
2020
Completed Phase 1
~110

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Fulcrum TherapeuticsLead Sponsor
7 Previous Clinical Trials
570 Total Patients Enrolled
John Ziegler, MD, FASAStudy DirectorFulcrum Therapeutics
2 Previous Clinical Trials
122 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
New England Journal of MedicineJournal
Sickle Cell Disease
Piel, Frédéric B., Martin H. Steinberg, and David C. Rees. 2017. “Sickle Cell Disease”. Edited by Dan L. Longo. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmra1510865.
BloodJournal
Fetal Hemoglobin in Sickle Cell Anemia: A Glass Half Full?
Steinberg, Martin H., David H. K. Chui, George J. Dover, Paola Sebastiani, and Abdulrahman Alsultan. 2014. “Fetal Hemoglobin in Sickle Cell Anemia: A Glass Half Full?”. Blood. American Society of Hematology. doi:10.1182/blood-2013-09-528067.
Paediatric Respiratory ReviewsJournal
Differences in the Clinical and Genotypic Presentation of Sickle Cell Disease Around the World
Saraf, Santosh L., Robert E. Molokie, Mehdi Nouraie, Craig A. Sable, Lori Luchtman-Jones, Gregory J. Ensing, Andrew D. Campbell, et al.. 2014. “Differences in the Clinical and Genotypic Presentation of Sickle Cell Disease Around the World”. Paediatric Respiratory Reviews. Elsevier BV. doi:10.1016/j.prrv.2013.11.003.
British Journal of HaematologyJournal
Fetal Haemoglobin Levels and Haematological Characteristics of Compound Heterozygotes for Haemoglobin S and Deletional Hereditary Persistence of Fetal Haemoglobin
Ngo, Duyen A., Banu Aygun, Idowu Akinsheye, Jane S. Hankins, Ishir Bhan, Hong Y. Luo, Martin H. Steinberg, and David H. K. Chui. 2011. “Fetal Haemoglobin Levels and Haematological Characteristics of Compound Heterozygotes for Haemoglobin S and Deletional Hereditary Persistence of Fetal Haemoglobin”. British Journal of Haematology. Wiley. doi:10.1111/j.1365-2141.2011.08916.x.
~25 spots leftby Apr 2025
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