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FTX-6058 for Sickle Cell Disease

Phase 1
Recruiting
Research Sponsored by Fulcrum Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant is 18 to 65 years of age at the time informed consent is obtained
≥4 episodes of SCD pain crisis over 12 months, or ≥2 over 6 months prior to screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline and at days 1, 14, 28, 42, 56, 70, 84, 88, 91, and 112
Awards & highlights

Study Summary

This trial is testing a new drug to see if it is safe and effective for people with sickle cell disease.

Who is the study for?
Adults aged 18-65 with sickle cell disease (SCD), specific genotypes, and low fetal hemoglobin can join. They must have had multiple SCD complications despite treatment with drugs like Hydroxyurea or be ineligible for chronic transfusions due to side effects.Check my eligibility
What is being tested?
The trial is testing FTX-6058 oral capsules to see if they're safe and how they affect the body in people with SCD. It looks at how the drug moves through and out of the body, as well as its impact on disease factors.See study design
What are the potential side effects?
Specific side effects are not listed but generally, participants will be monitored for any adverse reactions related to taking FTX-6058 capsules which could range from mild digestive issues to more serious blood-related problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 18 and 65 years old.
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I've had 4 or more pain crises in the last year, or 2 or more in the last 6 months.
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I've had 2 or more sickle cell pain crises and either chest pain, liver or spleen issues, or prolonged erections in the last year.
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I have used Hydroxyurea for 6 months without improvement or could not tolerate it.
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I have been diagnosed with Sickle Cell Disease (SCD) of specific genotypes.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline and at days 1, 14, 28, 42, 56, 70, 84, 88, 91, and 112
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline and at days 1, 14, 28, 42, 56, 70, 84, 88, 91, and 112 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Plasma Concentrations of FTX-6058
Treatment-Emergent Adverse Events
Secondary outcome measures
Change from Baseline in % Reticulocytes
Change from Baseline in Absolute Reticulocyte Count
Change from Baseline in Red cell distribution width
+2 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: FTX-6058 oral capsule(s) in Sickle Cell participantsExperimental Treatment1 Intervention
Cohort 1 will receive 6 mg of FTX-6058 by mouth once daily. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. A total of seven cohorts may be included. Following the first cohort, doses for all subsequent cohorts will be determined following DMC review of the safety and pharmacokinetic data observed in participants from the prior and ongoing cohorts. Alternate dosing schedules may be evaluated in some of the cohorts.

Find a Location

Who is running the clinical trial?

Fulcrum TherapeuticsLead Sponsor
7 Previous Clinical Trials
609 Total Patients Enrolled
John Ziegler, MD, FASAStudy DirectorFulcrum Therapeutics
2 Previous Clinical Trials
161 Total Patients Enrolled
William Engelman, MDStudy DirectorFulcrum Therapeutics

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many sites are currently utilized to conduct this experiment?

"Currently, 10 different clinical trial sites are enrolling patients; three of these being in Chapel Hill, Atlanta and Baltimore. To reduce the burden associated with travelling to participate, it is advised that you select a site closest to your residence."

Answered by AI

What is the total recruitment size of this clinical trial?

"This trial necessitates that 40 fitting applicants must be recruited. They can join from different testing facilities, such as the University of North carolina at Chapel Hill in Chapel Hill and Visionaries Research in Atlanta."

Answered by AI

Is this medical trial currently registering participants?

"The current status of the trial, as reported on clinicaltrials.gov, is that it is actively recruiting for participants. This medical experiment was initially posted back in December 2021 and has had its details revised most recently in October 2022."

Answered by AI

What safety parameters must patients meet to take FTX-6058 oral capsules?

"As this is a Phase 1 trial with limited data supporting safety and efficacy, our experts at Power assigned FTX-6058 capsules a score of 1 on their risk assessment scale."

Answered by AI

What are the primary aims of this experiment?

"This clinical trial, which has a maximal duration of 15 weeks, is aimed at assessing the concentration of FTX-6058 in plasma. Secondary objectives include tracking changes from baseline in fetal hemoglobin through HPLC, percentage reticulocytes with flow cytometry and absolute reticulocyte count via microscopy/cytometry."

Answered by AI

Am I eligible to be a subject in this experiment?

"This clinical trial requires 40 participants with anemia and sickle cell between the ages of 18 and 65 to meet specified criteria, including a documented HbF ≤ 20%, total hemoglobin ≥ 5.5 g/dL (males) or ≤ 10.6 g/dL (females), 0-6 Vaso-Occlusive Crisis episodes in 12 months prior to screening, BMI between 18 and 32 kg/m2, Signed informed consent form before any study procedures are performed, Highly effective methods of contraception for 90 days after last dose if female or male respectively, Documented SCD at time of screening confirmed through medical"

Answered by AI

Are persons above the age of 40 able to partake in this trial?

"The eligibility for this trial has a strict age restriction, with the minimum being 18 years old and the maximum 65."

Answered by AI
~22 spots leftby Apr 2025