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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

Must not have

History of bone marrow transplant or human stem cell transplant or gene therapies

Participants with a history of severe renal disease defined as estimated glomerular filtration rate < 30 mL/min/1.73m^2. Participants on dialysis of any kind are excluded

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

No Placebo-Only Group

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had a bone marrow, stem cell, or gene therapy.

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I do not have severe kidney disease or require dialysis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline and at days 1, 14, 28, 42, 56, 70, 84, 88, 91, and 112

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

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

Primary study objectives

Plasma Concentrations of FTX-6058

Treatment-Emergent Adverse Events

Secondary study objectives

Change from Baseline in % Reticulocytes

Change from Baseline in Absolute Reticulocyte Count

Change from Baseline in Red cell distribution width

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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.

0 / 7Eligibility criteria met