FTX-6058 for Sickle Cell Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called FTX-6058 for individuals with sickle cell disease. The study aims to assess the treatment's safety, tolerability, and how it moves through and works in the body. Participants will take varying doses to determine the optimal and safest amount. This trial may suit those with sickle cell disease who frequently experience pain crises or complications like acute chest syndrome and have not found success with other treatments. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.
Do I need to stop my current medications to join the trial?
Yes, you will need to stop taking certain medications before joining the trial. Specifically, you must stop taking voxelotor and crizanlizumab at least 60 days before starting the study drug, and L-glutamine at least 24 hours before.
Is there any evidence suggesting that FTX-6058 is likely to be safe for humans?
Research shows that FTX-6058, the treatment under study, is generally safe. In earlier studies, participants tolerated this drug well, with no serious safety concerns reported.
The treatment, also known as Pociredir, is administered in varying doses to determine the optimal and safest amount for patients. So far, patients have responded well, indicating positive signs for its safety.
These results come from early-stage trials, which are specifically designed to assess the safety of a new treatment before wider use.12345Why do researchers think this study treatment might be promising?
Researchers are excited about FTX-6058 for sickle cell disease because it represents a novel approach by potentially increasing fetal hemoglobin levels in patients, which can help alleviate the symptoms of the disease. Unlike traditional treatments like hydroxyurea, which primarily works by reducing the frequency of painful crises, FTX-6058 targets the underlying genetic issues causing sickle cell disease. This new mechanism of action could offer more comprehensive management of the condition and improve quality of life for patients by addressing the root cause rather than just the symptoms.
What evidence suggests that FTX-6058 might be an effective treatment for sickle cell disease?
Research has shown that FTX-6058, which participants in this trial may receive, may help treat sickle cell disease. In studies, patients experienced significant increases in fetal hemoglobin (HbF), which can reduce pain and improve blood health. For example, HbF levels increased from 7.6% to 16.2% over 12 weeks. Early research indicated that FTX-6058 could raise HbF levels to as much as 40% of total hemoglobin. Higher HbF levels are associated with fewer sickle cell complications, suggesting this treatment could be beneficial.12356
Who Is on the Research Team?
Adeyemi Adenola, MD
Principal Investigator
Fulcrum Therapeutics
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Pociredir for 12 weeks with varying doses across cohorts
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- FTX-6058
Find a Clinic Near You
Who Is Running the Clinical Trial?
Fulcrum Therapeutics
Lead Sponsor