FLT201 for Gaucher Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial monitors the long-term effects of FLT201, a treatment for Gaucher disease type 1, a genetic disorder that impairs the body's ability to break down a specific type of fat. The researchers aim to track participants over five years to assess the treatment's effectiveness and safety over time. It targets individuals who have already received FLT201 in a previous study and can continue participating while following the study guidelines. As a Phase 1 and Phase 2 trial, this research seeks to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.
Do I need to stop my current medications for the FLT201 trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that FLT201 is likely to be safe for humans?
Research has shown that FLT201, a gene therapy for Gaucher disease type 1, has promising safety results from earlier studies. Of the five patients who received FLT201, four stopped their previous standard treatments, indicating good tolerance of the therapy. Additionally, FLT201 appears to reduce symptoms of Gaucher disease by lowering harmful substances in the body. These findings suggest that FLT201 could be a safe option for those exploring new treatments for Gaucher disease. However, as this is an early-phase study, more research is needed to fully understand its long-term safety.12345
Why do researchers think this study treatment might be promising?
Unlike the standard enzyme replacement therapies for Gaucher Disease, which require regular infusions, FLT201 offers a novel approach by utilizing gene therapy to potentially provide a long-term solution. This treatment is designed to deliver a single dose that could permanently correct the underlying genetic defect, reducing or even eliminating the need for ongoing treatments. Researchers are excited because this approach targets the root cause of the disease, offering the possibility of a one-time, lasting remedy rather than temporary symptom management.
What evidence suggests that FLT201 might be an effective treatment for Gaucher disease?
Studies have shown that FLT201, a gene therapy under investigation in this trial, has promising results for people with Gaucher disease type 1. Research suggests it could offer a long-lasting treatment by enabling the liver to produce the necessary enzyme. Early findings indicate that patients who stopped their usual treatment after receiving FLT201 continued to benefit for up to two years. Specifically, there have been lasting reductions in lyso-Gb1 levels, a marker of disease activity. This suggests that FLT201 might improve symptoms that current treatments don't fully address. Overall, early evidence suggests FLT201 could significantly advance the treatment of Gaucher disease type 1.23678
Are You a Good Fit for This Trial?
This trial is for individuals with Type-1 Gaucher Disease who have previously received FLT201 treatment in an earlier study. They will be monitored over a period of 5 years to understand the long-term effects of the treatment.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants received FLT201 treatment in a preceding clinical trial
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- FLT201
Find a Clinic Near You
Who Is Running the Clinical Trial?
Freeline Therapeutics
Lead Sponsor
Spur Therapeutics
Lead Sponsor