VX-828 for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment, VX-828, for people with cystic fibrosis to evaluate its absorption and safety. Participants will receive a single dose of VX-828, either as a liquid or a tablet, and researchers will compare the two forms. As a Phase 1 trial, it represents an early stage of testing to gather data on safety and absorption. Individuals with cystic fibrosis who are nonsmokers or have quit smoking for at least three months and weigh more than 50 kg (about 110 pounds) might be suitable candidates. Participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.
Will I have to stop taking my current medications?
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 VX-828 is likely to be safe for humans?
Research has shown that VX-828 underwent safety testing in earlier studies. It was generally well-tolerated by both healthy individuals and those with cystic fibrosis, with no major safety issues reported. Now in early testing stages, researchers closely monitor its effects to ensure human safety. This stage typically involves small groups to assess safety, and so far, the treatment appears promising in terms of tolerance.12345
Why do researchers think this study treatment might be promising?
VX-828 is unique because it offers a new form of delivery for cystic fibrosis treatment. While most current treatments involve inhaled medications or complex drug combinations targeting faulty proteins, VX-828 is being tested in two forms: a suspension and a tablet. This provides more flexible dosing options and potentially improves ease of use for patients. Researchers are excited because if VX-828 proves effective, it could simplify treatment regimens and enhance patient compliance, making life a bit easier for those dealing with this challenging condition.
What evidence suggests that VX-828 might be an effective treatment for cystic fibrosis?
Research has shown that VX-828 is under study as a potential treatment for cystic fibrosis. It targets the faulty protein causing the disease, which might improve lung function. In this trial, participants will receive VX-828 either as a suspension or in tablet form. Early results suggest that VX-828, particularly when combined with other treatments, could enhance the overall health of individuals with cystic fibrosis. Although more data is needed to confirm its efficacy, addressing the root cause of cystic fibrosis remains promising. Initial studies have demonstrated that VX-828 is generally well-tolerated, indicating a positive outlook for its future use.12678
Are You a Good Fit for This Trial?
This trial is for individuals with a BMI of 18.0 to 32.0 kg/m^2, weighing over 50 Kg, who are nonsmokers or have quit smoking for at least 3 months. It's aimed at those not capable of childbearing and suffering from Cystic Fibrosis or related liver disease.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single dose of VX-828 in either tablet or suspension form to evaluate relative bioavailability
Follow-up
Participants are monitored for safety and tolerability after receiving the study drug
What Are the Treatments Tested in This Trial?
Interventions
- VX-828
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vertex Pharmaceuticals Incorporated
Lead Sponsor
Dr. David Altshuler
Vertex Pharmaceuticals Incorporated
Chief Medical Officer since 2020
MD, PhD
Dr. Reshma Kewalramani
Vertex Pharmaceuticals Incorporated
Chief Executive Officer since 2020
MD, trained in internal medicine and nephrology