VX-581 for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called VX-581 for people with cystic fibrosis, a genetic condition affecting the lungs and digestive system. The study aims to determine if VX-581 is safe and how the body processes the drug at different doses. Some participants will receive the actual treatment, while others will receive a placebo, which resembles the treatment but contains no active drug. This trial suits those with cystic fibrosis who weigh more than 50 kg (about 110 pounds) and have no recent illnesses. 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 have to stop taking my current medications for the 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 for more details.
Is there any evidence suggesting that VX-581 is likely to be safe for humans?
Research shows that VX-581 is under study to determine its safety and tolerability. The studies test reactions to various doses of VX-581, including both single and multiple doses.
Currently, limited information exists on the safety of VX-581 for humans. As the study is in its early stages, it primarily seeks to identify any side effects. At this point, the treatment is not approved for any condition. Early trials like this one offer initial insights into safety but have not been widely tested.
For those considering participation, it's important to know that the main goal is to understand how the body processes VX-581 and to identify any potential issues. For further questions, consulting the study team can provide clarity on what to expect in the trial.12345Why do researchers think this study treatment might be promising for cystic fibrosis?
Most treatments for cystic fibrosis, like CFTR modulators, work by improving the function of the faulty protein caused by the CFTR gene mutation. But VX-581 works differently, targeting specific pathways to enhance chloride ion transport across cell membranes. Researchers are excited about VX-581 because it could offer a new approach by possibly treating more underlying aspects of cystic fibrosis, potentially improving lung function and slowing disease progression. Unlike some existing options that primarily focus on symptom management, VX-581's unique mechanism might provide more comprehensive benefits for patients with cystic fibrosis.
What evidence suggests that VX-581 might be an effective treatment for cystic fibrosis?
Research shows that VX-581 is a new treatment for cystic fibrosis. Although limited data exists on VX-581's effectiveness, it belongs to a group of treatments called CFTR modulators. These treatments help correct the protein problem in people with cystic fibrosis. Other treatments in this group have led to significant improvements, such as better lung function and a higher quality of life for patients. These early results are encouraging and suggest that VX-581 might offer similar benefits. However, further research is needed to confirm VX-581's effectiveness in treating cystic fibrosis. Participants in this trial will receive either VX-581 or a placebo to evaluate its effectiveness.16789
Are You a Good Fit for This Trial?
This trial is for healthy individuals over 50 kg in weight, with a BMI of 18.0 to 32.0 kg/m^2. Participants must be of non-childbearing potential, which includes women who are postmenopausal or have had surgery to prevent pregnancy and men who are surgically sterile.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive single and multiple ascending doses of VX-581 or placebo
Safety Follow-up
Participants are monitored for safety and pharmacokinetics after treatment
What Are the Treatments Tested in This Trial?
Interventions
- VX-581
Trial Overview
The study is testing VX-581, a new drug for cystic fibrosis. It involves giving participants either the actual drug or a placebo without any active ingredients to compare effects on safety and how the body processes the drug at different doses.
How Is the Trial Designed?
4
Treatment groups
Experimental Treatment
Placebo Group
Participants will be randomized to receive multiple doses of VX-581.
Participants will be randomized to receive a single dose of VX-581.
Participants will be randomized to receive multiple doses of placebo matched to VX-581.
Participants will be randomized to receive a single dose of placebo matched to VX-581.
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
Citations
Dose Escalation Study Evaluating the Safety and ...
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of VX-581. Detailed Description.
Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in ...
At 6 months into ETI therapy, ppFEV1 improved 9.76 percentage points (95% confidence interval [CI], 8.76 to 10.76) from baseline, cystic fibrosis questionnaire– ...
3.
news.vrtx.com
news.vrtx.com/news-releases/news-release-details/vertex-presents-new-data-across-portfolio-cystic-fibrosisVertex Presents New Data Across Portfolio of Cystic Fibrosis ...
Data presented on outcomes following treatment with CFTR modulators add to growing body of evidence that reduced level of sweat chloride is ...
Improvements in health-related quality of life in people with ...
Vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) was shown to provide greater restoration of cystic fibrosis transmembrane conductance ...
Elexacaftor-tezacaftor-ivacaftor in people with cystic ...
At the end of this initial treatment period, effectiveness was judged by a centralized adjudication committee, which analyzed all available data ...
6.
htsyndication.com
htsyndication.com/us-fed-news/article/clinical-trial%3A-dose-escalation-study-evaluating-the-safety-and-pharmacokinetics-of-vx-581-in-healthy-participants/555782462Clinical Trial: Dose Escalation Study Evaluating the Safety ...
Clinical Trial: Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants. Posted On: 2025-12-17.
VX-581 in Cystic Fibrosis - Clinical Trials Registry - ICH GCP
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of VX-581.
Cystic fibrosis - PMC - PubMed Central - NIH
Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. ... Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study.
Clinical Study Protocol
Data from all safety and efficacy endpoints will be analyzed using descriptive statistics. For efficacy endpoints, separate tables will be presented for ...
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