Evaluating VX-828 in healthy individuals and those with cystic fibrosis
A Phase 1, Study of VX-828 in Healthy Subjects and in Subjects With Cystic Fibrosis
This study is testing a new drug called VX-828 to see if it is safe and effective for healthy people and those with cystic fibrosis, while also looking at how it affects lung function and overall health.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 255 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Vertex Pharmaceuticals Incorporated Industry-sponsored |
| Locations | 12 sites (Hollywood, Florida and 11 other locations) |
| Trial ID | NCT06154447 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to assess the safety, tolerability, and pharmacokinetics of VX-828, both alone and in combination with other drugs, in healthy participants and individuals diagnosed with cystic fibrosis. The study will involve multiple cohorts, including healthy adults aged 18 to 55 and cystic fibrosis patients who meet specific genetic criteria. Participants will receive either VX-828, a placebo, or other medications to determine the best therapeutic approach. The trial will also evaluate the drug's effects on lung function and overall health in cystic fibrosis patients.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of cystic fibrosis who meet specific genetic criteria.
Not a fit: Patients who are not heterozygous for the F508del mutation or those with a CFTR allele responsive to existing therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved treatment options for patients with cystic fibrosis.
How similar studies have performed: Other studies have shown promise in evaluating similar drug combinations for cystic fibrosis, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: Parts A-D: * Participants between the ages of 18 and 55 years * Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m\^2) * A total body weight of more than (\>) 50 kg * Nonsmoker or ex-smoker for at least 3 months before screening with current nonsmoking status confirmed by urine or blood cotinine at screening * Cohort C2 only: Willing to provide a single DNA sample Part E: * Participants 18 years or older * Confirmed diagnosis of CF as determined by the investigator * A total body weight of more than or equal to (\>=) 35 kg * Participants must be heterozygous for F508del with a second CFTR allele carrying a minimal function mutation that is not responsive to ELX/TEZ/IVA therapy * Participants must have a forced expiratory volume in 1 second (FEV1) of greater than or equal to (≥) 40% of predicted normal for age, sex, and height Key Exclusion Criteria: Parts A-D: * History of febrile illness or other acute illness within 14 days before the first dose of study drug * Any condition possibly affecting drug absorption Part E: * An acute illness not related to CF (e.g., gastroenteritis) within 14 days before the first dose of study drug * History of solid organ or hematological transplantation * History of clinically significant cirrhosis with or without portal hypertension * Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria will apply.
Where this trial is running
Hollywood, Florida and 11 other locations
- Joe DiMaggio Cycstic Fibrosis & Pulmonary Center — Hollywood, Florida, United States (Recruiting)
- AdventHealth Medical Group Pulmonology at Orlando Ridgewood — Orlando, Florida, United States (Recruiting)
- Altasciences Clinical Kansas — Overland Park, Kansas, United States (Completed)
- Kentucky Children's Hospital — Lexington, Kentucky, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- University of Minnesota -Pulmonology — Minneapolis, Minnesota, United States (Recruiting)
- Billings Clinic, Pediatric Pulmonary Dept. — Billings, Montana, United States (Recruiting)
- New York Medical College — Hawthorne, New York, United States (Recruiting)
- ProMedica Toledo Children's Hospital & ProMedica Central Physicians, LLC — Toledo, Ohio, United States (Recruiting)
- Cook Children's Pulmonology — Fort Worth, Texas, United States (Recruiting)
- University of Utah Hospital - Pulmonology — Salt Lake City, Utah, United States (Recruiting)
- Vermont Lung Center — Colchester, Vermont, United States (Recruiting)
Study contacts
- Study coordinator: Medical Information
- Email: medicalinfo@vrtx.com
- Phone: 617-341-6777
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.