Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Nov 23, 2023

Keywords

ClinConnect Summary

This clinical trial is testing a new drug called VX-828 to see how safe it is and how the body handles it in healthy people. The goal is to gather important information that can help in the future treatment of cystic fibrosis, a condition that affects the lungs and digestive system. The trial is currently looking for participants aged between 18 and 65 who have a body weight over 50 kg and a body mass index (BMI) between 18.0 and 32.0. Additionally, participants should not be smokers or should have quit smoking at least three months before joining the study.

If you or someone you know meets these criteria and decides to participate, you can expect to undergo some health screenings to ensure you’re a good fit for the study. Participants will be closely monitored throughout the trial to check for any side effects and to see how the medication works in the body. It's important to note that people who have had recent illnesses or conditions that might affect how the drug is absorbed will not be eligible to participate. This study is a crucial step in understanding how VX-828 could potentially help those with cystic fibrosis in the future.

Gender

ALL

Eligibility 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.