A Phase 1/2 Study of VX-522 in Subjects with Cystic Fibrosis

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 20.0Level: PTClassification code: 10011762Term: Cystic fibrosis Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2023-504786-23-00
• Lead Sponsor: Vertex Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-05-16
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

Subject will sign and date an informed consent form., Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures., Subjects (male and female) between the ages of 18 and 65 years, inclusive., Body mass index (BMI) of <30.0 kg/m2 and a total body weight >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., CFTR mutations on both alleles that are not responsive to CFTR modulator therapy., Stable CF disease, as judged by the investigator, and FEV1 value, percent of predicted mean for age, sex, and height =40%, Willing to remain on a stable CF treatment regimen through completion of study participation.

Exclusion Criteria

History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drugs to the subject., Lung infection with organisms associated with a more rapid decline in pulmonary status., Alcohol or drug abuse in the past year, including, but not limited to, abuse of cannabis, cocaine, and opiates., Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject., Arterial oxygen saturation on room air <94% at screening or use of supplemental oxygen within 28 days before the first dose of study drug., Any of the following abnormal laboratory values at screening: Hemoglobin <10 g/dL, Total bilirubin =2 × ULN, AST, ALT, GGT, or ALP =3 × ULN, Abnormal renal function, defined as glomerular filtration rate =50 mL/min/1.73 m2, For female subjects: Pregnant or breast-feeding. Females of childbearing potential must have a negative pregnancy test at the Screening Visit and before the first dose of study drug. For male subjects: Male subjects with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 180 days after the last dose of study drug., Standard 12 lead ECG median of triplicate demonstrating QTcF >450 msec at screening., An acute upper or lower respiratory tract infection, PEx, or changes in therapy for pulmonary disease within 28 days before the first dose of study drug.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified