A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2018-002496-18-PT
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-02-05
• Study Completion: 2019-12-10
• Last Update Posted: 5 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 87

Inclusion Criteria

- Subjects aged 18 years or older on the date of informed consent.
- Female subjects that have a negative pregnancy test at time of screening.
- Body weight =35 kg
- Parts 1 & 3: Heterozygous for F508del and an MF mutation (F/MF)
- Part 2: Homozygous for F508del
- FEV1 value =40% and =90% of predicted mean for age, sex, and height
Other protocol defined Inclusion criteria may apply

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 108
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- History of any comorbidity that might confound the results of the study or pose an additional risk in administering study drug to the subject.
- History of clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- An acute illness not related to CF within 14 days before the first dose of study drug.
- History of solid organ or hematological transplantation
Other protocol defined Exclusion criteria may apply

Study & Design

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