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Recruiting
Phase 1, Phase 2
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.

ClinicalTrials.gov Identifier
NCT05668741
Eligibility criteria
Sex
Age
18 Years - 65 Years
Phase
1, 2
Key inclusion criteria
  • Body mass index is less than (<) 30.0 kilograms per meter square (kg/m^2)
  • A total body weight greater than (>) 50 kg
  • Stable CF disease
  • CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy
  • o Example mutations include but are not limited to, mutations that do not produce CFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice mutations (e.g., 621+1G->T)
  • Forced expiratory volume in 1 second (FEV1) value for SAD: greater than or equal to (≥)40 percent (%), MAD: ≥ 40% to less than or equal to (≤) 90%
Key exclusion criteria
  • History of uncontrolled asthma within a year prior to screening
  • History of solid organ or hematological transplantation
  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • Arterial oxygen saturation on room air less than (<) 94% at screening
Other protocol defined Inclusion/Exclusion criteria may apply.
Trial details
Condition
Cystic Fibrosis
Study duration
February 2023 - March 2025
Study type
Interventional
Intervention/Treatment
Drug : VX-522 mRNA therapy, Drug : IVA
Estimated enrollment
36

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