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

NCT05668741

Active Not RecruitingPhase 1, Phase 2

Sponsor: Vertex Pharmaceuticals Incorporated

Conditions: Cystic Fibrosis

Interventions: VX-522 mRNA therapy, IVA

Countries: United States, Australia, Belgium, Canada, Germany, Sweden, United Kingdom

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.

Eligibility overview

Sex: ALL

Age: 18 Years to 65 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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: ≥ 50% 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.
Locations (26)
  • Birmingham, Alabama, United States
  • Long Beach, California, United States
  • Palo Alto, California, United States
  • Denver, Colorado, United States
  • Kansas City, Kansas, United States
  • Baltimore, Maryland, United States
  • Baltimore, Maryland, United States
  • Boston, Massachusetts, United States
  • Boston, Massachusetts, United States
  • Minneapolis, Minnesota, United States
  • St Louis, Missouri, United States
  • Cincinnati, Ohio, United States
  • Charleston, South Carolina, United States
  • Salt Lake City, Utah, United States
  • Melbourne, Australia
  • Ghent, Belgium
  • Calgary, Canada
  • Québec, Canada
  • Essen, Germany
  • Stockholm, Sweden
  • Cambridge, United Kingdom
  • Glasgow, United Kingdom
  • London, United Kingdom
  • Manchester, United Kingdom
  • Penarth, United Kingdom
  • Southampton, United Kingdom