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

Purpose

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.

Condition

  • Cystic Fibrosis

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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%

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.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Single Ascending Dose (SAD) 		Participants grouped into different cohorts will receive a single ascending dose of VX-522.
  • Drug: VX-522 mRNA therapy
    Oral inhalation using nebulizer.
Experimental
Multiple Ascending Dose (MAD) Arm 1: VX-522 		Participants grouped into different cohorts will receive multiple ascending doses of VX-522 in treatment arm 1 (T1).
  • Drug: VX-522 mRNA therapy
    Oral inhalation using nebulizer.
Experimental
MAD Arm 2: VX522+ IVA 		Following run-in period with ivacaftor (IVA), participants grouped into different cohorts will receive multiple ascending doses of VX-522 with IVA in treatment arm (T2).
  • Drug: VX-522 mRNA therapy
    Oral inhalation using nebulizer.
  • Drug: IVA
    Tablet for oral administration.
    Other names:
    • ivacaftor
    • VX-770

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66160

More Details

Status
Recruiting
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

Medical Information
617-341-6777
medicalinfo@vrtx.com