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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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.