Vanderbilt Clinical Trials

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

Arm	Description	Assigned 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

Vanderbilt University Medical Center
Nashville, Tennessee 37232

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.