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. |
|
|
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). |
|
|
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). |
|
Recruiting Locations
Vanderbilt University Medical Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Vertex Pharmaceuticals Incorporated