A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
- Conditions
- Cystic Fibrosis
- Interventions
- Drug: VX-522 mRNA therapyDrug: IVA
- Registration Number
- NCT05668741
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- SUSPENDED
- Sex
- All
- Target Recruitment
- 36
-
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%
Key
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Single Ascending Dose (SAD) VX-522 mRNA therapy Participants grouped into different cohorts will receive a single ascending dose of VX-522. Multiple Ascending Dose (MAD) Arm 1: VX-522 VX-522 mRNA therapy Participants grouped into different cohorts will receive multiple ascending doses of VX-522 in treatment arm 1 (T1). MAD Arm 2: VX522+ IVA VX-522 mRNA therapy 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). MAD Arm 2: VX522+ IVA 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).
- Primary Outcome Measures
Name Time Method Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Day 1 Through Safety Follow-up Visit [up to Week 24 for SAD, and Week 28 for T1 and T2 (MAD)]
- Secondary Outcome Measures
Name Time Method MAD: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline at Day 29
Trial Locations
- Locations (41)
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Miller Children's Hospital / Long Beach Memorial
🇺🇸Long Beach, California, United States
Stanford University Clinical and Translational Research Unit
🇺🇸Palo Alto, California, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
University of Florida, Shands Hospital
🇺🇸Gainesville, Florida, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
Baltimore - Early Phase Clinical Unit
🇺🇸Baltimore, Maryland, United States
Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
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