Status:
RECRUITING
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborating Sponsors:
Moderna, Inc
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
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 conducta...
Eligibility Criteria
Key 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: ≥ 50% to less than or equal to (≤) 90%
Key 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.
Key Trial Info
Start Date :
February 27 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 12 2027
Estimated Enrollment :
39 Patients enrolled
Trial Details
Trial ID
NCT05668741
Start Date
February 27 2023
End Date
May 12 2027
Last Update
March 25 2026
Active Locations (43)
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1
University of Alabama at Birmingham - Child Health Research Unit
Birmingham, Alabama, United States, 35233
2
Memorial Health Services on behalf of Long Beach Memorial Medical Center d/b/a Miller Children's Hospital Long Beach
Long Beach, California, United States, 90806
3
Stanford University - Palo Alto - Pulmonology
Palo Alto, California, United States, 94304
4
National Jewish Health
Denver, Colorado, United States, 80206