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.
Conditions
Cystic Fibrosis
Recruitment Status
RECRUITING
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: ≥ 40% 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.
Intervention
Intervention Type
Intervention Name
DRUG
VX-522 mRNA therapy
DRUG
IVA
Phase
PHASE1, PHASE2
Gender
ALL
Min Age
18 Years
Max Age
65 Years
Download Date
2024-10-15
Principal Investigator
This field has been modified from ClinicalTrials.gov to show a contact specific to Boston Children's.
Primary Contact Information
robert.fowler@childrens.harvard.edu
This field has been modified from ClinicalTrials.gov to show a contact specific to Boston Children's.
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: