Summary
The purpose of this study is to evaluate the safety and tolerability 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
Detailed Description
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4) (A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
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, percent of predicted mean for age, sex, and height (equations of the Global Lung Function Initiative [GLI])18 ≥40%
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
Phase
Phase 1
Gender
All
Min Age
18 Years
Max Age
65 Years
Download Date
November 1, 2023
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: