Current Environment:

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

2024-11-22

Principal Investigator

Ahmet Uluer

This field has been modified from ClinicalTrials.gov to show a contact specific to Boston Children's.

Primary Contact Information

Robert Fowler
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:

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) NCT05668741 Robert Fowler robert.fowler@childrens.harvard.edu