A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

Description

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

Talk to us

Study Overview

On this page

Study Details

Study overview

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.

A Phase 1/2 Dose Escalation Study Evaluating the Safety, and Tolerability and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

Condition
Cystic Fibrosis
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama at Birmingham, Birmingham, Alabama, United States, 35233

Long Beach

Miller Children's Hospital / Long Beach Memorial, Long Beach, California, United States, 90806

Palo Alto

Stanford University, Palo Alto, California, United States, 94304

Denver

National Jewish Health, Denver, Colorado, United States, 80206

Gainesville

University of Florida, Shands Hospital, Gainesville, Florida, United States, 32610

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Baltimore

Baltimore - Early Phase Clinical Unit, Baltimore, Maryland, United States, 21225

Baltimore

Johns Hopkins Hospital, Baltimore, Maryland, United States, 21287

Boston

Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center, Boston, Massachusetts, United States, 02114

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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%
  • * 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

Ages Eligible for Study

18 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Vertex Pharmaceuticals Incorporated,

Study Record Dates

2025-03