SUSPENDED

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

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Conditions

Cystic Fibrosis

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2023-02-27
Study Completion:2027-05-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:SUSPENDED

Study ID

NCT05668741

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.

Ages Eligible for Study:18 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Body mass index is less than (\<) 30.0 kilograms per meter square (kg/m\^2)
  2. * A total body weight greater than (\>) 50 kg
  3. * Stable CF disease
  4. * CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy
  5. * 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%
  1. * History of uncontrolled asthma within a year prior to screening
  2. * History of solid organ or hematological transplantation
  3. * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  4. * Arterial oxygen saturation on room air less than (\<) 94% at screening

Contacts and Locations

Study Locations (Sites)

University of Alabama at Birmingham
Birmingham, Alabama, 35233
United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, 90806
United States
Stanford University Clinical and Translational Research Unit
Palo Alto, California, 94304
United States
National Jewish Health
Denver, Colorado, 80206
United States
University of Florida, Shands Hospital
Gainesville, Florida, 32610
United States
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
Baltimore - Early Phase Clinical Unit
Baltimore, Maryland, 21225
United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287
United States
Massachusetts General Hospital
Boston, Massachusetts, 02114
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
University of Minnesota
Minneapolis, Minnesota, 55455
United States
Washington University School of Medicine / St. Louis Children's Hospital
Saint Louis, Missouri, 63110
United States
Lenox Hill Hospital
New York, New York, 10028
United States
Columbia University Medical Center
New York, New York, 10032
United States
UC Health Holmes
Cincinnati, Ohio, 45220
United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
United States
Medical University of South Carolina - Nexus Research Clinic
Charleston, South Carolina, 29425
United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
United States
Baylor College of Medicine
Houston, Texas, 77030
United States
University of Utah
Salt Lake City, Utah, 84132
United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792
United States

Collaborators and Investigators

Sponsor: Vertex Pharmaceuticals Incorporated

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-02-27
Study Completion Date2027-05-12

Study Record Updates

Study Start Date2023-02-27
Study Completion Date2027-05-12

Terms related to this study

Additional Relevant MeSH Terms

  • Cystic Fibrosis