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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05668741
• Other study ID #: VX21-522-001
• Secondary ID: 2022-000726-2520
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: February 27, 2023
• Est. completion date: March 2025

Study information

• Verified date: April 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: Medical Information
• Phone: 617-341-6777
• Email: medicalinfo[@]vrtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 36
• Est. completion date: March 2025
• Est. primary completion date: March 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VX-522 mRNA therapy
Oral inhalation using nebulizer.
• IVA
Tablet for oral administration.

Locations

Country	Name	City	State
Australia	Alfred Hospital	Melbourne
Canada	University of Calgary Medical Clinic of the Foothills Medical Centre	Calgary
Canada	Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval	Quebec
United Kingdom	Royal Papworth Hospital NHS Foundation Trust	Cambridge
United Kingdom	Clinical Research Facility, Queen Elizabeth University Hospital	Glasgow
United Kingdom	Royal Brompton Hospital	London
United Kingdom	Wythenshawe Hospital	Manchester
United Kingdom	All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough	Penarth
United Kingdom	Southampton General Hospital	Southampton
United States	Baltimore - Early Phase Clinical Unit	Baltimore	Maryland
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center	Boston	Massachusetts
United States	Medical University of South Carolina	Charleston	South Carolina
United States	UC Health Holmes	Cincinnati	Ohio
United States	Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	National Jewish Health	Denver	Colorado
United States	University of Florida, Shands Hospital	Gainesville	Florida
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Miller Children's Hospital / Long Beach Memorial	Long Beach	California
United States	University of Wisconsin Hospital and Clinics	Madison	Wisconsin
United States	University of Minnesota	Minneapolis	Minnesota
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Columbia University Medical Center	New York	New York
United States	Lenox Hill Hospital	New York	New York
United States	Stanford University	Palo Alto	California
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine / St. Louis Children's Hospital	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah

Sponsors (2)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated	Moderna, Inc

Countries where clinical trial is conducted

United States,  Australia,  Canada,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Day 1 Through Safety Follow-up Visit [up to Week 24 for SAD, and Week 28 for T1 and T2 (MAD)]
Secondary	T1 (MAD): Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)		From Baseline at Day 29
Secondary	T2 (MAD): Change From Pre-Run-in Baseline in ppFEV1		From Pre-Run Baseline at Day 29