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

Primary Purpose

Cystic Fibrosis

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
VX-522 mRNA therapy
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Alabama at BirminghamRecruiting
  • Miller Children's Hospital / Long Beach MemorialRecruiting
  • Stanford UniversityRecruiting
  • National Jewish HealthRecruiting
  • University of Florida, Shands HospitalRecruiting
  • University of Kansas Medical CenterRecruiting
  • Baltimore - Early Phase Clinical UnitRecruiting
  • Johns Hopkins HospitalRecruiting
  • Massachusetts General Hospital Cystic Fibrosis Center Clinical Research CenterRecruiting
  • Boston Children's HospitalRecruiting
  • University of MinnesotaRecruiting
  • Washington University School of Medicine / St. Louis Children's HospitalRecruiting
  • Columbia University Medical CenterRecruiting
  • University of CincinnatiRecruiting
  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical CenterRecruiting
  • Nationwide Children's HospitalRecruiting
  • UPMC Children's Hospital of PittsburghRecruiting
  • Medical University of South CarolinaRecruiting
  • Vanderbilt University Medical CenterRecruiting
  • University of UtahRecruiting
  • University of Wisconsin Hospital and ClinicsRecruiting
  • Royal Papworth Hospital NHS Foundation TrustRecruiting
  • Clinical Research Facility, Queen Elizabeth University HospitalRecruiting
  • Royal Brompton HospitalRecruiting
  • Southampton General HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-522

Arm Description

Participants will receive a single dose of different dose levels of VX-522.

Outcomes

Primary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Full Information

First Posted
December 19, 2022
Last Updated
October 20, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05668741
Brief Title
A Phase 1 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Official Title
A Phase 1 Single Dose Escalation Study Evaluating the Safety and Tolerability of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 27, 2023 (Actual)
Primary Completion Date
January 2024 (Anticipated)
Study Completion Date
January 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No

5. Study Description

Brief 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.
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.).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
VX-522
Arm Type
Experimental
Arm Description
Participants will receive a single dose of different dose levels of VX-522.
Intervention Type
Drug
Intervention Name(s)
VX-522 mRNA therapy
Intervention Description
Oral inhalation using nebulizer.
Primary Outcome Measure Information:
Title
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
From Day 1 Through Safety Follow-up Visit (Week 24)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information
Phone
617-341-6777
Email
medicalinfo@vrtx.com
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Individual Site Status
Recruiting
Facility Name
Miller Children's Hospital / Long Beach Memorial
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Individual Site Status
Recruiting
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Individual Site Status
Recruiting
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Florida, Shands Hospital
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Name
Baltimore - Early Phase Clinical Unit
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21225
Country
United States
Individual Site Status
Recruiting
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Recruiting
Facility Name
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Name
Washington University School of Medicine / St. Louis Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Cincinnati
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45220
Country
United States
Individual Site Status
Recruiting
Facility Name
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Individual Site Status
Recruiting
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Individual Site Status
Recruiting
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Individual Site Status
Recruiting
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Individual Site Status
Recruiting
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Individual Site Status
Recruiting
Facility Name
Royal Papworth Hospital NHS Foundation Trust
City
Cambridge
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Clinical Research Facility, Queen Elizabeth University Hospital
City
Glasgow
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Royal Brompton Hospital
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Learn more about this trial

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

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