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

Sponsor

Vertex Pharmaceuticals Incorporated (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05668741

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.8

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Biological: VX-522	Phase 1

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.).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

9 participants

Allocation:

N/A

Intervention Model:

Sequential Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Anticipated Study Start Date :

Jan 1, 2023

Anticipated Primary Completion Date :

Jan 1, 2024

Anticipated Study Completion Date :

Jan 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: VX-522 Participants will receive a single dose of different dose levels of VX-522.	Biological: VX-522 Oral inhalation using nebulizer.

Arm

Intervention/Treatment

Experimental: VX-522

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

Biological: VX-522

Oral inhalation using nebulizer.

Outcome Measures

Primary Outcome Measures

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 (Week 24)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Body mass index (BMI) of greater than or equal to (≥)18.0 to 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, 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Baltimore - Early Phase Clinical Unit	Baltimore	Maryland	United States	21225

Sponsors and Collaborators

Vertex Pharmaceuticals Incorporated

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Vertex Pharmaceuticals Incorporated

ClinicalTrials.gov Identifier:

NCT05668741

Other Study ID Numbers:

VX21-522-001
2022-000726-25

First Posted:

Dec 30, 2022

Last Update Posted:

Jan 6, 2023

Last Verified:

Dec 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Additional relevant MeSH terms:

Cystic Fibrosis

Fibrosis

Study Results

No Results Posted as of Jan 6, 2023