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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05668741




Registration number
NCT05668741
Ethics application status
Date submitted
19/12/2022
Date registered
30/12/2022

Titles & IDs
Public title
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
Scientific 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
Secondary ID [1] 0 0
2022-000726-25
Secondary ID [2] 0 0
VX21-522-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - VX-522 mRNA therapy
Treatment: Drugs - IVA

Experimental: Single Ascending Dose (SAD) - Participants grouped into different cohorts will receive a single ascending dose of VX-522.

Experimental: Multiple Ascending Dose (MAD) Arm 1 - Participants grouped into different cohorts will receive multiple ascending doses of VX-522 in treatment arm 1 (T1).

Experimental: MAD Arm 2: VX522+ IVA - Following run-in period with ivacaftor (IVA), participants will receive multiple doses of VX-522 with IVA in treatment arm (T2).


Treatment: Drugs: VX-522 mRNA therapy
Oral inhalation using nebulizer.

Treatment: Drugs: IVA
Tablet for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [1] 0 0
From Day 1 Through Safety Follow-up Visit [up to Week 24 for SAD, and Week 28 for T1 and T2 (MAD)]
Secondary outcome [1] 0 0
T1 (MAD): Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Timepoint [1] 0 0
From Baseline at Day 29
Secondary outcome [2] 0 0
T2 (MAD): Change From Pre-Run-in Baseline in ppFEV1
Timepoint [2] 0 0
From Pre-Run Baseline at Day 29

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/02/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2025
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Alfred Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
South Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Wisconsin
Country [17] 0 0
Canada
State/province [17] 0 0
Calgary
Country [18] 0 0
Canada
State/province [18] 0 0
Quebec
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Cambridge
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Glasgow
Country [21] 0 0
United Kingdom
State/province [21] 0 0
London
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Manchester
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Penarth
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Moderna, Inc
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Information
Address 0 0
Country 0 0
Phone 0 0
617-341-6777
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
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


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05668741