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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05668741
Recruitment Status : Recruiting
First Posted : December 30, 2022
Last Update Posted : April 25, 2024
Sponsor:
Collaborator:
Moderna, Inc
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-522 mRNA therapy Drug: IVA Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : February 27, 2023
Estimated Primary Completion Date : March 2025
Estimated Study Completion Date : March 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arms and Interventions
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Arm Intervention/treatment
Experimental: Single Ascending Dose (SAD)
Participants grouped into different cohorts will receive a single ascending dose of VX-522.
 Drug: VX-522 mRNA therapy
Oral inhalation using nebulizer.
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).
 Drug: VX-522 mRNA therapy
Oral inhalation using nebulizer.
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).
 Drug: VX-522 mRNA therapy
Oral inhalation using nebulizer.

Drug: IVA
Tablet for oral administration.
Other Names:
  • ivacaftor
  • VX-770


Outcome Measures
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Primary Outcome Measures :
  1. 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 [up to Week 24 for SAD, and Week 28 for T1 and T2 (MAD)] ]

Secondary Outcome Measures :
  1. T1 (MAD): Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline at Day 29 ]
  2. T2 (MAD): Change From Pre-Run-in Baseline in ppFEV1 [ Time Frame: From Pre-Run Baseline at Day 29 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05668741


Contacts
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Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Moderna, Inc
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT05668741    
Other Study ID Numbers: VX21-522-001
2022-000726-25 ( EudraCT Number )
2023-504786-23-00 ( Other Identifier: EU CT Number )
First Posted: December 30, 2022    Key Record Dates
Last Update Posted: April 25, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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

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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
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action