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Trial record 1 of 1 for:    VX18-121-101
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A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03912233
Recruitment Status : Recruiting
First Posted : April 11, 2019
Last Update Posted : July 16, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-121 Drug: TEZ Drug: VX-561 Drug: TEZ/IVA Drug: IVA Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 81 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis
Actual Study Start Date : April 30, 2019
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Part 1: F/MF genotypes VX-121 in TC with TEZ/VX-561
Subjects will receive multiple dose levels of VX-121 in TC with TEZ/VX-561.
Drug: VX-121
VX-121 tablet for oral administration.

Drug: TEZ
TEZ tablet for oral administration.
Other Names:
  • VX-661
  • Tezacaftor

Drug: VX-561
VX-561 tablet for oral administration.
Other Name: CTP-656

Placebo Comparator: Part 1: Placebo Drug: Placebo
Matched Placebo.

Experimental: Part 2: F/F genotypes VX-121 in TC with TEZ/VX-561
Subjects will receive VX-121 in TC with TEZ/VX-561
Drug: VX-121
VX-121 tablet for oral administration.

Drug: TEZ
TEZ tablet for oral administration.
Other Names:
  • VX-661
  • Tezacaftor

Drug: VX-561
VX-561 tablet for oral administration.
Other Name: CTP-656

Active Comparator: Part 2: Placebo + TEZ/IVA Drug: TEZ/IVA
100 mg TEZ/150-mg IVA, film-coated fixed-dose combination (FDC) tablet for oral administration.
Other Names:
  • VX-661/VX-770
  • Tezacaftor/Ivacaftor

Drug: IVA
150-mg film-coated tablet for oral administration.
Other Names:
  • VX-770
  • Ivacaftor

Drug: Placebo
Matched Placebo.




Primary Outcome Measures :
  1. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From baseline up to Day 75 ]
  2. Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: From baseline through Day 29 ]

Secondary Outcome Measures :
  1. Absolute change in sweat chloride concentrations [ Time Frame: From baseline through Day 29 ]
  2. Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: From baseline at Day 29 ]
  3. Maximum observed concentration (Cmax) of VX-121, TEZ, VX-561, IVA (Part 2), and relevant metabolites [ Time Frame: From Day 1 up to Day 29 ]
  4. Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-121, TEZ, VX-561, IVA (Part 2), and relevant metabolites [ Time Frame: From Day 1 up to Day 29 ]
  5. Observed pre-dose concentration (Ctrough) of VX-121, TEZ, VX-561, IVA (Part 2), and relevant metabolites [ Time Frame: From Day 1 up to Day 29 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Part 1: Heterozygous for F508del and an MF mutation (F/MF)
  • Part 2: Homozygous for F508del (F/F)
  • FEV1 value ≥40% and ≤90% of the predicted mean for age, sex, and height

Key Exclusion Criteria:

  • History of clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • History of solid organ or hematological transplantation

Other protocol-defined Inclusion/Exclusion criteria may apply


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): NCT03912233


Contacts
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Contact: Medical Information 6173416777 medicalinfo@vrtx.com

Locations
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United States, California
Keck Medical Center of University of Southern California Recruiting
Los Angeles, California, United States, 90033
United States, Kentucky
University of Kentucky. Recruiting
Lexington, Kentucky, United States, 40536
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
United States, North Carolina
Wake Forest University Baptist Medical Center Recruiting
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Oklahoma
Santiago Reyes, M.D. Recruiting
Oklahoma City, Oklahoma, United States, 73112
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
Germany
Charite Paediatric Pulmonology Department Recruiting
Berlin, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen Recruiting
Essen, Germany
Pneumologisches Studienzentrum Muenchen-West Recruiting
Muenchen, Germany
United Kingdom
University Hospitals Birmingham NHS Foundation Trust Recruiting
Birmingham, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital Recruiting
London, United Kingdom
Wythenshawe Hospital Recruiting
Manchester, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary Recruiting
Newcastle Upon Tyne, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough Recruiting
Penarth, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03912233     History of Changes
Other Study ID Numbers: VX18-121-101
First Posted: April 11, 2019    Key Record Dates
Last Update Posted: July 16, 2019
Last Verified: July 2019
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.: Yes

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