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A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03912233
Recruitment Status : Completed
First Posted : April 11, 2019
Last Update Posted : January 10, 2020
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)
Actual Enrollment : 87 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
Actual Primary Completion Date : October 22, 2019
Actual Study Completion Date : December 10, 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 ]


Information from the National Library of Medicine

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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


Locations
Show Show 26 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03912233    
Other Study ID Numbers: VX18-121-101
First Posted: April 11, 2019    Key Record Dates
Last Update Posted: January 10, 2020
Last Verified: January 2020
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