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A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

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ClinicalTrials.gov Identifier: NCT03460990
Recruitment Status : Completed
First Posted : March 9, 2018
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-659 Drug: TEZ Drug: IVA Drug: TEZ/IVA Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 116 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
Actual Study Start Date : May 1, 2018
Actual Primary Completion Date : September 26, 2018
Actual Study Completion Date : October 8, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Triple Combination
Subjects will receive 240 mg VX-659 / 100 mg TEZ / 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
 Drug: VX-659
Fixed-dose combination (FDC) tablet (VX-659/TEZ/IVA)

Drug: TEZ
FDC tablet (VX-659/TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (VX-659/TEZ/IVA)
Other Name: ivacaftor; VX-770

Drug: IVA
IVA tablet
Other Name: ivacaftor; VX-770
Active Comparator: TEZ/IVA
Subjects will receive 100 mg TEZ/150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
 Drug: TEZ/IVA
FDC tablet TEZ/IVA
Other Name: ivacaftor/tezacaftor

Drug: IVA
IVA tablet
Other Name: ivacaftor; VX-770


Outcome Measures
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Primary Outcome Measures :
  1. Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline at Week 4 ]

Secondary Outcome Measures :
  1. Absolute change in CF Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Week 4 ]
  2. Absolute change in Sweat Chloride (SwCl) [ Time Frame: from baseline at Week 4 ]
  3. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through 4-week safety follow-up (up to12 weeks) ]
  4. Observed pre-dose concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through Week 4 ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Homozygous for the F508del mutation (F/F)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Solid organ or hematological transplantation

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): NCT03460990


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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03460990     History of Changes
Other Study ID Numbers: VX17-659-103
2017-004133-82 ( EudraCT Number )
First Posted: March 9, 2018    Key Record Dates
Last Update Posted: December 4, 2018
Last Verified: December 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Fibrosis
Cystic 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