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Trial record 1 of 2 for:    445-103
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A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

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ClinicalTrials.gov Identifier: NCT03525548
Recruitment Status : Active, not recruiting
First Posted : May 15, 2018
Last Update Posted : December 4, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the efficacy of VX-445 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-445 Drug: TEZ Drug: IVA Drug: TEZ/IVA Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 113 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
Actual Study Start Date : August 3, 2018
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : March 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Triple Combination
Subjects will receive 200 mg VX-445/ 100 mg TEZ/ 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening
Drug: VX-445
Fixed-dose combination (FDC) tablet (VX-445/TEZ/IVA)

Drug: TEZ
FDC tablet (VX-445/TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (VX-445/TEZ/IVA)
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 Names:
  • tezacaftor/ivacaftor
  • VX-661/VX-770




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 the number of adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up visit (up to 16 weeks) ]
  4. Observed pre-dose concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through Week 16 ]


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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


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): NCT03525548


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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03525548     History of Changes
Other Study ID Numbers: VX17-445-103
2018-000184-89 ( EudraCT Number )
First Posted: May 15, 2018    Key Record Dates
Last Update Posted: December 4, 2018
Last Verified: December 2018

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

Additional relevant MeSH terms:
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