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A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

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ClinicalTrials.gov Identifier: NCT03447249
Recruitment Status : Active, not recruiting
First Posted : February 27, 2018
Last Update Posted : September 18, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

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 heterozygous for F508del and a minimal function mutation (F/MF subjects)

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 385 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 Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Actual Study Start Date : March 7, 2018
Estimated Primary Completion Date : April 16, 2019
Estimated Study Completion Date : April 16, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

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

Placebo Comparator: Placebo Drug: Matched Placebos
Placebo will be used as a comparator.




Primary Outcome Measures :
  1. Absolute change in ppFEV1 [ Time Frame: from baseline at Week 4 ]

Secondary Outcome Measures :
  1. Absolute change in ppFEV1 [ Time Frame: from baseline through Week 24 ]
  2. Number of pulmonary exacerbations (PEx) [ Time Frame: through Week 24 ]
  3. Absolute change in body mass index (BMI) [ Time Frame: from baseline at Week 24 ]
  4. Absolute change in Sweat Chloride (SwCl) [ Time Frame: from baseline at Week 4 and through Week 24 ]
  5. Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Week 4 and through Week 24 ]
  6. Time-to-first PEx [ Time Frame: through Week 24 ]
  7. Absolute change in BMI z score [ Time Frame: from baseline at Week 24 ]
  8. Absolute change in body weight [ Time Frame: from baseline at Week 24 ]
  9. 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 to 28 weeks) ]
  10. Observed pre-dose concentration (Ctrough) of VX-659, 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:

  • Heterozygous for F508del and an MF mutation (as defined in the protocol ).
  • 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
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency
  • 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): NCT03447249


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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03447249     History of Changes
Other Study ID Numbers: VX17-659-102
2017-004132-11 ( EudraCT Number )
First Posted: February 27, 2018    Key Record Dates
Last Update Posted: September 18, 2018
Last Verified: May 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