A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
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ClinicalTrials.gov Identifier: NCT03224351 |
Recruitment Status :
Completed
First Posted : July 21, 2017
Last Update Posted : March 5, 2019
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Condition or disease | Intervention/treatment | Phase |
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Cystic Fibrosis | Drug: TEZ/IVA Drug: VX-659 Drug: IVA Drug: Matched Placebos Drug: TEZ Drug: VX-561 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 124 participants |
Allocation: | Randomized |
Intervention Model: | Parallel 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-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis |
Actual Study Start Date : | August 8, 2017 |
Actual Primary Completion Date : | February 28, 2018 |
Actual Study Completion Date : | February 28, 2018 |

Arm | Intervention/treatment |
---|---|
Experimental: Part 1: F/MF genotype -TC Low
Subjects will receive 80 mg of VX-659 qd in TC with TEZ and IVA for 4 weeks.
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Drug: TEZ/IVA
100-mg TEZ/150-mg IVA fixed-dose combination (FDC) tablet for oral administration
Other Name: VX-661/VX-770 Drug: VX-659 80-mg VX-659 tablet for oral administration Drug: IVA 150-mg IVA tablet for oral administration
Other Name: VX-770 |
Experimental: Part 1: F/MF genotype - TC Mid
Subjects will receive 240 mg of VX-659 qd in TC with TEZ and IVA for 4 weeks.
|
Drug: TEZ/IVA
100-mg TEZ/150-mg IVA fixed-dose combination (FDC) tablet for oral administration
Other Name: VX-661/VX-770 Drug: VX-659 80-mg VX-659 tablet for oral administration Drug: IVA 150-mg IVA tablet for oral administration
Other Name: VX-770 |
Experimental: Part 1: F/MF genotype - TC High
Subjects will receive 400 mg VX-659 qd in TC with TEZ and IVA for 4 weeks.
|
Drug: TEZ/IVA
100-mg TEZ/150-mg IVA fixed-dose combination (FDC) tablet for oral administration
Other Name: VX-661/VX-770 Drug: VX-659 80-mg VX-659 tablet for oral administration Drug: IVA 150-mg IVA tablet for oral administration
Other Name: VX-770 |
Placebo Comparator: Part 1: F/MF genotype - Placebo
Subjects will receive placebo for 4 weeks.
|
Drug: Matched Placebos
Placebo will be used as a comparator. |
Experimental: Part 2: F/F genotype - TC
Subjects will receive 400 mg of VX-659 qd in TC with TEZ and IVA for 4 weeks.
|
Drug: TEZ/IVA
100-mg TEZ/150-mg IVA fixed-dose combination (FDC) tablet for oral administration
Other Name: VX-661/VX-770 Drug: VX-659 80-mg VX-659 tablet for oral administration Drug: IVA 150-mg IVA tablet for oral administration
Other Name: VX-770 |
Active Comparator: Part 2: F/F genotype - TEZ/IVA
Subjects will receive TEZ and IVA for 4 weeks.
|
Drug: TEZ/IVA
100-mg TEZ/150-mg IVA fixed-dose combination (FDC) tablet for oral administration
Other Name: VX-661/VX-770 Drug: IVA 150-mg IVA tablet for oral administration
Other Name: VX-770 Drug: Matched Placebos Placebo will be used as a comparator. |
Experimental: Part 3: F/MF genotype - TC
Subjects will receive 400 mg of VX-659 qd in TC with TEZ and VX-561 for 4 weeks.
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Drug: VX-659
80-mg VX-659 tablet for oral administration Drug: TEZ 50-mg tablet for oral administration. Drug: VX-561 50-mg VX-561 tablet for oral administration.
Other Name: CTP-656 |
Placebo Comparator: Part 3: F/MF genotype - Placebo
Subjects will receive placebo for 4 weeks.
|
Drug: Matched Placebos
Placebo will be used as a comparator. |
- Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From baseline through safety follow-up (20 Weeks) ]Number of subjects with AEs and SAEs will be reported.
- Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline through Day 29 ]Absolute change in ppFEV1 will be reported.
- Absolute change in sweat chloride concentrations [ Time Frame: from baseline through Day 29 ]Absolute change in sweat chloride concentrations will be reported.
- Relative change in ppFEV1 [ Time Frame: from baseline through Day 29 ]Relative change in ppFEV1 will be reported.
- Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Day 29 ]The absolute change in CFQ-R respiratory domain score will be reported.
- Maximum observed concentration (Cmax) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561 [ Time Frame: from Day 1 through Day 29 ]Maximum plasma concentration [CMax] will be reported.
- Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561 [ Time Frame: from Day 1 through Day 29 ]The area under the plasma concentration time curve over the dosing interval (AUCtau) will be reported.
- Observed pre-dose concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561 [ Time Frame: from Day 1 through Day 29 ]The observed predose concentration (Ctrough) will be reported.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Body weight ≥35 kg.
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Subjects must have an eligibleCFTR genotype.
- Part 1 and Part 3: Heterozygous for F508del and an MF mutation (F/MF)
- Part 2: Homozygous for F508del (F/F)
- FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height
Key Exclusion Criteria:
- History of 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.
- History of solid organ or hematological transplantation.
Other protocol defined Inclusion/Exclusion criteria may apply.

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): NCT03224351

Responsible Party: | Vertex Pharmaceuticals Incorporated |
ClinicalTrials.gov Identifier: | NCT03224351 |
Other Study ID Numbers: |
VX16-659-101 2016-003585-11 ( EudraCT Number ) |
First Posted: | July 21, 2017 Key Record Dates |
Last Update Posted: | March 5, 2019 |
Last Verified: | February 2019 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
VX-659 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 |