A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

NCT02347657 | Completed Phase 3 Results DMC

• 1 other identifier: VX14-661-106
• Study Type: interventional
• Target: 510
• Locations: 12 countries
• Sites: 74

Brief Summary

This is a Phase 3, randomized, double blind, placebo controlled, parallel group, multicenter study in people with cystic fibrosis (CF) who are homozygous for the F508del CF transmembrane conductance regulator (CFTR) gene mutation.

Conditions

Cystic Fibrosis

Keywords

Homozygous for the F508del CFTR Mutation

Outcome Measures

Primary Outcomes (1)

• Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24

  FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  Day 1, Through Week 24

Secondary Outcomes (10)

• Relative Change From Baseline (Day 1) in ppFEV1 Through Week 24

  Day 1, Through Week 24

• Number of Pulmonary Exacerbations Per Year

  Day 1 through Week 24

• Absolute Change From Baseline (Day 1) Body Mass Index (BMI) at Week 24

  Day 1, Week 24

• Absolute Change From Baseline (Day 1) in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24

  Day 1, Through Week 24

• Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Day 1 up to Week 28

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA tablet administered orally in the evening up to Week 24.

Drug: VX-661 Plus Ivacaftor Combination Placebo; Drug: Ivacaftor placebo

VX-661/IVA

EXPERIMENTAL

VX-661 100 mg plus IVA 150 mg FDC tablet administered orally in the morning and IVA 150 mg tablet administered orally in the evening up to Week 24.

Drug: VX-661 Plus Ivacaftor Combination; Drug: Ivacaftor

Interventions

VX-661 Plus Ivacaftor Combination DRUG

FDC tablet, oral use

VX-661/IVA

Ivacaftor DRUG

Tablet, oral use

VX-661/IVA

VX-661 Plus Ivacaftor Combination Placebo DRUG

FDC tablet, oral use

Placebo

Ivacaftor placebo DRUG

Tablet, oral use

Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Homozygous for the F508del CFTR mutation, genotype to be confirmed at the Screening Visit
• Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
• Forced expiratory volume at one second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height during screening
• Stable CF disease as judged by the investigator
• Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit

You may not qualify if:

• History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant.
• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)
• Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Day 1)
• Sexually active participants of reproductive potential who are not willing to follow the contraception requirements

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (74)

Unknown Facility

Little Rock, Arkansas, United States

Location

Unknown Facility

Long Beach, California, United States

Location

Unknown Facility

Denver, Colorado, United States

Location

Unknown Facility

Orlando, Florida, United States

Location

Unknown Facility

Tampa, Florida, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Peoria, Illinois, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Manchester, New Hampshire, United States

Location

Unknown Facility

Long Branch, New Jersey, United States

Location

Unknown Facility

Albuquerque, New Mexico, United States

Location

Unknown Facility

Albany, New York, United States

Location

Unknown Facility

Buffalo, New York, United States

Location

Unknown Facility

New Hyde Park, New York, United States

Location

Unknown Facility

Rochester, New York, United States

Location

Unknown Facility

Syracuse, New York, United States

Location

Unknown Facility

Akron, Ohio, United States

Location

Unknown Facility

Columbus, Ohio, United States

Location

Unknown Facility

Oklahoma City, Oklahoma, United States

Location

Unknown Facility

Charleston, South Carolina, United States

Location

Unknown Facility

Sioux Falls, South Dakota, United States

Location

Unknown Facility

Fort Worth, Texas, United States

Location

Unknown Facility

Tyler, Texas, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Milwaukee, Wisconsin, United States

Location

Unknown Facility

Calgary, Alberta, Canada

Location

Unknown Facility

Vancouver, British Columbia, Canada

Location

Unknown Facility

Halifax, Nova Scotia, Canada

Location

Unknown Facility

Toronto, Ontario, Canada

Location

Unknown Facility

Montreal, Quebec, Canada

Location

Unknown Facility

Copenhagen O, Denmark

Location

Unknown Facility

Marseille, Bouches-du-Rhone, France

Location

Unknown Facility

Roscoff, Finistere, France

Location

Unknown Facility

Bordeaux, Girande, France

Location

Unknown Facility

Lille, Nord, France

Location

Unknown Facility

Paris, Paris, France

Location

Unknown Facility

Pierre-Bénite, Rhone, France

Location

Unknown Facility

Heidelberg, Baden-Wurttemberg, Germany

Location

Unknown Facility

Munich, Bavaria, Germany

Location

Unknown Facility

Würzburg, Bavaria, Germany

Location

Unknown Facility

Frankfurt am Main, Hesse, Germany

Location

Unknown Facility

Giessen, Hesse, Germany

Location

Unknown Facility

Belfast, State of Berlin, Germany

Location

Unknown Facility

Berlin, Germany

Location

Unknown Facility

Cork, Ireland

Location

Unknown Facility

Dublin, Ireland

Location

Unknown Facility

Genova, Italy

Location

Unknown Facility

Palermo, Italy

Location

Unknown Facility

Roma, Italy

Location

Unknown Facility

Torino, Italy

Location

Unknown Facility

Verona, Italy

Location

Unknown Facility

Groningen, Netherlands

Location

Unknown Facility

Nijmegen, Netherlands

Location

Unknown Facility

Rotterdam, Netherlands

Location

Unknown Facility

The Hague, Netherlands

Location

Unknown Facility

Utrecht, Netherlands

Location

Unknown Facility

Barcelona, Spain

Location

Unknown Facility

Madrid, Spain

Location

Unknown Facility

Seville, Spain

Location

Unknown Facility

Valencia, Spain

Location

Unknown Facility

Gothenburg, Sweden

Location

Unknown Facility

Stockholm, Sweden

Location

Unknown Facility

Uppsala, Sweden

Location

Unknown Facility

Bern, Switzerland

Location

Unknown Facility

Zurich, Switzerland

Location

Unknown Facility

Exeter, Devon, United Kingdom

Location

Unknown Facility

Liverpool, Lancashire, United Kingdom

Location

Unknown Facility

Sheffield, South Yorkshire, United Kingdom

Location

Unknown Facility

Newcastle upon Tyne, Tyne & Wear, United Kingdom

Location

Unknown Facility

Birmingham, West Midlands, United Kingdom

Location

Unknown Facility

Belfast, United Kingdom

Location

Unknown Facility

London, United Kingdom

Location

Related Publications (2)

• Acaster S, Mukuria C, Rowen D, Brazier JE, Wainwright CE, Quon BS, Duckers J, Quittner AL, Lou Y, Sosnay PR, McGarry LJ. Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised. Value Health. 2023 Apr;26(4):567-578. doi: 10.1016/j.jval.2022.12.002. Epub 2022 Dec 9.

  PMID: 36509366 DERIVED

• Taylor-Cousar JL, Munck A, McKone EF, van der Ent CK, Moeller A, Simard C, Wang LT, Ingenito EP, McKee C, Lu Y, Lekstrom-Himes J, Elborn JS. Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del. N Engl J Med. 2017 Nov 23;377(21):2013-2023. doi: 10.1056/NEJMoa1709846. Epub 2017 Nov 3.

  PMID: 29099344 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

tezacaftor; ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

617-341-6777

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: January 12, 2015
• First Posted: January 27, 2015
• Study Start: January 1, 2015
• Primary Completion: January 20, 2017
• Study Completion: January 20, 2017
• Last Updated: June 12, 2018
• Results First Posted: June 12, 2018

Record last verified: 2018-05

Locations

GB (7); DK (1); DE (7); IE (2); IT (5); NL (5); SE (3); CH (2); US (27); CA (5); FR (6); ES (4)