Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

NCT02544451 | Completed Phase 3 Results DMC

• 1 other identifier: VX15-809-110
• Study Type: interventional
• Target: 246
• Locations: 9 countries
• Sites: 60

Brief Summary

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Day 1 up to Week 100

Secondary Outcomes (21)

• Absolute Change in Lung Clearance Index (LCI) 2.5

  From Parent Study Baseline at Week 96

• Absolute Change in Sweat Chloride

  From Parent Study Baseline at Week 96

• Absolute Change in Body Mass Index (BMI)

  From Parent Study Baseline at Week 96

• Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

  From Parent Study Baseline at Week 96

• Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)

  Day 1 up to Week 100

Study Arms (4)

Treatment Period 1: LUM/IVA to LUM/IVA

EXPERIMENTAL

Drug: LUM/IVA

Treatment Period 1: Placebo (PBO) to LUM/IVA

EXPERIMENTAL

Drug: LUM/IVA

Treatment Period 1: Observational Cohort

NO INTERVENTION

Treatment Period 2: LUM/IVA

EXPERIMENTAL

Drug: LUM/IVA

Interventions

LUM/IVA DRUG

Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age). LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).

Also known as: VX-809/VX-770, lumacaftor/ivacaftor

Treatment Period 1: LUM/IVA to LUM/IVA; Treatment Period 1: Placebo (PBO) to LUM/IVA

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects entering the Treatment Cohort must meet both of the following criteria:
• Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
• Willing to remain on a stable CF medication through the Safety Follow-up Visit.
• Subjects entering the Observational Cohort must meet 1 of the following criteria:
• Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
• Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.

You may not qualify if:

• History of any comorbidity or laboratory abnormality 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 subject (e.g., cirrhosis with portal hypertension).
• Pregnant and nursing females.
• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
• History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
• History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
• Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (60)

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Birmingham, Alabama, United States

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Tucson, Arizona, United States

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Long Beach, California, United States

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Palo Alto, California, United States

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Aurora, Colorado, United States

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Wilmington, Delaware, United States

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Jacksonville, Florida, United States

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Orlando, Florida, United States

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Indianapolis, Indiana, United States

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Iowa City, Iowa, United States

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Boston, Massachusetts, United States

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Minneapolis, Minnesota, United States

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Kansas City, Missouri, United States

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St Louis, Missouri, United States

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Omaha, Nebraska, United States

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Lebanon, New Hampshire, United States

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Buffalo, New York, United States

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Syracuse, New York, United States

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Chapel Hill, North Carolina, United States

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Cincinnati, Ohio, United States

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Cleveland, Ohio, United States

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Dayton, Ohio, United States

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Portland, Oregon, United States

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Pittsburgh, Pennsylvania, United States

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Charleston, South Carolina, United States

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Austin, Texas, United States

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Houston, Texas, United States

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Salt Lake City, Utah, United States

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Colchester, Vermont, United States

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Charlottesville, Virginia, United States

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Norfolk, Virginia, United States

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Richmond, Virginia, United States

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Seattle, Washington, United States

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Madison, Wisconsin, United States

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Milwaukee, Wisconsin, United States

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Parkville, Victoria, Australia

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Herston, Australia

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New Lambton Heights, Australia

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Subiaco, Australia

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Westmead, Australia

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Brussels, Belgium

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Leuven, Belgium

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Vancouver, British Columbia, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Copenhagen, Denmark

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Bron, Cedex, France

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Bordeaux, France

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Paris, France

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Berlin, Germany

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Cologne, Germany

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Giessen, Germany

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Hanover, Germany

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Stockholm, Sweden

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Leeds, West Yorkshire, United Kingdom

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Belfast, United Kingdom

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Edinburgh, United Kingdom

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London, United Kingdom

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Related Publications (1)

• Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28.

  PMID: 33516285 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lumacaftor, ivacaftor drug combination

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: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2015
• First Posted: September 9, 2015
• Study Start: August 1, 2015
• Primary Completion: August 1, 2018
• Study Completion: April 1, 2020
• Last Updated: May 24, 2021
• Results First Posted: October 29, 2019

Record last verified: 2021-02

Locations

CA (3); AU (5); BE (2); DK (1); DE (4); GB (4); SE (1); FR (3); US (37)