Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

NCT02390219 | Completed Phase 3 Results

• 1 other identifier: VX14-809-106
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 6

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation

Conditions

Cystic Fibrosis; Advanced Lung Disease

Outcome Measures

Primary Outcomes (1)

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

  AE: any untoward medical occurrence in a participant during the study; event does not necessarily have a causal relationship with treatment. This includes any newly occurring event/previous condition that has increased in severity/frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event, which falls into any of the following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs: AEs that started/ worsened on/after the start of study drug through the Safety Follow up Visit (4 weeks after the last dose of study drug). Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification.

  Day 1 up to Week 28

Secondary Outcomes (6)

• Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24

  Baseline, Up to Week 24

• Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24

  Baseline, Up to Week 24

• Duration For Which Participants Received Intravenous (IV) Antibiotics

  Baseline through Week 24

• Number of Hospitalizations

  Baseline through Week 24

• Absolute Change From Baseline in Sweat Chloride at Average of Day 15 and Week 4

  Baseline, Day 15 and Week 4

Study Arms (1)

Lumacaftor/Ivacaftor combination

EXPERIMENTAL

Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks.

Drug: Lumacaftor; Drug: Ivacaftor

Interventions

Lumacaftor DRUG

Also known as: VX-809

Lumacaftor/Ivacaftor combination

Ivacaftor DRUG

Also known as: VX-770

Lumacaftor/Ivacaftor combination

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; historical genotype must be documented in the participant's source documents.
• Percent predicted FEV1 \<40 of adjusted for age, sex, and height at Screening

You may not qualify if:

• Participant currently receiving invasive mechanical ventilation.
• 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
• Any clinically significant laboratory abnormalities at screening that would interfere with the study assessments or pose an undue risk for the subject
• A 12-lead electrocardiograms (ECG) demonstrating QTcF \>450 msec at Screening
• History of solid organ or hematological transplantation
• History of alcohol or drug abuse in the past year
• Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
• Use of strong inhibitors, moderate inducers, or strong inducers of CYP3A
• Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at Screening and Day 1.
• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
• Use of beta blockers or the equivalent at Screening.

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (6)

Unknown Facility

Denver, Colorado, United States

Location

Unknown Facility

Tampa, Florida, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Related Publications (1)

• Taylor-Cousar JL, Jain M, Barto TL, Haddad T, Atkinson J, Tian S, Tang R, Marigowda G, Waltz D, Pilewski J; VX14-809-106 Investigator Group. Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. J Cyst Fibros. 2018 Mar;17(2):228-235. doi: 10.1016/j.jcf.2017.09.012. Epub 2017 Nov 8.

  PMID: 29126871 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

lumacaftor; 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: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2015
• First Posted: March 17, 2015
• Study Start: March 1, 2015
• Primary Completion: October 1, 2016
• Study Completion: October 1, 2016
• Last Updated: December 6, 2017
• Results First Posted: November 1, 2017

Record last verified: 2017-10

Locations

US (6)