A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Completed · Phase 3 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

Study VX16-661-114 (Study 114) is a Phase 3b, randomized, double-blind, placebo-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are homozygous for the F508del mutation on the cystic fibrosis transmembrane conductance regulator gene (CFTR) gene and who discontinued treatment with Orkambi due to respiratory symptoms considered related to treatment. This study is designed to evaluate the safety and efficacy of Tezacaftor/Ivacaftor (TEZ/IVA).

Key facts

Study ID

NCT03150719

Run by

Vertex Pharmaceuticals Incorporated

People needed

98

Starts

2017-05-24

Expected to finish

2018-08-09

Last updated by the study team

2019-09-12

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
• Prior discontinuation of Orkambi, with at least 1 respiratory sign or symptom considered related to therapy.
• Resolution or stabilization of qualifying event(s) >28 days prior to Screening.
• Discontinuation of Orkambi therapy must have occurred within approximately 12 weeks from the first dose of Orkambi.
• Homozygous for F508del mutation in the CFTR gene as documented in the subject's medical record. If genotype documentation is not available in the medical record, genotyping will be performed during screening.
• FEV1 ≥25% and ≤90% of predicted normal for age, sex, and height.
• Stable CF disease as judged by the investigator.
• Other protocol defined inclusion criteria could apply.

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 subject.
• Recent rapid or progressive deterioration in respiratory status.
• Receiving continuous oxygen at >2L/min or on face-mask ventilation.
• Any protocol-defined exclusionary laboratory values at Screening.
• Child-Pugh Class B or C hepatic impairment.
• An acute upper or lower respiratory infection, pulmonary exacerbation, or change in therapy for pulmonary disease within 28 days before Day 1.
• Documentation of colonization with organisms associated with a more rapid decline in pulmonary status.
• History of lung transplantation since most recent initiation of Orkambi.
• History of alcohol or drug abuse in the past year as deemed by the investigator.
• Participation in an investigational drug study or use of a CFTR modulator within 28 days or 5 terminal half-lives of the investigational drug or modulator (whichever is longer).
• Use of restricted medications or foods within the specified window before the first dose of study drug, or an anticipated need or use of restricted medication or foods after the first dose of study drug.
• Pregnant or nursing females: Females of child-bearing potential must have a negative pregnancy test at Screening and Day 1.
• Other protocol defined exclusion criteria could apply.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• UCSF - Fresno, Community Regional Medical Center — Fresno, California, United States
• Miller Children's Hospital / Long Beach Memorial — Long Beach, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Rady Children's Hospital — San Diego, California, United States
• National Jewish Health — Denver, Colorado, United States
• Yale New Haven Hospital — New Haven, Connecticut, United States
• Central Florida Pulmonary Group — Orlando, Florida, United States
• Arnold Palmer Hospital — Orlando, Florida, United States
• Tampa General Hospital Cardiac and Lung Transplant Clinic — Tampa, Florida, United States
• Children's Speciality Services at North Druid Hills — Atlanta, Georgia, United States
• St. Luke's CF Center of Idaho — Boise, Idaho, United States
• Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants — Niles, Illinois, United States
• Southern Illinois University — Springfield, Illinois, United States
• Riley Hospital for Children at Indiana University Health — Indianapolis, Indiana, United States
• The University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Kentucky Clinic — Lexington, Kentucky, United States
• Tulane Medical Center — New Orleans, Louisiana, United States
• Massachusetts General Hospital Cystic Fibrosis Center — Boston, Massachusetts, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Billings Clinic — Billings, Montana, United States
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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