A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
A Phase 3, Rollover Study to Evaluate the Safety of Long-term Treatment With Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
2 other identifiers
interventional
57
2 countries
20
Brief Summary
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started May 2017
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 19, 2017
CompletedFirst Posted
Study publicly available on registry
April 24, 2017
CompletedStudy Start
First participant enrolled
May 12, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 17, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
July 17, 2019
CompletedResults Posted
Study results publicly available
August 7, 2020
CompletedAugust 7, 2020
July 1, 2020
2.2 years
April 19, 2017
July 15, 2020
July 15, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 98
Secondary Outcomes (15)
Absolute Change in Sweat Chloride
From Parent Study 115B Baseline at Week 96
Absolute Change in Body Mass Index (BMI)
From Parent Study 115B Baseline at Week 96
Absolute Change in BMI-for-age Z-score
From Parent Study 115B Baseline at Week 96
Absolute Change in Weight
From Parent Study 115B Baseline at Week 96
Absolute Change in Weight-for-age Z-score
From Parent Study 115B Baseline at Week 96
- +10 more secondary outcomes
Study Arms (1)
LUM/IVA
EXPERIMENTALLUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.
Interventions
Participants received LUM/IVA every q12h.
Eligibility Criteria
You may qualify if:
- Subjects entering the Treatment Cohort must meet the following criteria:
- Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study VX15-809-115 Part B (Study 115B, NCT02797132)
- Willing to remain on a stable CF medication regimen through the Safety Follow-up Visit
- Subjects entering the Observational Cohort must meet 1 of the following criteria:
- Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study 115B, but do not want to enroll in the Treatment Cohort.
- Received at least 4 weeks of LUM/IVA treatment and completed visits up to Week 24 and the Safety Follow-up Visit, if required, of Study 115B but are not taking LUM/IVA at the end of the Study 115B Treatment Period (i.e., Week 24) because of a drug interruption and either did not receive Vertex approval to enroll in the Treatment Cohort or do not want to enroll in the Treatment Cohort.
- Permanently discontinued LUM/IVA in Study 115B after receiving at least 4 weeks of treatment and remained in the study from the time of treatment discontinuation through the Week 24 Visit and Safety Follow-up Visit, if required.
You may not qualify if:
- Prematurely discontinued LUM/IVA treatment in Study 115B.
- 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 LUM/IVA to the subject
- History of drug intolerance or other serious reactions to LUM/IVA in Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor.
- Subjects with a history of allergy or hypersensitivity to LUM/IVA.
- Liver function test (LFT) abnormality meeting criteria for LUM/IVA treatment interruption at the completion of Study 115B, for which no convincing alternative etiology is identified.
- QTc value at the completion of Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor
- History of poor compliance with LUM/IVA and/or procedures in Study 115B as deemed by the investigator.
- Participation in an investigational drug trial (including studies investigating LUM and/or IVA) other than Study 115B.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
Stanford University
Palo Alto, California, 94305, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minn
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
The Lung & Cystic Fibrosis Center at Women's & Children's Hospital of Buffalo
Buffalo, New York, 14222, United States
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
University Hospitals Cleveland Medical Center/Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
British Columbia's Children's Hospital
Vancouver, British Columbia, V6H 3N1, Canada
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Quebec, H4A 3J1, Canada
Related Publications (1)
Hoppe JE, Chilvers M, Ratjen F, McNamara JJ, Owen CA, Tian S, Zahigian R, Cornell AG, McColley SA. Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study. Lancet Respir Med. 2021 Sep;9(9):977-988. doi: 10.1016/S2213-2600(21)00069-2. Epub 2021 May 6.
PMID: 33965000DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- 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
April 19, 2017
First Posted
April 24, 2017
Study Start
May 12, 2017
Primary Completion
July 17, 2019
Study Completion
July 17, 2019
Last Updated
August 7, 2020
Results First Posted
August 7, 2020
Record last verified: 2020-07