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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02544451
• Date of registration: 01/09/2015
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
• Scientific title: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
• Date of first enrolment: August 2015
• Target sample size: 246
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02544451
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Denmark, France, Germany, Sweden, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

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.

Exclusion Criteria (Treatment Cohort Only):

- 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).



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: LUM/IVA

Primary Outcome(s)

Treatment Cohort: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 100]

Secondary Outcome(s)

Absolute Change in Body Mass Index (BMI) [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Height-for-age Z-score [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Height [Time Frame: From Parent Study Baseline at Week 96]

Number of Pulmonary Exacerbation Events Per Patient-year [Time Frame: From Parent Study Baseline through Week 96]

Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 100]

Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event [Time Frame: From Parent Study Baseline through Week 96]

Time-to-first Pulmonary Exacerbation [Time Frame: From Parent Study Baseline through Week 96]

Absolute Change in BMI-for-age Z-score [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Weight [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Weight-for-age Z-score [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Sweat Chloride [Time Frame: From Parent Study Baseline at Week 96]

Rate of Change in LCI 2.5 [Time Frame: Day 15 after first dose of LUM/IVA through Week 96]

Rate of Change in LCI 5.0 [Time Frame: Day 15 after first dose of LUM/IVA through Week 96]

Rate of Change in ppFEV1 [Time Frame: Day 15 after first dose of LUM/IVA through Week 96]

Absolute Change in LCI 5.0 [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score [Time Frame: From Parent Study Baseline at Week 96]

Absolute Change in Lung Clearance Index (LCI) 2.5 [Time Frame: From Parent Study Baseline at Week 96]

Relative Change in ppFEV1 [Time Frame: From Parent Study Baseline at Week 96]

Secondary ID(s)

VX15-809-110

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 29/10/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02544451