Phase 3
Completed N=245
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Source: ClinicalTrials.gov NCT02544451 ↗Enrolled (actual)
245
Serious AEs
28.6%
Results posted
Oct 2019
Primary outcomePrimary: 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) — 142; 94; 43; 29 participants
◆ Published Evidence
Established
42citations · ~8 / year
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.
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.
Linked Publications
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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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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) |
142; 94; 43; 29 | — |
| SECONDARY Absolute Change in Lung Clearance Index (LCI) 2.5 |
-0.85; -0.86 | — |
| SECONDARY Absolute Change in Sweat Chloride |
-22.9; -22.8 | — |
| SECONDARY Absolute Change in Body Mass Index (BMI) |
1.78; 2.04 | — |
| SECONDARY Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
7.4; 6.6 | — |
| SECONDARY Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) |
1 | — |
| SECONDARY Absolute Change in LCI 5.0 |
-0.21; -0.31 | — |
| SECONDARY Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
3.1; 0.0 | — |
| SECONDARY Relative Change in ppFEV1 |
4.9; 0.5 | — |
| SECONDARY Absolute Change in BMI-for-age Z-score |
0.17; 0.31 | — |
| SECONDARY Absolute Change in Weight |
10.3; 11.0 | — |
| SECONDARY Absolute Change in Weight-for-age Z-score |
0.12; 0.24 | — |
| SECONDARY Absolute Change in Height |
13.4; 13.5 | — |
| SECONDARY Absolute Change in Height-for-age Z-score |
-0.01; 0.02 | — |
| SECONDARY Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score |
5.1; 3.9 | — |
| SECONDARY Time-to-first Pulmonary Exacerbation |
720.00; NA | — |
| SECONDARY Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event |
49.5; 32.3 | — |
| SECONDARY Number of Pulmonary Exacerbation Events Per Patient-year |
0.45; 0.30 | — |
| SECONDARY Rate of Change in LCI 2.5 |
-0.01 | — |
| SECONDARY Rate of Change in LCI 5.0 |
0.00 | — |
| SECONDARY Rate of Change in ppFEV1 |
0.58 | — |
| SECONDARY Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
9; 0 | — |
Eligibility 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).
Data sourced from ClinicalTrials.gov (NCT02544451) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.