Phase 3
Completed N=204
A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
Source: ClinicalTrials.gov NCT02514473 ↗Enrolled (actual)
204
Serious AEs
11.8%
Results posted
Oct 2017
Primary outcomePrimary: Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24 — 0.08; -1.01 Ratio — p=< 0.0001
◆ Published Evidence
Highly cited
295citations · ~33 / year
Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial.
Summary
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
Linked Publications (3)
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Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial.
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24 |
0.08; -1.01 | < 0.0001 sig |
| SECONDARY Average Absolute Change From Baseline in Sweat Chloride at Day 15 and Week 4 |
0.8; -20.0 | — |
| SECONDARY Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 |
0.27; 0.38 | — |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24 |
3.0; 5.5 | — |
| SECONDARY Absolute Change From Baseline in Lung Clearance Index 5.0 (LCI5.0) Through Week 24 |
0.08; -0.36 | — |
| SECONDARY Absolute Change From Baseline in Sweat Chloride at Week 24 |
3.2; -21.6 | — |
| SECONDARY Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24 |
-1.3; 1.1 | — |
| SECONDARY Relative Change From Baseline in ppFEV1 Through Week 24 |
-0.9; 2.2 | — |
| SECONDARY Absolute Change From Baseline in BMI-for-age Z-score at Week 24 |
0.05; 0.08 | — |
| SECONDARY Absolute Change From Baseline in Weight at Week 24 |
1.7; 2.0 | — |
| SECONDARY Absolute Change From Baseline in Weight-for-age Z-score at Week 24 |
0.02; 0.06 | — |
| SECONDARY Absolute Change From Baseline in Height at Week 24 |
2.6; 2.9 | — |
| SECONDARY Absolute Change From Baseline in Height-for-age Z-score at Week 24 |
0.00; 0.03 | — |
| SECONDARY Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domains Through Week 24 |
0.6; 1.7; 0.4; -1.0; 9.9; 11.9 | — |
| SECONDARY Number of Pulmonary Exacerbation Events |
18; 24 | — |
| SECONDARY Percentage of Participants With At Least 1 Pulmonary Exacerbation Event |
14.9; 19.4 | — |
| SECONDARY Time-to-first Pulmonary Exacerbation |
NA; NA | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
98; 98; 11; 13 | — |
| SECONDARY Average Pre-dose Concentration (Ctrough,Ave) and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Ave) For Lumacaftor and Ivacaftor |
10200; 20600; 107; 821 | — |
Eligibility Criteria
Inclusion Criteria
- Subjects who weigh ≥15 kg without shoes a the Screening Visit
- Subjects with confirmed diagnosis of CF at the Screening Visit.
- Subjects who are homozygous for the F508del CFTR mutation
- Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height
- Subjects with a screening LCI2.5 result greater than or equal to 7.5
Exclusion Criteria
- History of any comorbidity reviewed at the Screening Visit 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.
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
- Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
- History of solid organ or hematological transplantation at the Screening Visit
Data sourced from ClinicalTrials.gov (NCT02514473) 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.