Phase 3
Completed N=563
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Source: ClinicalTrials.gov NCT01807949 ↗
Enrolled (actual)
563
Serious AEs
24.9%
Results posted
Sep 2015
Primary outcomePrimary: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 — -0.15; 2.46; 2.85 percent predicted of FEV1 — p=0.0004
Summary
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 |
-0.15; 2.46; 2.85 | 0.0004 sig |
| SECONDARY Relative Change From Baseline in Percent Predicted FEV1 at Week 24 |
0.00; 4.42; 5.25 | 0.0007 sig |
| SECONDARY Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 |
0.07; 0.48; 0.43 | <0.0001 sig |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24 |
2.81; 5.02; 5.66 | 0.1651 |
| SECONDARY Percentage of Participants With Response Based on Percent Predicted FEV1 |
22.5; 45.9; 41.2 | <0.0001 sig |
| SECONDARY Number of Pulmonary Exacerbation Events |
1.18; 0.82; 0.67 | 0.0116 sig |
| SECONDARY Absolute Change From Baseline in Weight at Week 24 |
0.44; 1.57; 1.38 | <0.0001 sig |
| SECONDARY Absolute Change From Baseline in BMI-for-age Z-score at Week 24 |
-0.0674; 0.1640; 0.1544 | 0.0005 sig |
| SECONDARY Time-to-First Pulmonary Exacerbation |
NA; NA; NA | 0.0384 sig |
| SECONDARY Percentage of Participants With At Least 1 Pulmonary Exacerbation Event |
47.1; 36.8; 28.9 | 0.0393 sig |
| SECONDARY Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24 |
0.0117; 0.0090; 0.0108 | 0.7679 |
| SECONDARY Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24 |
3.3; 5.7; 6.6 | 0.1034 |
| SECONDARY Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Scores at Week 24 |
-8.49; 0.15; 3.12; 2.03; -1.14; -2.26 | 0.0005 sig |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) |
181; 181; 177; 57; 51; 31 | — |
| SECONDARY Pre-dose Concentration (Ctrough), Average Pre-dose Concentration (Ctrough,Avg), 3 to 6 Hours Post-dose Concentration (C3-6h), and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Avg) |
30.8; 20.2; 30.4; 23.9; 8.11; 13.2 | — |
Eligibility Criteria
Inclusion Criteria
- Confirmed diagnosis of CF
- Homozygous for the F508del CFTR mutation
- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) and less than or equal to (=<) 90% of predicted normal for age, sex, and height
- Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit
Exclusion Criteria
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose of study drug
- History of solid organ or hematological transplantation
- History of alcohol or drug abuse in the past year
- Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
- Use of strong inhibitors, moderate inducers, or strong inducers of Cytochrome P450 3A (CYP3A) within 14 days before Day 1 of dosing
Data sourced from ClinicalTrials.gov (NCT01807949). 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.