Phase 3
Completed N=46
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
Cystic fibrosis · Advanced Lung Disease
Source: ClinicalTrials.gov NCT02390219 ↗
Enrolled (actual)
46
Serious AEs
39.1%
Results posted
Nov 2017
Primary outcomePrimary: Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) — 43; 18 Participants
◆ Published Evidence
Established
69citations · ~9 / year
Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR.
Summary
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
Linked Publications
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Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) |
43; 18 | — |
| SECONDARY Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24 |
-0.4 | — |
| SECONDARY Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24 |
-0.02 | — |
| SECONDARY Duration For Which Participants Received Intravenous (IV) Antibiotics |
11.38 | — |
| SECONDARY Number of Hospitalizations |
23 | — |
| SECONDARY Absolute Change From Baseline in Sweat Chloride at Average of Day 15 and Week 4 |
-16.4 | — |
| SECONDARY Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score Through Week 24 |
2.5 | — |
Eligibility Criteria
Inclusion Criteria
- Homozygous for the F508del-CFTR mutation; historical genotype must be documented in the participant's source documents.
- Percent predicted FEV1 450 msec at Screening
- 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 CYP3A
- Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at Screening and Day 1.
- Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
- Use of beta blockers or the equivalent at Screening.
Data sourced from ClinicalTrials.gov (NCT02390219) 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.