Phase 2
Completed N=51
A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
Source: ClinicalTrials.gov NCT03625466 ↗Enrolled (actual)
51
Serious AEs
21.0%
Results posted
Dec 2021
Primary outcomePrimary: Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48 — -0.3; -1.7 score on a scale
Summary
This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48 |
-0.3; -1.7 | — |
| SECONDARY Part 1: Absolute Change in Lung Clearance Index2.5 (LCI2.5) Through Week 48 |
0.32; -0.37 | — |
| SECONDARY Part 1: Absolute Change in Weight-for-age Z-score at Week 48 |
-0.07; 0.13 | — |
| SECONDARY Part 1: Absolute Change in Stature-for-age Z-score at Week 48 |
0.10; 0.09 | — |
| SECONDARY Part 1: Absolute Change in Body Mass Index (BMI)-For-age Z-score at Week 48 |
-0.24; 0.20 | — |
Eligibility Criteria
Key Inclusion Criteria
- Subjects with confirmed diagnosis of CF.
- Homozygous for F508del (F/F).
- Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit.
Key Exclusion Criteria
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
- Solid organ or hematological transplantation.
- History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study.
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03625466). 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.