Methods
Inclusion and exclusion criteria
Key inclusion criteria
- Aged 6–17 years
- Evidence of fibrosing ILD
- FVC ≥25% predicted
- Clinically significant disease:
- Fan score ≥3, or
- Evidence of clinical progression over time:
- 5–10% relative decline in FVC % predicted + worsening symptoms
- ≥10% relative decline in FVC % predicted
- Increased fibrosis on HRCT
- Other measures, e.g. increased oxygen requirement, decreased diffusion capacity
Key exclusion criteria
- Underlying chronic liver disease
- Significant PAH
- Cardiovascular disease (defined by protocol)
- Bleeding risk
Evidence of fibrosing ILD, as confirmed by central review
aCo-existing cystic abnormalities or ground-glass opacity are acceptable; however, co-existing multifocal, non-fibrotic, non-dependent consolidations (e.g. organizing pneumonia, infection) will not be permitted.
bWith or without ground-glass opacification
HRCT, high-resolution computed tomography; ILD, interstitial lung disease; NSIP, nonspecific interstitial pneumonia; UIP, usual interstitial pneumonia.