Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for F508del-CFTR: A Phase 2 Placebo-controlled Clinical Trial.
Mirjam StahlJobst RoehmelMonika EichingerFelix DoellingerLutz NaehrlichMatthias V KoppAnna-Maria DittrichChristopher LeeOlaf SommerburgSimon TianTu XuPan WuAniket JoshiPartha RayMargaret E DuncanMark Oliver WielpützMarcus Alexander MallPublished in: Annals of the American Thoracic Society (2023)
This placebo-controlled study suggests the potential for early disease modification with LUM/IVA treatment, including that assessed by chest MRI, in children as young as 2 years. Clinical trial registered with ClinicalTrials.gov (NCT03625466).
Keyphrases
- cystic fibrosis
- clinical trial
- double blind
- pseudomonas aeruginosa
- placebo controlled
- lung function
- phase ii
- young adults
- study protocol
- phase iii
- magnetic resonance imaging
- open label
- contrast enhanced
- risk assessment
- magnetic resonance
- chronic obstructive pulmonary disease
- human health
- rectal cancer
- diffusion weighted imaging