Monthly administration of liposome-delivered gene therapy for cystic fibrosis (CF) improved forced expiratory volume (FEV) in a randomized, double-blinded, placebo-controlled phase 2b clinical trial, according to a study published in Lancet Respiratory Medicine.
Patients received the gene–liposome complex or saline placebo by nebulizer every 28 days for a year at 18 facilities in the United Kingdom. They continued standard treatments except for DNase for 24 hours before and after treatment. Subgroups also received the treatment or placebo in a nasal spray or underwent bronchoscopy before the first dose and after the final dose.
At the 12-month follow-up, investigators found “a significant, albeit modest, treatment effect” among the 62 treated patients vs the 54 patients in the placebo group (3.7%; 95% confidence interval [CI], 0.1 – 7.3; P = .046).
However, treatment effect among patients with more severe disease was 6.4% (95% CI, 0.8 – 12.1), and among those with less severe disease, it was 0.2% (95% CI, ?4.6 to 4.9), suggesting a larger benefit among more severely affected individuals.
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