A study to evaluate the dose-exposure, safety, and exploratory efficacy of nerandomilast in children and adolescents from 2 years to less than 18 years of age with fibrosing interstitial lung disease (Part A: double-blind, placebo controlled in children from 6 to less than 18 years of age and open label active treatment in children from 2 to less than 6 years of age), followed by an open-label phase with active treatment (Part B)

未提供0 个研究点目标入组 35 人开始时间: 待定

适应症

试验速览

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•Children and adolescents 2 to <18 years old at Visit
•Participants with evidence of fibrosing ILD on high-resolution computed tomography (HRCT) within 12 months of Visit 1 as assessed by the investigator and confirmed by central review.
•For children >=6 years: Participants with forced vital capacity (FVC) % predicted >=25% at Visit
•Participants with clinically significant fibrosing ILD at Visit 2, as assessed by the investigator based on any of the following:
•Fan score >=3, or
•Documented evidence of clinical progression over time based on either
•a 5-10% relative decline in FVC % predicted accompanied by worsening symptoms, or
•a >=10% relative decline in FVC % predicted, or
•increased fibrosis on HRCT, or
•other measures of clinical worsening attributed to progressive lung disease (e.g. increased oxygen requirement, decreased diffusion capacity).
•Further inclusion criteria apply.

•Previous treatment with nerandomilast.
•Participants treated with other oral/systemic PDE4 and non-selective PDE inhibitors within 30 days before Visit
•Participants treated with pirfenidone in the 8 weeks prior to Visit
•Unstable pulmonary arterial hypertension (PAH).
•Active vasculitis, unstable or uncontrolled within 8 weeks prior to Visit 1 or during the screening period.
•Any suicidal behaviour (i.e. actual attempt, interrupted attempt, aborted attempt, or preparatory acts or behaviour) in the past (lifetime).
•Any suicidal ideation of type 4 or 5 on the columbia suicidal severity rating scale (C-SSRS) in the past 3 months at Visit 1 or at Visit 2 (i.e. active suicidal thought with method and intent but without specific plan; or active suicidal thought with method, intent, and plan).
•Participants with clinically significant depression symptoms defined as the short version of mood and feeling questionnaire (SMFQ) score >=
•Further exclusion criteria apply.

发起方

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