A double blind, randomised, placebo-controlled trial to evaluate the dose-exposure and safety of nintedanib per os on top of standard of care for 24 weeks, followed by open label treatment with nintedanib of variable duration, in children and adolescents (6 to 17 year-old) with clinically significant fibrosing Interstitial Lung Disease.
- Conditions
- Interstitiale longziekten met significante fribrotiseringinterstitial lung diseaselungfibrose
- Registration Number
- NL-OMON49284
- Lead Sponsor
- Boehringer Ingelheim
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 1
-Children and adolescents 6 to 17 years old at Visit 2.
-Signed and dated written informed consent and assent, where applicable, in
accordance with ICH-GCP and local legislation prior to admission to the trial.
-Male or female patients. Female of childbearing potential (WOCBP) must confirm
that sexual abstinence is standard practice and will be continued until 3
months after last drug intake, or be ready and able to use a highly effective
method of birth control per ICH M3 (R2) that results in a low failure rate of
less than 1% per year when used consistently and correctly, in combination with
one barrier method, from 28 days prior to initiation of study treatment, during
treatment and until 3 months after last drug intake. Sexual abstinence is
defined as abstinence from any sexual act that may result in pregnancy.
-Patients with evidence of fibrosing ILD on HRCT within 12 months of Visit 1 as
assessed by the investigator and confirmed by central review.
-Patients with FVC % predicted *25% at Visit 2.
-Patients with clinically significant disease 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).
AST and/or ALT and/or bilirubin >1.5 x ULN, and/or creatinine clearance
<30 mL/min (Schwartz formula), and/or underlying chronic liver disease
(Child Pugh A, B or C hepatic impairment) at Visit 1; previous treatment with
nintedanib, other investigational therapy received within 1 month or 5
half-lives (whichever is shorter but *1 week) prior to Visit 2; significant
pulmonary arterial hypertension, any cardiovascular disease excluded by
protocol, history of thrombotic event within 12 months of Visit 1, other
disease that may interfere with testing procedures or trial participation, or
may put the patient at risk; bleeding risk; life expectancy for any concomitant
disease other than ILD <2.5 years (investigator assessment); any diagnosed
growth disorder or any genetic disorder associated with short stature and/or
treatment with growth hormone therapy within 6 months before Visit 2; <13.5
kg of weight at Visit 1.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary endpoints:<br /><br>* PK: AUC*,ss based on sampling at steady state (at week 2 and week 26);<br /><br>* N (%) of patients with treatment-emergent adverse events at week 24.<br /><br><br /><br>Please see protocol section 2.1</p><br>
- Secondary Outcome Measures
Name Time Method