A study to evaluate long-term safety of nintedanib in children and adolescents with interstitial lung disease (InPedILD™-ON)

Phase 1

Conditions: Interstitial Lung Disease
MedDRA version: 21.1Level: LLTClassification code 10066393Term: Respiratory bronchiolitis-associated interstitial lung diseaseSystem Organ Class: 100000004855
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: EUCTR2020-005554-23-IT

Lead Sponsor: BOEHRINGER-INGELHEIM ITALIA S.P.A.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 60

Inclusion Criteria

For new patients:
1. Children and adolescents 6 to 17 years old at Visit 2.
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.
3. Male or female patients. Female of childbearing potential (WOCBP1)
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.
4. Patients with evidence of fibrosing ILD on HRCT within 12 months of
Visit 1 as assessed by the investigator and confirmed by central review.
5. Patients with FVC % predicted =25% at Visit 2.
6. 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
o a 5-10% relative decline in FVC% predicted accompanied by
worsening symptoms, or
o a =10% relative decline in FVC % predicted, or
o increased fibrosis on HRCT, or
o other measures of clinical worsening attributed to progressive lung
disease
For roll-over patients from the InPedILD™ study:
Only criteria 2 and 3 listed for new patients are applicable with the
following additional inclusion criterion:
7. Patients who completed the InPedILD™ trial as planned and who did
not permanently prematurely discontinue study treatment.
For patients who discontinued treatment permanently in 1199-0337 but
are potentially eligible:
Criteria for new patients are applicable except criteria 4, and 6
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

For new patients:
1. AST and/or ALT >1.5 x ULN at Visit 1.
2. Bilirubin >1.5 x ULN at Visit 1.
3. eGFR <30 mL/min calculated by Schwartz formula at Visit 1
4. Patients with underlying chronic liver disease (Child Pugh A, B or C
hepatic impairment) at Visit 1.
5. Other investigational therapy received within 1 month or 5 half-lives
(whichever is shorter but =1 week) prior to Visit 2 except investigational
therapy received in InPedILD™ trial.
6. Significant pulmonary arterial hypertension (PAH) defined by any of
the following:
a. Previous clinical or echocardiographic evidence of significant right
heart failure
b. History of right heart catheterization showing a cardiac index =2
l/min/m²
c. PAH requiring parenteral therapy with epoprostenol/treprostinil
7. In the opinion of the Investigator, other clinically significant
pulmonary abnormalities.
8. Cardiovascular diseases, any of the following:
a. Severe hypertension, uncontrolled under treatment, within 6 months
of Visit 1.
Uncontrolled hypertension is defined as
i. In children 6 to =12 years old: =95th percentile + 12 mm Hg or =
140/90 mm Hg
(whichever is lower) (systolic or diastolic blood pressure equal to or
greater than
the calculated target value) (please refer to Appendix 10.5)
ii. In adolescents 13 to 17 years old: systolic blood pressure =140 mm
Hg or diastolic blood pressure =90 mm Hg (please refer to Appendix 10.5)
b. Myocardial infarction within 6 months of Visit 1
c. Unstable cardiac angina within 6 months of Visit 1
9. Bleeding risk, any of the following:
a. Known genetic predisposition to bleeding
b. Patients who require
i. Fibrinolysis, full-dose therapeutic anticoagulation (e.g. vitamin K
antagonists, direct thrombin inhibitors, heparin, hirudin)
ii. High dose antiplatelet therapy
c. History of haemorrhagic central nervous system (CNS) event within 12
months of Visit 1
d. Any of the following within 3 months of Visit 1:
i. Haemoptysis or haematuria
ii. Active gastro-intestinal (GI) bleeding or GI – ulcers
iii. Major injury or surgery (investigator's judgment)
e. Any of the following coagulation parameters at Visit 1:
i. International normalized ratio (INR) >2
ii. Prolongation of prothrombin time (PT) by >1.5 x ULN
iii. Prolongation of activated partial thromboplastin time (aPTT) by >1.5
x ULN
10. History of thrombotic event (including stroke and transient ischemic
attack) within 12 months of Visit 1.
For other criteria, see protocol

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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