A Study to Evaluate Long-term Safety of Nintedanib in Children and Adolescents With Interstitial Lung Disease (InPedILD®-ON)

NCT ID: NCT05285982

Last Updated: 2025-10-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 54 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-04-06
• Study Completion Date: 2025-08-13

Brief Summary

This study is open to children and adolescents with interstitial lung disease (ILD) that causes lung fibrosis. This is a study for people who took part in a previous study called InPedILD (study 1199-0337) and for people who are between 6 and 17 years old (in France, between 12 and 17 years old) and have fibrosing ILD.

This study tests a medicine called nintedanib. Nintedanib is already used to treat different types of lung fibrosis in adults. The purpose of the study is to find out how well long-term treatment with nintedanib is tolerated in children and adolescents.

All participants take nintedanib capsules twice a day. Participants coming from the previous study are in this study for at least 3 years or until nintedanib or other treatment options become available outside of this study. New participants are in the study until the overall end of study meaning for at least 1.5 years. Participants visit the study site about 15 times for a study participation of 3 years. Afterwards, they visit the study site every 3 months. The doctors collect information on any health problems of the participants.

Conditions

Lung Diseases, Interstitial

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Patients rolling over from the InPedILD® study

Group Type: EXPERIMENTAL

Nintedanib (Ofev®)

Intervention Type: DRUG

Nintedanib (Ofev®) soft capsules

Patients newly enrolled in this study

Group Type: EXPERIMENTAL

Nintedanib (Ofev®)

Intervention Type: DRUG

Nintedanib (Ofev®) soft capsules

Interventions

Nintedanib (Ofev®)

Nintedanib (Ofev®) soft capsules

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

For new patients:

1. Children and adolescents 6 to 17 years old at Visit 2. In France, only adolescents 12 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 Interstitial Lung Disease (ILD) on High-Resolution Computed Tomography (HRCT) within 12 months of Visit 1 as assessed by the investigator and confirmed by central review.

5. Patients with Forced Vital Capacity (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

• 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).

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 prematurely discontinued treatment permanently in 1199-0337 but are potentially eligible and for completed patients from parent trial not able to roll over into the extension trial within 12 weeks following their End of Treatment Visit in the parent trial:

Exclusion Criteria

For new patients:

1. Aspartate Aminotransferase (AST) and/or Alanine Aminotransferase (ALT) >1.5 x Upper limit of normal (ULN) at Visit 1.

2. Bilirubin >1.5 x ULN at Visit 1.

3. Estimated Glomerular Filtration Rate (eGFR) <30 mL/min/1.73 m² 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:

• Previous clinical or echocardiographic evidence of significant right heart failure
• History of right heart catheterization showing a cardiac index ≤2 l/min/m²
• 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:

• Severe hypertension, uncontrolled under treatment, within 6 months of Visit 1. Uncontrolled hypertension is defined as

• 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). Not applicable in France.
• In adolescents 13 to 17 years old: systolic blood pressure ≥140 mm Hg or diastolic blood pressure ≥90 mm Hg. Not applicable in France.
• Myocardial infarction within 6 months of Visit 1
• Unstable cardiac angina within 6 months of Visit 1

9. Bleeding risk, any of the following:

• Known genetic predisposition to bleeding
• Patients who require

• Fibrinolysis, full-dose therapeutic anticoagulation (e.g. vitamin K antagonists, direct thrombin inhibitors, heparin, hirudin)
• High dose antiplatelet therapy
• History of haemorrhagic central nervous system (CNS) event within 12 months of Visit 1
• Any of the following within 3 months of Visit 1:

• Haemoptysis or haematuria
• Active gastro-intestinal (GI) bleeding or GI - ulcers
• Major injury or surgery (investigator's judgment)
• Any of the following coagulation parameters at Visit 1:

• International normalized ratio (INR) >2
• Prolongation of prothrombin time (PT) by >1.5 x ULN
• 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.

11. Known hypersensitivity to the trial medication or its components (i.e. soya lecithin).

12. Patients with documented allergy to peanut or soya.

13. Other disease that may interfere with testing procedures or in the judgment of the investigator may interfere with trial participation or may put the patient at risk when participating in this trial.

14. Life expectancy for any concomitant disease other than ILD <2.5 years (investigator assessment).

15. Female patients who are pregnant, nursing, or who plan to become pregnant while in the trial.

16. Patients not able or willing to adhere to trial procedures, including intake of study medication.

17. Patients who must or wish to take any drug considered likely to interfere with the safe conduct of the trial according to investigator's benefit-risk assessment for the individual patient

18. Patients with any diagnosed growth disorder such as growth hormone deficiency or any genetic disorder that is associated with short stature (e.g. Turner Syndrome, Noonan Syndrome, Russell-Silver Syndrome) and/or treatment with growth hormone therapy within 6 months before Visit 2. Patients with short stature considered by the investigator to be due to glucocorticoid therapy may be included.

19. Patients <13.5 kg of weight at Visit 1 (same threshold to be used for male and female patients).

For roll-over patients from the InPedILD® study:

Only criteria 11, 12, 13, 15, 16, 17 and 19, listed for new patients are applicable with the following additional exclusion criterion:

For patients who prematurely discontinued treatment permanently in 1199-0337 but are potentially eligible and for completed patients from parent trial not able to roll over into the extension trial within 12 weeks following their End of Treatment Visit in the parent trial:

21. Patients who experienced drug-related adverse events during parent trial leading to permanent study treatment discontinuation.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boehringer Ingelheim

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Weill Cornell Medicine-New York-60569

New York, New York, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Hospital de Pediatria Prof. Dr. Juan P. Garrahan

CABA, , Argentina

Hospital de Niños Dr. Ricardo Gutierrez

CABA, , Argentina

Brussels - UNIV HUDERF

Brussels, , Belgium

Serviços Medicos Respirar Sul Fluminense

Barra Mansa, , Brazil

Centro de Pesquisa Clinica do Instituto da Crianca - HCFMUSP

São Paulo, , Brazil

BC Children's Hospital

Vancouver, British Columbia, Canada

The Hospital for Sick Children

Toronto, Ontario, Canada

Teaching Hospital Motol, Oncology Clinic

Prague, , Czechia

Tampere University Hospital

Tampere, , Finland

HOP Intercommunal

Créteil, , France

Hamburger Zentrum für Kinder- und Jugendrheumatologie

Hamburg, , Germany

General Hospital of Thessaloniki "Ippokrateio"

Thessaloniki, , Greece

Azienda Ospedaliera Meyer

Florence, , Italy

Osp. Pediatrico Bambin Gesù

Roma, , Italy

Clinical Research Institute S.C.

Tlalnepantla, , Mexico

Oslo Universitetssykehus HF, Rikshospitalet

Oslo, , Norway

Independent Public Teaching Children's Hospital

Warsaw, , Poland

ULS de São José, E.P.E. - Hospital Dona Estefânia

Lisbon, , Portugal

ULS de Santa Maria, E.P.E

Lisbon, , Portugal

Hospital Universitari Vall D Hebron

Barcelona, , Spain

Hospital Virgen del Rocío

Seville, , Spain

King's College Hospital

London, , United Kingdom

Countries

United States; Argentina; Belgium; Brazil; Canada; Czechia; Finland; France; Germany; Greece; Italy; Mexico; Norway; Poland; Portugal; Spain; United Kingdom

Related Links

https://www.mystudywindow.com

Related Info

Other Identifiers

2020-005554-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1305-7514

Identifier Type: REGISTRY

Identifier Source: secondary_id

2024-515743-27-00

Identifier Type: CTIS

Identifier Source: secondary_id

1199-0378

Identifier Type: -

Identifier Source: org_study_id