Nintedanib in children and adolescents with Interstitial Lung Disease.
Research type
Research Study
Full title
A double blind, randomised, placebo-controlled trial to evaluate the dose-exposure and safety of oral nintedanib 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.
IRAS ID
270919
Contact name
Steve Cunningham,
Contact email
Sponsor organisation
Boehringer Ingelheim
Eudract number
2018-004530-14
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 5 months, 25 days
Research summary
Research Summary
This is a double blind, randomised, placebo-controlled trial to evaluate the dose-exposure and safety of oral nintedanib 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. This trial will be conducted in approximately 24 countries and will include at least 30 randomised patients of at least 20 adolescents aged 12-17 years. Participants will undergo 24 weeks of double blind, randomised, placebo-controlled treatment on top of Standard of Care (SOC) (Part A), followed by open label active treatment of variable duration (Part B). During Part B patients will continue treatment with nintedanib on top of SOC until the end of the trial (i.e. the date of the last visit of the last patient in the whole trial) or premature discontinuation.
The main purpose of this trial is to find out how nintedanib is taken up in the body and how well it is tolerated in children and adolescents with childhood Interstitial Lung Disease (ILD). In addition the trial will investigate whether treatment with nintedanib has any effect on the participants lung using breathing tests, his/her ability to exercise, and how the participant is feeling during the trial.Summary of Results
A study to find out how nintedanib is taken up in the body and how well it is tolerated in children and adolescents with Interstitial Lung Disease (ILD) This is a summary of results from 1 clinical study.
We thank all study participants. You helped us to answer important questions about nintedanib and the treatment of Interstitial Lung Disease (ILD).What was this study about?
The purpose of this study was to find out how nintedanib is taken up in the body and how well it is tolerated in children and adolescents. Nintedanib is a medicine that is already used to treat different types of lung fibrosis in adults. In this study, nintedanib was given to children and adolescents who have fibrosing Interstitial Lung Disease (ILD).Who took part in this study?
Children and adolescents who were 6 to 17 years old could take part in this study. They could participate if they had fibrosing ILD.
In this study, 39 children and adolescents received study treatment. 24 were girls and 15 were boys. The youngest participant was 6 years old and the oldest 17 years old. The average age was 13.
There were participants in the study in 21 countries. Participants in 16 countries received the study medicine.How was this study done?
This study had 2 parts. Part 1 was planned to last 6 months. In Part 1, participants were divided into 2 groups. The groups were:
• Nintedanib group: participants took nintedanib capsules twice a day.
The dose depended on the weight of the participant.
• Placebo group: participants took placebo capsules twice a day.
Every participant had a 2 in 3 chance of being in the nintedanib group compared with the placebo group. Placebo capsules looked like nintedanib capsules but did not contain any medicine. The participants and doctors did not know whether the participants were in the nintedanib group or in the placebo group. We compared nintedanib with placebo to find out how well nintedanib is tolerated by children and adolescents.
In Part 2 of the study, all participants took nintedanib capsules twice a day. This means that participants taking placebo switched treatment to nintedanib. All participants knew that they were now taking nintedanib.
In both parts of the study, we measured the amount of nintedanib in the blood at different time points to find out how it is taken up in the body of children and adolescents.
The time in the study and duration of treatment was different for each participant, depending on when they joined the study. Participants stayed in the study between about half a year and 2 years.
Participants visited the doctors regularly. During these visits, the doctors collected information about the participants’ health.What were the results of this study?
85% of participants in the nintedanib group and 85% of participants in the placebo group had health problems during the 6 months (Part 1) of the study. This means that the percentage of participants with health problems was the same in both groups.
The amount of nintedanib in the blood was similar in children and adolescents. The doses that the participants took depending on their body weight led to similar levels of nintedanib in the blood as seen in adults. Because the blood levels were similar, researchers think that the effect of nintedanib in children and adolescents may be similar to the effect in adults.Did participants have any unwanted effects?
Yes, participants in both groups had unwanted effects. Unwanted effects are health problems that the doctors think were caused by nintedanib or placebo.
In Part 1 of this study, 14 out of 26 participants (54%) in the nintedanib group had unwanted effects. 5 out of 13 participants (39%) in the placebo group had unwanted effects.
The listing below shows the most common unwanted effects in the nintedanib group that occurred in Part 1. The listing also shows how many participants in the nintedanib and the placebo group had each of these unwanted effects.
Unwanted effects during Part 1 of the study:
Diarrhoea
• Nintedanib (26 participants): 7 participants (27%)
• Placebo (13 participants): 1 participant (8%)
Stomach pain (abdominal pain)
• Nintedanib (26 participants): 5 participants (19%)
• Placebo (13 participants): 0 participants
Vomiting
• Nintedanib (26 participants): 4 participants (15%)
• Placebo (13 participants): 1 participant (8%)
Nausea
• Nintedanib (26 participants): 3 participants (12%)
• Placebo (13 participants): 1 participant (8%)
Pain in the upper stomach (abdominal pain upper)
• Nintedanib (26 participants): 2 participants (8%)
• Placebo (13 participants): 0 participants
Fatigue
• Nintedanib (26 participants): 2 participants (8%)
• Placebo (13 participants): 1 participant (8%)
In Part 2 of the study, participants in the placebo group switched from placebo capsules to nintedanib capsules. This means that in Part 2 all participants took nintedanib.
During the whole study (Parts 1 and 2), 18 out of 26 participants (69%) who took nintedanib from the beginning had unwanted effects. 7 out of 13 participants (54%) who took placebo in the beginning (Part 1) had unwanted effects.
The listing below shows the most common unwanted effects that occurred during the whole study. The listing also shows how many participants had each of these unwanted effects.
Unwanted effects that occurred during the whole study:
Diarrhoea
• Participants who took nintedanib from the beginning (26 participants): 9 participants (35%)
• Participants who took placebo in the beginning (13 participants): 4 participants (31%)
Nausea
• Participants who took nintedanib from the beginning (26 participants): 4 participants (15%)
• Participants who took placebo in the beginning (13 participants): 6 participants (46%)
Stomach pain (abdominal pain)
• Participants who took nintedanib from the beginning (26 participants): 5 participants (19%)
• Participants who took placebo in the beginning (13 participants): 2 participants (15%)
Vomiting
• Participants who took nintedanib from the beginning (26 participants): 4 participants (15%)
• Participants who took placebo in the beginning (13 participants): 3 participant (23%)
Tooth development disorder
• Participants who took nintedanib from the beginning (26 participants): 4 participants (15%)
• Participants who took placebo in the beginning (13 participants): 0 participants
Pain in the upper stomach (abdominal pain upper)
• Participants who took nintedanib from the beginning (26 participants): 3 participants (12%)
• Participants who took placebo in the beginning (13 participants): 0 participants
Serious unwanted effects:
Some unwanted effects in the whole study were serious because the doctor thought they were serious for any other reason. 2 participants (8%) who took nintedanib from the beginning had serious unwanted effects. 1 participant (8%) who switched from placebo to nintedanib had serious unwanted effects. The serious unwanted effects were liver injury and tooth development disorder.Is there more information about this study?
The following listing shows the regions and countries with the number of participants who received study medicine in brackets:
• Europe (19): Belgium (2), Czech Republic (2), France (2), Greece (1), Italy (2), Poland (4), Portugal (2), Russia (1), Spain (1), Ukraine (1), United Kingdom (1)
• North America (13): United States of America (12), Canada (1)
• Rest of the World (7): Argentina (2), Brazil (3), Mexico (2)
The total number of participants in the European Union was 16.You can find further information about this study at these websites:
1. Go to https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrialsregister.eu%2Fctr-search&data=05%7C01%7Capprovals%40hra.nhs.uk%7C275f2ffe11a84e85c30e08db6e78e510%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638225237737615398%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=e%2B9QqscbG0a%2BB4aDsd%2FHPFmRjrN71a8x98Tjhe50ng8%3D&reserved=0 and search for the EudraCT number 2018-004530-14.
2. Go to https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C01%7Capprovals%40hra.nhs.uk%7C275f2ffe11a84e85c30e08db6e78e510%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638225237737615398%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=P7S%2FcEuGPnaUgfFzhARBnhJNOZdemi0BVDPo4%2BknHp8%3D&reserved=0 and search for the NCT number NCT04093024.
Boehringer Ingelheim sponsored this study. For contact details, please visit https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.boehringer-ingelheim.com%2Fcontact-us&data=05%7C01%7Capprovals%40hra.nhs.uk%7C275f2ffe11a84e85c30e08db6e78e510%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638225237737615398%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=BpzhXUNBqaILIG8EOLQQle85nbt90ZgHS203qHm%2FYBg%3D&reserved=0.
InPedILD®: 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 This was a Phase 3 study. This study started in February 2020 and finished in May 2022.Are there additional studies?
There is an additional study with nintedanib for children and adolescents with fibrosing ILD (study number 1199-0378). You can find more information about this study on the websites listed above.Important notice
This lay summary is provided as part of Boehringer Ingelheim’s commitment to publicly share clinical study results.
This summary shows only the results from one study and may not represent all of the knowledge about the medicine studied. Other studies may have different results. Usually, more than one study is carried out to find out how well a medicine works and to determine the side effects of a medicine.
This lay summary may include uses, formulations, or treatment regimens for the medicine studied that may be approved or not approved in your country. This lay summary is not intended to promote any product or indication, to guide treatment decisions, or to replace the advice of a healthcare professional.
You should not change your therapy based on the results of this study. Always consult with your treating physician about your therapy.
©2022 Boehringer Ingelheim International GmbHREC name
West of Scotland REC 1
REC reference
20/WS/0082
Date of REC Opinion
9 Jun 2020
REC opinion
Favourable Opinion