This study is open to children and adolescents aged 2 to 17 years with interstitial lung disease (ILD). Nerandomilast has just been approved in some countries to help adults with a lung condition called idiopathic pulmonary fibrosis. The purpose of this study is to understand how nerandomilast is tolerated and handled by the body and whether nerandomilast also helps children and adolescents with ILD. For participants aged 6 to 17 years when joining, the study has 2 parts. In the first part, participants are put into 1 of 2 groups randomly, which means by chance. One group gets nerandomilast and the other group placebo. Placebo looks like nerandomilast but does not contain any medicine. Participants are twice as likely to be in the nerandomilast group. They take tablets twice a day for 6 months. After these 6 months, in the second part of this study, they get nerandomilast for at least 2 years regardless of what they got in the first part. Young participants aged 2 to 5 years when joining get nerandomilast from the start. They receive tablets twice a day for at least 2 and a half years. Depending on when a person joins, the study lasts between 2 and a half years and up to 5 years. During this time, participants may visit the study site about 18 to 30 times. Study doctors collect blood samples to check participants' health and to find out how their body handles the study medicine. Doctors also check the function of the lungs, body growth, and how participants feel. The study doctors also regularly check participants' health and take note of any changes. For participants aged 6 to 17 years, the results are compared between the groups to see whether nerandomilast treatment helps children and adolescents.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
35
Nerandomilast
Placebo
Children's Hospital Los Angeles
Los Angeles, California, United States
NOT_YET_RECRUITINGChildren's Hospital Colorado
Aurora, Colorado, United States
NOT_YET_RECRUITINGJohns Hopkins Hospital
Baltimore, Maryland, United States
NOT_YET_RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
NOT_YET_RECRUITINGArea under the concentration curve (AUC) T,SS based on sampling at steady state using rich sampling in participants from 6 years to less than 18 years and sparse sampling in participants younger than 6 years
Time frame: At week 2 in Part A and Week 28 in Part B
Occurrence of a treatment-emergent adverse event
Time frame: up to Week 26
Absolute change from baseline in oxygen saturation (SpO2) [%] on room air at rest
Time frame: at Week 26 and Week 52
Absolute change from baseline in height [cm]
Time frame: at Week 26 and Week 52
Absolute change from baseline in pediatric quality of life inventory (PedsQL™)
Health-related quality of life will be assessed in children using the PedsQL™ questionnaire. For younger children who are unable to perform self-assessment, a parent proxy may be required. The score ranges from 0-100, a higher score indicates a better health related quality of life.
Time frame: at Week 26 and Week 52
Occurrence of a treatment-emergent adverse event (Yes/No) over the whole trial
Time frame: up to 5 years
Time to first respiratory-related hospitalisation [days] over the whole trial
Time frame: up to 5 years
Time to first acute interstitial lung disease (ILD) exacerbation or death [days] over the whole trial
Time frame: up to 5 years
Time to death [days] over the whole trial
Time frame: up to 5 years
Acceptability based on number/size of tablets
Acceptability is defined as the overall ability and willingness of the participant to use the medicinal product as intended. Assessment of acceptability will be performed by the participant using the acceptability questionnaire.
Time frame: at Week 2 and Week 26
Acceptability based on the use of the dispenser
Time frame: at Week 2 and Week 26
Absolute change from baseline in FVC [% predicted] (applicable to participants ≥6 years)
Time frame: at Week 26 and Week 52
Absolute change from baseline in 6-min walk distance [m] (applicable to participants ≥6 years)
Time frame: at Week 26 and Week 52
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M Health Fairview Masonic Children's Hospital
Minneapolis, Minnesota, United States
NOT_YET_RECRUITINGNationwide Children's Hospital
Columbus, Ohio, United States
NOT_YET_RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
NOT_YET_RECRUITINGFundacion Respirar
CABA, Argentina
RECRUITINGWomen's and Children's Hospital
North Adelaide, South Australia, Australia
NOT_YET_RECRUITINGBrussels - UNIV HUDERF
Brussels, Belgium
NOT_YET_RECRUITING...and 37 more locations