A study to find out how nerandomilast is tolerated, handled by the body, and if it helps children and adolescents with interstitial lung disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Boehringer Ingelheim International GmbH, Boehringer Ingelheim Espana S.A.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Decision date (initial)

2026-07-07

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2025-523369-32-00

WHO UTN

U1111-1326-6351

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Pharmacokinetic, Efficacy

The main objective of the trial is to evaluate the dose-exposure and safety of nerandomilast in children and adolescents with fibrosing ILD.

Secondary objectives 1

1. The secondary objectives of the trial are to evaluate the safety and exploratory efficacy of nerandomilast in children and adolescents with fibrosing ILD.

Conditions and MedDRA coding

Fibrosing interstitial lung disease

Regulatory references

Scientific advice from competent authorities

Center For Drug Evaluation, Food And Drug Administration, European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002901-PIP01-20

Plan to share IPD

Yes

IPD plan description

"Researchers can request access to the documents regarding this study using the link https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing, and upon a signed “Document Sharing Agreement”. Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined on the website. Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program. Access Criteria: For study documents – upon signing of a ‚Document Sharing Agreement‘. For study data – 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement."

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1. Children and adolescents 2 to <18 years old at Visit 2.
2. 2. Participants with evidence of fibrosing ILD on HRCT within 12 months of Visit 1 as assessed by the investigator and confirmed by central review.
3. 3. For children ≥6 years: Participants with FVC % predicted ≥25% at Visit 2.
4. "4. Participants with clinically significant fibrosing ILD 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 (e.g. increased oxygen requirement, decreased diffusion capacity)."
5. Further inclusion criteria apply.

Exclusion criteria 9

1. 1. Previous treatment with nerandomilast.
2. 2. Participants treated with other oral/systemic PDE4 and non-selective PDE inhibitors within 30 days before Visit 1.
3. 3. Participants treated with pirfenidone in the 8 weeks prior to Visit 1.
4. 4. Unstable pulmonary arterial hypertension (PAH).
5. 5. Active vasculitis, unstable or uncontrolled within 8 weeks prior to Visit 1 or during the screening period.
6. 6. Any suicidal behaviour (i.e. actual attempt, interrupted attempt, aborted attempt, or preparatory acts or behaviour) in the past (lifetime).
7. "7. Any suicidal ideation of type 4 or 5 on the C-SSRS in the past 3 months at Visit 1 or at Visit 2 (i.e. active suicidal thought with method and intent but without specific plan; or active suicidal thought with method, intent, and plan)."
8. 8. Participants with clinically significant depression symptoms defined as the short version of mood and feeling questionnaire (SMFQ) score ≥8.
9. Further exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. "AUCT,SS based on sampling at steady state using rich sampling (at Week 2 in Part A and at Week 28 in Part B) in participants from 6 years to less than 18 years and sparse sampling (at Week 2 of Part A) in participants younger than 6 years "
2. Occurrence of a treatment-emergent adverse event (Yes/No) up to Week 26

Secondary endpoints 11

1. Absolute change from baseline in oxygen saturation (SpO2) [%] on room air at rest at Week 26 and Week 52
2. Absolute change from baseline in height [cm] at Week 26 and Week 52
3. Absolute change from baseline in PedsQL™ at Week 26 and Week 52
4. Occurrence of a treatment-emergent adverse event (Yes/No) over the whole trial
5. Time to first respiratory-related hospitalisation [days] over the whole trial
6. Time to first acute ILD exacerbation or death [days] over the whole trial
7. Time to death [days] over the whole trial
8. Acceptability based on number/size of tablets at Week 2 and Week 26
9. Acceptability based on the use of the dispenser at Week 2 and Week 26
10. Absolute change from baseline in FVC [% predicted] at Week 26 and Week 52 (applicable to participants ≥6 years)
11. Absolute change from baseline in 6-min walk distance [m] at Week 26 and Week 52 (applicable to participants ≥6 years)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Boehringer Ingelheim International GmbH

Sponsor organisation

Boehringer Ingelheim International GmbH

Address

Binger Strasse 173

City

Ingelheim Am Rhein

Postcode

55216

Country

Germany

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Third parties 1

Boehringer Ingelheim Espana S.A.

Sponsor organisation

Boehringer Ingelheim Espana S.A.

Address

Carrer Prat De La Riba 50, Sant Cugat Del Valles Sant Cugat Del Valles

City

Barcelona

Postcode

08174

Country

Spain

Scientific contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim International GmbH

Contact name

CT Disclosure & Data Transparency

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

5 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 266 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications