A long-term study to monitor the health status of people with cystic fibrosis who took part in a previous study with BI 3720931 (LenticlairTM-ON)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

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

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

3 Jul 2025 → ongoing

Decision date (initial)

2024-06-17

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2023-504909-37-00

WHO UTN

U1111-1291-2932

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

The primary objective is to investigate long-term safety in trial participants treated with at least one dose of BI 3720931.

Secondary objectives 1

1. The secondary objective of the trial is to investigate the duration of clinical efficacy in trial participants after initial dosing of BI 3720931 over at least 2 years.

Conditions and MedDRA coding

Cystic fibrosis

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Medicines And Healthcare Products Regulatory Agency, Paul-Ehrlich-Institut, Medicines Evaluation Board

Plan to share IPD

Yes

IPD plan description

Researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, 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 2

1. All trial participants who participated in a previous BI sponsored trial with BI 3720931 and have received at least one dose of BI 3720931, or matching placebo, are eligible for participation in extension trial 1504-0003 and should be enrolled.
2. Signed and dated written informed consent in accordance with International Council for Harmonisation (ICH)-Good Clinical Practice (GCP) and local legislation prior to admission to the trial.

Exclusion criteria 1

1. There are no exclusion criteria for enrolment into the trial.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Occurrence of treatment-emergent delayed AEs defined as new malignancies, new serious neurologic disorders, new serious rheumatologic or autoimmune disorders, new serious hematologic disorders, or new serious potentially product-related infections, until 15 years from enrolment

Secondary endpoints 5

1. Time to loss of efficacy defined as the drop to below 5% (absolute) above baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp)
2. Time to first PEX from dosing
3. Occurrence of AEs up to 2 years after enrolment
4. Occurrence of serious adverse events (SAEs) throughout the study (up to 15 years)
5. Occurrence of replication-competent lentivirus (RCL) throughout the study (up to 15 years)

Investigational products

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

Test 2

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

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

Sponsor responsibilities

Article 77 compliance

Boehringer Ingelheim International GmbH

Contact point sponsor

Boehringer Ingelheim International GmbH

Article 77 implementation

Boehringer Ingelheim International GmbH

Locations

4 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 48 files

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

Application history

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