A study to test how well BI 3720931 is tolerated and whether it improves lung function in people with cystic fibrosis (LenticlairTM 1)

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

19 Dec 2024 → 3 Feb 2026

Decision date (initial)

2024-06-24

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2023-503281-23-00

WHO UTN

U1111-1291-0800

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety, Others, Pharmacokinetic, Pharmacodynamic

"Phase I: To investigate safety and tolerability of a single inhaled dose of BI 3720931 based on number of trial participants with at least one drug-related, treatment emergent AE up to Week 24 after dosing.
Phase II: To demonstrate superiority of the higher BI 3720931 dose over PBO on the primary endpoint, the absolute change from baseline in FEV1pp at Week 8. If superiority for the high dose is established, superiority of the lower dose versus PBO will be tested in a hierarchical manner on the primary endpoint. "

Secondary objectives 5

1. Phase I: Evaluate clinical efficacy after a single inhaled dose of BI 3720931 based on the number of responders, as part of an interim analysis prior to moving to Phase II. Response is defined as an absolute change from baseline ≥5% in FEV1pp using the mean of the values at Weeks 4, 6, and 8 after dosing
2. Phase I: Evaluate clinical efficacy after a single inhaled dose of BI 3720931 based on absolute change from baseline in FEV1pp at Week 24
3. Phase I: Evaluate safety after a single inhaled dose of BI 3720931 based on occurrence of dose limiting toxicities (DLTs) up to Week 24
4. Phase II: Investigate safety and tolerability of the BI 3720931 doses based on occurrence of drug related AEs and serious adverse events (SAEs) up to Week 24
5. Phase II: Evaluate efficacy of the BI 3720931 doses versus PBO on the absolute change from baseline in FEV1pp at Week 24

Conditions and MedDRA coding

Cystic fibrosis

Study design 10 periods

Regulatory references

Scientific advice from competent authorities

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

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 7

1. Male or female of non-childbearing potential trial participants with a CF-pulmonary phenotype and a confirmed diagnosis of CF: - Positive sweat chloride ≥60 mEq/L by pilocarpine iontophoresis OR - Genotype with 2 identifiable CF-causing mutations accompanied by one or more clinical features if sweat chloride testing is between 30 and 59 mmol/L
2. Trial participants who are not eligible for treatment with CFTRmt due to their genotype with 2 identified CFTR-mutations (including Class I CFTR gene mutations) and are also not expected to become eligible during the trial according to investigator´s opinion
3. Trial participants able to perform acceptable spirometric maneuvers according to American Thoracic Society/European Respiratory Society 2019 standards
4. FEV1pp ≥50% and ≤100% of predicted normal at Visit 1. Predicted value based on Global Lung Initiative lung function reference equations
5. Stable CF disease with no pulmonary exacerbation 4 weeks prior to the screening visit and during the screening period and stable drug- and non-drug therapy for CF in the 4 weeks prior to dosing
6. Trial participants either naïve to prior gene therapy or exposed to prior viral or non-viral gene therapies for cystic fibrosis. If prior exposure to gene therapy for CF exists, then the following applies as per investigator judgement: a. No apparent residual side effects associated with prior gene therapy as per investigator assessment, and b. Drug-free interval of -- 6 months after last dose of prior non-viral gene therapy -- 24 months after last dose of prior viral gene therapy
7. Further inclusion criteria apply.

Exclusion criteria 3

1. Trial participants with ongoing or planned CFTRmt, or participants not eligible for CFTRmt based on contraindications (e.g. liver failure) or who needed to withdraw CFTRmt due to intolerability are not appropriate candidates for this Phase I/II trial
2. Trial participants requiring chronic use of systemic corticosteroids or immunosuppressants to treat another condition
3. Further exclusion criteria apply.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Phase I: Occurrence of any drug-related, treatment-emergent AE up to Week 24 after drug administration
2. Phase II: Absolute change from baseline in FEV1pp at Week 8 after drug administration

Secondary endpoints 6

1. Phase I: Occurrence of treatment response defined as change from baseline ≥5% in FEV1pp, comparing the mean of 3 pre-treatment FEV1pp measured in the screening period with the mean of 3 post treatment FEV1pp values at Weeks 4, 6, and 8
2. Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration
3. Phase I: Occurrence of any DLTs up to Week 24 after drug administration
4. Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration
5. Phase II: Occurrence of any SAEs up to Week 24 after drug administration
6. Phase II: Occurrence of any drug-related, treatment-emergent AEs up to Week 24 after drug administration

Investigational products

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

Test 2

Placebo 1

Auxiliary 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

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 Espana S.A.

Contact name

CT Disclosure & Data Transparency

Public contact point

Organisation

Boehringer Ingelheim Espana S.A.

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

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 4 · Art. 38 CTR

Corrective measures 1 · Art. 77 CTR

Results and documents

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

Documents 57 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