A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1

NCT ID: NCT04059094

Last Updated: 2021-06-04

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 52 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-16
• Study Completion Date: 2020-04-24

Brief Summary

The primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic fibrosis.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Placebo

2 puffs ofmatching placebowere inhaledorally via theRespimat®inhaler twice dailyfor a treatmentperiod of 4 weeksin patients withcystic fibrosis.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Inhalation solution

BI 1265162 50 μg b.i.d.

2 puffs of 25micrograms (μg)BI 1265162(Total: 50μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:100μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis.

Group Type: EXPERIMENTAL

BI 1265162

Intervention Type: DRUG

Inhalation solution

BI 1265162 100 μg b.i.d.

2 puffs of 50micrograms (μg)BI 1265162(Total: 100μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:200μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis.

Group Type: EXPERIMENTAL

BI 1265162

Intervention Type: DRUG

Inhalation solution

BI 1265162 200 μg b.i.d.

2 puffs of 100micrograms (μg)BI 1265162(Total: 200μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:400μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis.

Group Type: EXPERIMENTAL

BI 1265162

Intervention Type: DRUG

Inhalation solution

BI 1265162 20 μg b.i.d.

2 puffs of 10micrograms (μg)BI 1265162(Total: 20μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:40μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis.

Group Type: EXPERIMENTAL

BI 1265162

Intervention Type: DRUG

Inhalation solution

Interventions

BI 1265162

Inhalation solution

Intervention Type: DRUG

Placebo

Inhalation solution

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male or female patients, 12 years of age or older at screening;
• Documented diagnosis of cystic fibrosis including:

• positive sweat chloride ≥ 60 mEq/L, by pilocarpine iontophoresis OR
• genotype with 2 identifiable mutations consistent with cystic fibrosis accompanied by one or more clinical features with cystic fibrosis phenotype;
• Patients able to perform acceptable spirometric manoeuvres according to American Thoracic Society (ATS) standards;
• FEV1 ≥ 40% and ≤ 90% of predicted values at screening and predose at Visit 2;
• Women of childbearing potential (WOCBP) must be willing and able to use highly effective methods of birth control per ICH M3 (R2) that result in a failure rate of less than1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the patient (or patient's legal guardian) information;
• Signed and dated written informed consent and assent in accordance with ICH Harmonized Guideline for Good Clinical Practice (GCP) and local legislation prior to admission in the trial.

Exclusion Criteria

• Evidence of acute upper or lower respiratory tract infection within 4 weeks prior to randomization based on investigator's judgement;
• Pulmonary exacerbation requiring use of i.v./oral/inhaled antibiotics or oral corticosteroids within 4 weeks prior to randomisation;
• Patients with history of Acute Tubular Necrosis (ATN);
• Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal cell carcinoma of the skin or in situ carcinoma of uterine cervix;
• Patients unable to inhale trial drug in an appropriate manner from the Respimat® inhaler based on investigator's judgement;
• Patients who have started a new chronic medication for CF within 4 weeks of randomisation;
• Patients who have previously received a lung transplant or patients who are currently on a waiting list to receive a lung transplant;
• Patients with a significant history of allergy/hypersensitivity (including medication allergy) which is deemed relevant to the trial as judged by the investigator or with a known hypersensitivity to trial drug or its components. "Significance" in this context refers to any increased risk of hypersensitivity reaction to trial medication;
• Any clinically significant laboratory abnormalities at screening as judged by the investigator, or any of the following:

• Potassium > upper limit of normal (ULN) in non-haemolysed blood
• Abnormal renal function defined as estimated Glomerular Filtration Rate (eGFR) < 60ml/min/1.73m²
• Abnormal liver function, defined by serum level of either alanine transaminase (ALT), aspartate transaminase (AST) or total bilirubine ≥ 3 x upper limit of normal (ULN)
• Clinically significant disease or medical condition other than CF or CF-related conditions that, in the opinion of the investigator, would compromise the safety of the patient or the data quality. This includes significant haematological, hepatic, renal, cardiovascular and neurologic disease. Patients with diabetes may participate if their disease is under good control prior to screening;
• Patients not expected to comply with the protocol requirements or not expected to complete the trial as scheduled;
• Previous randomisation in this trial;
• Currently enrolled in another investigational device or drug trial, or less than 30 days or six half-lives (whichever is greater) since ending another investigational device or drug trial(s), or receiving other investigational treatment(s);
• Chronic alcohol or drug abuse or any condition that, in the investigator's opinion, makes them an unreliable trial patient or unlikely to complete the trial;
• Women who are pregnant, nursing, or who plan to become pregnant while in the trial.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boehringer Ingelheim

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Nemours Children's Hospital

Orlando, Florida, United States

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Virginia Commonwealth University Health Systems

Richmond, Virginia, United States

University of Washington

Seattle, Washington, United States

Brussels - UNIV UZ Brussel

Brussels, , Belgium

UZ Leuven

Leuven, , Belgium

St. Paul's Hospital

Vancouver, British Columbia, Canada

Centre Hospitalier de l'Universite de Montreal (CHUM)

Montreal, Quebec, Canada

HOP Arnaud de Villeneuve

Montpellier, , France

HOP Robert Debré

Paris, , France

HOP Cochin

Paris, , France

HOP Lyon Sud

Pierre-Bénite, , France

HOP Perharidy

Roscoff, , France

Charité - Universitätsmedizin Berlin

Berlin, , Germany

Ruhrlandklinik, Westdeutsches Lungenzentrum am Universitätsklinikum Essen gGmbH

Essen, , Germany

Universitätsklinikum Gießen und Marburg GmbH

Giessen, , Germany

Medizinische Hochschule Hannover

Hanover, , Germany

Universitätsklinikum Tübingen

Tübingen, , Germany

Hospital Vall d'Hebron

Barcelona, , Spain

Sahlgrenska US, Göteborg

Gothenburg, , Sweden

Stockholm CF-Center , B59, Huddinge Universitetssjukhus

Stockholm, , Sweden

Royal Brompton Hospital

London, , United Kingdom

Countries

United States; Belgium; Canada; France; Germany; Spain; Sweden; United Kingdom

References

Goss CH, Fajac I, Jain R, Seibold W, Gupta A, Hsu MC, Sutharsan S, Davies JC, Mall MA. Efficacy and safety of inhaled ENaC inhibitor BI 1265162 in patients with cystic fibrosis: BALANCE-CF 1, a randomised, phase II study. Eur Respir J. 2022 Feb 17;59(2):2100746. doi: 10.1183/13993003.00746-2021. Print 2022 Feb.

Reference Type: DERIVED

PMID: 34385272 (View on PubMed)

Goss CH, Jain R, Seibold W, Picard AC, Hsu MC, Gupta A, Fajac I. An innovative phase II trial to establish proof of efficacy and optimal dose of a new inhaled epithelial sodium channel inhibitor BI 1265162 in adults and adolescents with cystic fibrosis: BALANCE-CFTM 1. ERJ Open Res. 2020 Dec 7;6(4):00395-2020. doi: 10.1183/23120541.00395-2020. eCollection 2020 Oct.

Reference Type: DERIVED

PMID: 33313307 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.mystudywindow.com

Related Info

Other Identifiers

2019-000261-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

1399-0003

Identifier Type: -

Identifier Source: org_study_id