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

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.

Timeline

Start

2019-09-16

Primary completion

2020-04-16

Completion

2020-04-24

Outcome

Missed primary endpoint

Stopped: “Not due to safety reasons”

registry p = 0.5468; Adjusted means difference 1.5 (95% CI -3.5 to 6.5) NCT04059094 ↗

paper The final results including these patients (+1.5% ppFEV1, 200 µg twice-daily dose versus placebo) were not supportive of relevant clinical effect. PMID 34385272 ↗

Publications

• 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.
• 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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	BI 1265162	Unknown	20 ug	Inhaled
Subject	BI 1265162	Unknown	50 ug	Inhaled
Subject	BI 1265162	Unknown	100 ug	Inhaled
Subject	BI 1265162	Unknown	200 ug	Inhaled

Indications