Dose-range Finding Efficacy and Safety Study for QBW251 in COPD Patients

Phase 2 Completed 974 enrolled Novartis Pharmaceuticals

RandomizedParallel-groupTriple-blindTreatment

Summary

This clinical study was designed to support the dose selection for future studies by evaluating efficacy and safety of different QBW251 doses in Chronic obstructive pulmonary disease (COPD) patients with chronic bronchitis and a history of exacerbations, compared to placebo, when added to a triple inhaled therapy of LABA, LAMA and ICS.

Timeline

Start: 2019-09-12
Primary completion: 2021-10-08
Completion: 2022-02-01

Outcome

Missed primary endpoint

registry analysis (superiority test); QBW251 450 mg vs Placebo; p = 0.628; Least Squares Mean Difference 0.011 (90% CI -0.027 to 0.050); ANCOVA NCT04072887 ↗

paper Conclusions: The primary endpoint was negative, as icenticaftor did not improve trough FEV1 over 12 weeks. PMID 37411039 ↗

Publications

Martinez FJ, Criner GJ, Gessner C, Jandl M, Scherbovsky F, Shinkai M, Siler TM, Vogelmeier CF, Voves R, Wedzicha JA, Bartels C, Bottoli I, Byiers S, Cardenas P, Eckert JH, Gutzwiller FS, Knorr B, Kothari M, Parlikar R, Tanase AM, Franssen FME. Icenticaftor, a CFTR Potentiator, in COPD: A Multicenter, Parallel-Group, Double-Blind Clinical Trial. Am J Respir Crit Care Med. 2023 Aug 15;208(4):417-427. doi: 10.1164/rccm.202303-0458OC.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Icenticaftor	Small molecule	25 mg	Oral
Subject	Icenticaftor	Small molecule	75 mg	Oral
Subject	Icenticaftor	Small molecule	150 mg	Oral
Subject	Icenticaftor	Small molecule	300 mg	Oral
Subject	Icenticaftor	Small molecule	450 mg	Oral
Background	Fluticasone Furoate	Other / unclassified	—	Inhaled
Background	Umeclidinium	Other / unclassified	—	Inhaled
Background	Vilanterol	Other / unclassified	—	Inhaled