Proof of Concept Study of Rilzabrutinib in Adult Participants With Moderate-to-severe Asthma

Summary

This is a parallel, treatment, Phase 2, double-blind, 2 arm, 12-week Proof of Concept (PoC) study with 2 staggered cohorts (2 arms in each cohort) that is designed to assess the efficacy, safety, and tolerability of rilzabrutinib in adult participants (aged 18-70 years) with moderate-to-severe asthma who are not well controlled on ICS/LABA therapy. Study treatment includes investigational medicinal product (IMP) (rilzabrutinib or placebo) added-on to a background therapy of ICS/LABA (fluticasone/salmeterol \[non-investigational medicinal product\], standardized at screening). Background therapy of ICS/LABA will be withdrawn during the 12week randomized treatment period and resumed at the end of the IMP treatment period, as outlined below: * Screening period (4 weeks) * Randomized IMP treatment period (12 weeks ± 3 days) * Background therapy stabilization phase (4 weeks) * Background therapy withdrawal phase (4-5 weeks) * No background therapy phase (3-4 weeks) * Post IMP treatment safety follow-up period (4 weeks ± 3 days)

Timeline

Start

2021-12-12

Primary completion

2024-02-06

Completion

2024-02-28

Publications

• Maspero JF, Pavord ID, Wechsler ME, Busse W, Laidlaw TM, Mroz RM, Liu T, Mikol V, Suratt BT, Gereige J, Mannent L, Martincova R. Rilzabrutinib for patients with moderate-to-severe asthma with uncontrolled symptoms: a double-blind, placebo-controlled, phase 2 study. Lancet Respir Med. 2026 May;14(5):405-416. doi: 10.1016/S2213-2600(25)00439-4. Epub 2026 Apr 2.
• Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available.

Drugs

Indications