A Randomised, Parallel-Group, Double-Blind, Placebo-Controlled Phase 3 Study to Determine the Effectiveness of the Type I Interferon Receptor Antibody, Anifrolumab, in SYstemic Sclerosis: Daisy Study Design and Rationale

Authors

Dinesh Khanna
Christopher P Denton
Shervin Assassi
Masataka Kuwana
Yannick Allanore
Robyn T Domsic
Christi Kleoudis
John Xu
Eszter Csomor
Caroline Seo
Marius Albulescu
Raj Tummala
Hussein Al-Mossawi
Rubana N Kalyani
Francesco Del Galdo

Language

English

Publication Date

8-1-2024

Journal

Clinical and Experimental Rheumatology

DOI

10.55563/clinexprheumatol/s8qcyu

PMID

39152751

Abstract

Objectives: The type I interferon pathway is a promising target for treatment of patients with systemic sclerosis (SSc). Here, we describe the design of a multinational, randomised phase 3 study to Determine the effectiveness of the type I interferon receptor antibody, Anifrolumab, In SYstemic sclerosis (DAISY).

Methods: DAISY includes a 52-week double-blind, placebo-controlled treatment period, a 52-week open-label active treatment period, and a 12-week safety follow-up period. The patient population includes a planned 306 adults with limited or diffuse cutaneous active SSc who satisfied American College of Rheumatology/European Alliance of Associations for Rheumatology 2013 SSc criteria. Use of standard immunosuppressants, including mycophenolate mofetil, at a stable dose prior to randomisation is permitted in addition to weekly subcutaneous anifrolumab or placebo. Efficacy will be assessed at Week 52 via Revised-Composite Response Index in SSc (CRISS)-25 response (primary endpoint). Lung function and skin thickness will be assessed via change from baseline in forced vital capacity in patients with SSc-associated interstitial lung disease and modified Rodnan Skin Score, respectively (key secondary endpoints).

Conclusions: The DAISY trial will evaluate the efficacy and safety of anifrolumab as a first-in-class treatment option for patients with both limited and diffuse cutaneous SSc and will provide insight into the contributions of type I interferon to SSc pathogenesis. Revised-CRISS-25 can account for improvement and worsening in a broad set of validated clinical measures beyond lung function and skin thickness, including clinician- and patient-reported outcomes, capturing the heterogeneity of SSc.

Keywords

Adult, Humans, Antibodies, Monoclonal, Humanized, Clinical Trials, Phase III as Topic, Double-Blind Method, Randomized Controlled Trials as Topic, Receptor, Interferon alpha-beta, Scleroderma, Systemic, Treatment Outcome

Published Open-Access

yes

Recommended Citation

Khanna, Dinesh; Denton, Christopher P; Assassi, Shervin; et al., "A Randomised, Parallel-Group, Double-Blind, Placebo-Controlled Phase 3 Study to Determine the Effectiveness of the Type I Interferon Receptor Antibody, Anifrolumab, in SYstemic Sclerosis: Daisy Study Design and Rationale" (2024). Faculty, Staff and Student Publications. 5311.
https://digitalcommons.library.tmc.edu/uthgsbs_docs/5311