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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
D. Khanna1, C.P. Denton2, S. Assassi3, M. Kuwana4, Y. Allanore5, R.T. Domsic6, C. Kleoudis7, J. Xu8, E. Csomor9, C. Seo10, M. Albulescu11, R. Tummala12, H. Al-Mossawi13, R.N. Kalyani14, F. Del Galdo15
- University of Michigan Scleroderma Program, Ann Arbor, MI, USA. khannad@med.umich.edu
- Centre for Rheumatology, Division of Medicine, University College London, UK.
- McGovern Medical School, Division of Rheumatology, University of Texas Health Science Center at Houston, TX, USA.
- Department of Allergy and Rheumatology, Nippon Medical School, Tokyo, Japan.
- Rheumatology Department, Cochin Hospital, Université Paris Cité, INSERM U1016, Paris, France.
- University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
- Biopharmaceuticals R & D, AstraZeneca, Gaithersburg, MD, USA.
- Biopharmaceuticals R & D, AstraZeneca, Gaithersburg, MD, USA.
- BioPharmaceuticals R & D, AstraZeneca, Cambridge, UK.
- Biopharmaceuticals Medical Evidence, AstraZeneca, Gaithersburg, MD, USA.
- Biopharmaceuticals R & D, AstraZeneca, Cambridge, UK.
- Biopharmaceuticals R & D, AstraZeneca, Gaithersburg, MD, USA.
- Biopharmaceuticals R & D, AstraZeneca, Cambridge, UK.
- Biopharmaceuticals R & D, AstraZeneca, Gaithersburg, MD, USA.
- Department of Rheumatology, Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, UK. f.delgaldo@leeds.ac.uk
CER17799
2024 Vol.42, N°8
PI 1635, PF 1644
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PMID: 39152751 [PubMed]
Received: 24/04/2024
Accepted : 10/06/2024
In Press: 14/08/2024
Published: 14/08/2024
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.
