臨床研究等提出・公開システム

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis

Ravulizumab versus Placebo in Adult Participants with Dermatomyositis

Kobayashi Hiroshi

Alexion Pharma GK

Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan

JPDept-DevOps-PMCO@alexion.com

Kobayashi Hiroshi

Alexion Pharma GK

Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan

+81-3-3457-9559

JPDept-DevOps-PMCO@alexion.com

Complete

Mar. 16, 2022

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

-Meet 2017 ACR/EULAR classification criteria for definite or probable DM
-Participants who have an inadequate response or are intolerant to 2 or more DM treatments, including systemic glucocorticoids or ISTs (eg azathioprine, methotrexate, rituximab, IVIg) either in combination or as monotherapy.

Participants with significant muscle damage (eg, severe muscle atrophy, end stage muscle disease, MRI with severe atrophy or fibrofatty replacement) as per investigator opinion.

History of N meningitidis infection.

Both

Dermatomyositis

Eligible participants will be enrolled into the study and will be randomized in a 2:1 ratio (Part A) or a 1:1 ratio (Part B) to receive intravenous (IV) ravulizumab or placebo.
The study intervention dose for each participant will be based on body weight. The dosing regimen for both Parts A and B consists of a loading dose followed by maintenance doses administered every 8 weeks (q8w). The maintenance dosing will be initiated 2 weeks after the loading dose is administered.
The Randomized Controlled Period for Part A will be 26 weeks in duration. The Randomized Controlled Period for Part B will be 50 weeks in duration. Participants will receive ravulizumab during the OLE Period until ravulizumab is registered or approved (in accordance with country-specific regulations) or for up to 74 weeks (approximately 1.5 years), whichever occurs first. The total study treatment duration for Part A will be approximately 2 years and for Part B approximately 2.5 years.

Part A: Proportion of participants with a >= 20-point improvement response on IMACS-TIS (TIS40) at Week 26 of the Randomized Controlled Period
Part B: Proportion of participants with a >= 20-point improvement response on IMACS-TIS (TIS40) at Week 50 as per defined composite estimand of the Randomized Controlled Period

Part A:
-Mean TIS at Week 26
-Mean change from baseline in CDASI Activity Score at Week 26
-Change from baseline of each of the IMACS core set measures at Week 26
-Time to first CDASI Activity Score improvement (minimally clinically important differences [MCID] = 7-point improvement)
-Proportion of participants with CDASI MCID improvement at Week 26
-Change in CDA-IGA at Week 26
-Proportion of participants with TIS20 at each visit
-Proportion of participants with TIS40 at each visit
-Proportion of participants with TIS60 at each visit
-Time to First Response of TIS20, TIS40, or TIS60 respectively
-Time to first IMACS myositis core set measure improvements
Part B:
-Mean TIS at Week 50
-Mean change from baseline in MMT-8 at Week 50
-Mean change from baseline in extra-muscular disease activity based on MDAAT at Week 50
-Mean change from baseline in CDASI Activity Score at Week 50

+81-3-5800-8743

Nov. 24, 2021

No

USA/Italy/France/Germany/UK