JRCT ID: jRCT2031210681
Registered date:18/03/2022
Ravulizumab versus Placebo in Adult Participants with Dermatomyositis
Basic Information
Recruitment status | Recruiting |
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Health condition(s) or Problem(s) studied | Dermatomyositis |
Date of first enrollment | 21/12/2022 |
Target sample size | 180 |
Countries of recruitment | USA,Japan,Italy,Japan,France,Japan,Germany,Japan,UK,Japan |
Study type | Interventional |
Intervention(s) | 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. |
Outcome(s)
Primary Outcome | 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 |
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Secondary Outcome | 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 |
Key inclusion & exclusion criteria
Age minimum | >= 18age old |
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Age maximum | Not applicable |
Gender | Both |
Include criteria | -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. |
Exclude criteria | 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. |
Related Information
Primary Sponsor | Wuebbenhorst Hiroaki |
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Secondary Sponsor | |
Source(s) of Monetary Support | |
Secondary ID(s) | NCT04999020 |
Contact
Public contact | |
Name | Hiroaki Wuebbenhorst |
Address | Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan Tokyo Japan 108-0023 |
Telephone | +81-3-3457-9559 |
Hiroaki.Wuebbenhorst@alexion.com | |
Affiliation | Alexion Pharma GK |
Scientific contact | |
Name | Hiroaki Wuebbenhorst |
Address | Tamachi Station Tower N, 3-1-1, Shibaura, Minato-ku, Tokyo 108-0023, Japan Tokyo Japan 108-0023 |
Telephone | +81-3-3457-9559 |
Hiroaki.Wuebbenhorst@alexion.com | |
Affiliation | Alexion Pharma GK |