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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04999020




Registration number
NCT04999020
Ethics application status
Date submitted
4/08/2021
Date registered
10/08/2021
Date last updated
9/07/2025

Titles & IDs
Public title
Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
Scientific title
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
Secondary ID [1] 0 0
2021-001200-15
Secondary ID [2] 0 0
ALXN1210-DM-310
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dermatomyositis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ravulizumab
Treatment: Drugs - Placebo

Experimental: Ravulizumab - Participants will receive ravulizumab in both Parts A and B.

Placebo comparator: Placebo - Participants will receive placebo in both Parts A and B.


Treatment: Drugs: Ravulizumab
Intravenous dosing will consist 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.

Treatment: Drugs: Placebo
Intravenous dosing will consist of a loading dose followed by maintenance doses administered q8w. The maintenance dosing will be initiated 2 weeks after the loading dose is administered.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS-TIS) (TIS40) Response at Week 26 of the Randomized Controlled Period
Timepoint [1] 0 0
Week 26
Secondary outcome [1] 0 0
TIS at Week 26
Timepoint [1] 0 0
Week 26
Secondary outcome [2] 0 0
Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26
Timepoint [2] 0 0
Baseline, Week 26
Secondary outcome [3] 0 0
Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26
Timepoint [3] 0 0
Baseline, Week 26
Secondary outcome [4] 0 0
Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26
Timepoint [4] 0 0
Baseline, Week 26
Secondary outcome [5] 0 0
Change From Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26
Timepoint [5] 0 0
Baseline, Week 26
Secondary outcome [6] 0 0
Change From Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26
Timepoint [6] 0 0
Baseline, Week 26
Secondary outcome [7] 0 0
Change From Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26
Timepoint [7] 0 0
Baseline, Week 26
Secondary outcome [8] 0 0
Change From Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26
Timepoint [8] 0 0
Baseline, Week 26
Secondary outcome [9] 0 0
Number of Participants With CDASI Response (>=7-point Improvement) at Week 26
Timepoint [9] 0 0
Week 26
Secondary outcome [10] 0 0
Number of Participants With Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26
Timepoint [10] 0 0
Week 26
Secondary outcome [11] 0 0
Number of Participants With = 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26
Timepoint [11] 0 0
Week 26
Secondary outcome [12] 0 0
Number of Participants With = 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26
Timepoint [12] 0 0
Week 26
Secondary outcome [13] 0 0
Time to First Response of TIS20, TIS40, or TIS60
Timepoint [13] 0 0
Baseline through Week 26
Secondary outcome [14] 0 0
Number of Participants With Clinical Worsening (CW) During the RCP At 2 Consecutive Visits
Timepoint [14] 0 0
Baseline through Week 26
Secondary outcome [15] 0 0
Number of Participants Who Received Acute Rescue Therapy With Standard DM Treatment
Timepoint [15] 0 0
Baseline through Week 26

Eligibility
Key inclusion criteria
Key

* 18 years of age or older at the time of signing the informed consent.
* Body weight = 30 kilograms at the time of Screening.
* Male or female.
* Diagnosis: Meet 2017 American College of Rheumatology/European League Against Rheumatism classification criteria for definite or probable DM.
* Participants who have an inadequate response or are intolerant to 1 or more DM treatments, including systemic corticosteroids or immunosuppressive/immunomodulatory therapies (for example, azathioprine, methotrexate, rituximab, intravenous immunoglobulin), either in combination or as monotherapy.
* Vaccinated against Neisseria meningitidis within 3 years prior to initiating ravulizumab as per national and local guidelines. Participants must receive the vaccination at least 2 weeks before first study intervention. The sponsor recommends that national and local guidelines for prophylactic antibiotics should also be followed.
* Female participants of childbearing potential and male participants must follow specified contraception guidance as described in the protocol.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participants who have been diagnosed with cancer within the last 3 years need to have appropriate negative cancer screening as per local standard of care within 6 months before Screening (basal or squamous cell skin cancer or carcinoma in situ of the cervix needs to have been excised and without evidence of residual disease for at least 3 months before Screening).
* Evidence of active malignant disease or malignancies diagnosed within the previous 3 years including hematological malignancies and solid tumors.
* Participants with other forms of myositis.
* As per investigator discretion, participants with significant muscle damage (for example, severe muscle atrophy, end stage muscle disease, MRI with severe atrophy or fibrofatty replacement)
* History of Neisseria meningitidis infection.
* Human immunodeficiency virus (HIV) infection (evidenced by HIV Type 1 or Type 2 antibody titer).
* Active systemic bacterial, viral, or fungal infection within 14 days prior to ravulizumab administration.
* Presence of fever = 38°Celsius (100.4°Fahrenheit) within 7 days prior to study drug administration on Day 1.
* History of hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab.
* Pregnant, breastfeeding, or intending to conceive during the course of the study.
* Inability or unwillingness to adhere to the protocol requirements.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Research Site - Camperdown
Recruitment hospital [2] 0 0
Research Site - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
WA6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
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United States of America
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California
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United States of America
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District of Columbia
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United States of America
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Florida
Country [5] 0 0
United States of America
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Georgia
Country [6] 0 0
United States of America
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Illinois
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United States of America
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Kansas
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United States of America
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Maryland
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Nevada
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New York
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North Carolina
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Ohio
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Pennsylvania
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South Carolina
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Tennessee
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Texas
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United States of America
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Washington
Country [18] 0 0
Brazil
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Belo Horizonte
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Brazil
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Belém
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Brazil
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Campinas
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Brazil
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Juiz de Fora
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Brazil
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Natal
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Brazil
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Porto Alegre
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Brazil
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Salvador
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Brazil
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Sao Jose Do Rio Preto
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Brazil
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Serra
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Brazil
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São Paulo
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Brazil
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Vitória
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France
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Lille
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France
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Lyon
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France
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Nantes cedex 1
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France
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Paris Cedex 13
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France
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Paris
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France
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Strasbourg
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France
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Toulouse
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Germany
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Duesseldorf
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Germany
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Erlangen
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Germany
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Essen
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Germany
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Freiburg im Breisgau
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Germany
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Goettingen
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Germany
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Halle
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Germany
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Jena
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Italy
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Bari
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Italy
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Brescia
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Italy
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Catania
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Italy
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Firenze
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Italy
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Jesi
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Italy
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Messina
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Italy
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Milano
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Italy
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Pavia
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Italy
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Pisa
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Reggio Emilia
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Roma
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Rome
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Italy
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Rozzano
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Siena
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Torino
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Italy
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Torrette AN
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Udine
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Italy
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Verona
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Japan
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Bunkyo-ku
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Hiroshima-shi
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Iruma-Gun
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Nagoya-shi
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Okayama
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Osaka
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Sendai-shi
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Korea, Republic of
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Daejeon
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Jung-gu
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Korea, Republic of
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Seoul
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Poland
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Warszawa
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A Coruna
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Barakaldo
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Barcelona
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Bilbao (Vizcaya)
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Spain
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L'Hospitalet de Llobregat
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Spain
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Las Palmas de Gran Canaria
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Madrid
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Oviedo
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Santander
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Sevilla
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Spain
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Valencia
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Spain
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Zaragoza
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Taiwan
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Kaohsiung City
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Taiwan
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Taichung
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Taiwan
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Taoyuan City
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Turkey
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Altindag-Ankara
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Turkey
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Istanbul
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United Kingdom
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Edinburgh
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United Kingdom
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Liverpool
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United Kingdom
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London
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United Kingdom
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Salford
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United Kingdom
State/province [96] 0 0
West Bromwich

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.