Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

NCT04999020 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: ALXN1210-DM-3102021-001200-15
• Study Type: interventional
• Target: 38
• Locations: 13 countries
• Sites: 111

Brief Summary

This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in adult participants with dermatomyositis (DM).

Conditions

Dermatomyositis

Keywords

Ravulizumab; ALXN1210; Ultomiris; Pharmacokinetics; Pharmacodynamics; Efficacy; DM

Outcome Measures

Primary Outcomes (1)

• 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

  Data are presented for the number of participants with a TIS40 response, defined as an IMACS-TIS score ≥ 40 at Week 26. IMACS-TIS is a clinical instrument that encompasses 6 core set measure (CSMs) (physician, patient, extra-muscular global activity, muscle strength, Health Assessment Questionnaire \[HAQ\], and muscle enzyme levels). A Total Improvement Score (TIS: 0-100), was determined by summing scores in each CSM, and was based on the improvement and relative weight of each CSM. A higher score indicated greater improvement. TIS40 was considered a moderate improvement score.

  Week 26

Secondary Outcomes (15)

• TIS at Week 26

  Week 26

• Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26

  Baseline, Week 26

• Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26

  Baseline, Week 26

• Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26

  Baseline, Week 26

• Change From Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26

  Baseline, Week 26

Study Arms (2)

Ravulizumab

EXPERIMENTAL

Participants will receive ravulizumab in both Parts A and B.

Drug: Ravulizumab

Placebo

PLACEBO COMPARATOR

Participants will receive placebo in both Parts A and B.

Drug: Placebo

Interventions

Ravulizumab DRUG

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.

Also known as: ALXN1210, Ultomiris

Ravulizumab

Placebo DRUG

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.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (111)

Research Site

Phoenix, Arizona, 85032, United States

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Irvine, California, 92617, United States

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Santa Monica, California, 90404, United States

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Washington D.C., District of Columbia, 20037, United States

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Tampa, Florida, 33612, United States

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Atlanta, Georgia, 30322, United States

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Chicago, Illinois, 60637, United States

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Fairway, Kansas, 66205, United States

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Baltimore, Maryland, 21205, United States

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Las Vegas, Nevada, 89145, United States

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Manhasset, New York, 11030, United States

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New York, New York, 10021, United States

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Chapel Hill, North Carolina, 27599, United States

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Durham, North Carolina, 27710, United States

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Cleveland, Ohio, 44195, United States

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Pittsburgh, Pennsylvania, 15213, United States

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Charleston, South Carolina, 29425, United States

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Memphis, Tennessee, 38120, United States

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North Richland Hills, Texas, 76180, United States

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Seattle, Washington, 98122, United States

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Camperdown, 2050, Australia

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Murdoch, WA6150, Australia

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Belém, 66095-055, Brazil

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Belo Horizonte, 30150-221, Brazil

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Campinas, 13083, Brazil

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Juiz de Fora, 36010-570, Brazil

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Natal, 59025-050, Brazil

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Porto Alegre, 90035-000, Brazil

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Porto Alegre, 90480-000, Brazil

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Salvador, 40150-150, Brazil

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São José do Rio Preto, 15090-000, Brazil

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São Paulo, 01308-050, Brazil

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São Paulo, 05403-010, Brazil

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Serra, 29160-750, Brazil

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Vitória, 29050-400, Brazil

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Lille, 59037, France

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Lyon, 69437, France

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Nantes, 44093, France

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Paris, 75010, France

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Paris, 75651, France

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Strasbourg, 67098, France

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Toulouse, 31059, France

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Düsseldorf, 40225, Germany

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Erlangen, 91054, Germany

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Essen, 45147, Germany

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Freiburg im Breisgau, 79106, Germany

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Göttingen, 37075, Germany

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Halle, 06120, Germany

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Jena, 07747, Germany

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Bari, 70124, Italy

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Brescia, 25123, Italy

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Catania, 95123, Italy

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Florence, 50134, Italy

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Iesi, 60035, Italy

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Messina, 98124, Italy

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Milan, 20132, Italy

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Pavia, 27100, Italy

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Pisa, 56126, Italy

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Reggio Emilia, 42122, Italy

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Roma, 00128, Italy

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Roma, 00168, Italy

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Rome, 00189, Italy

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Rozzano, 20089, Italy

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Siena, 53100, Italy

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Torino, 10126, Italy

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Torrette AN, 60126, Italy

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Udine, 33100, Italy

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Verona, 37126, Italy

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Bunkyō City, 113-8655, Japan

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Hiroshima, 734-8551, Japan

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Iruma-Gun, 350-0495, Japan

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Nagoya, 467-8602, Japan

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Narita-shi, 286-8520, Japan

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Okayama, 700-8558, Japan

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Osaka, 565-0871, Japan

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Sapporo, 060-8648, Japan

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Sendai, 980-8574, Japan

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Urayasu-shi, 279-0021, Japan

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Warsaw, 02-637, Poland

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Daejeon, 35015, South Korea

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Junggu, 22332, South Korea

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Seoul, 02447, South Korea

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Seoul, 03080, South Korea

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Seoul, 04763, South Korea

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Seoul, 6591, South Korea

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A Coruña, 15006, Spain

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Barakaldo, 48903, Spain

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Barcelona, 08035, Spain

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Barcelona, 08036, Spain

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Bilbao (Vizcaya), 48013, Spain

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L'Hospitalet de Llobregat, 08907, Spain

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Las Palmas de Gran Canaria, 35020, Spain

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Madrid, 28034, Spain

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Madrid, 28040, Spain

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Madrid, 28041, Spain

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Oviedo, 33011, Spain

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Santander, 39008, Spain

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Seville, 41010, Spain

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Seville, 41013, Spain

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Valencia, 46026, Spain

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Zaragoza, 50009, Spain

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Kaohsiung City, 83301, Taiwan

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Taichung, 40705, Taiwan

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Taoyuan, 33305, Taiwan

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Altındağ-Ankara, 06230, Turkey (Türkiye)

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Istanbul, 34093, Turkey (Türkiye)

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Edinburgh, EH4 2XU, United Kingdom

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Liverpool, L9 7AL, United Kingdom

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London, SE5 9RS, United Kingdom

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Salford, M6 8HD, United Kingdom

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West Bromwich, B71 4HJ, United Kingdom

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Related Links

• Related Info

MeSH Terms

Conditions

Dermatomyositis

Interventions

ravulizumab

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Limitations and Caveats

Part A did not meet its primary endpoint and the study was terminated.

Results Point of Contact

• Title: Alexion Pharmaceuticals Inc.
• Organization: Alexion Pharmaceuticals Inc.

clinicaltrials@alexion.com

855-752-2356

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 4, 2021
• First Posted: August 10, 2021
• Study Start: November 19, 2021
• Primary Completion: October 26, 2023
• Study Completion: May 8, 2024
• Last Updated: July 9, 2025
• Results First Posted: December 6, 2024

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

FR (7); AU (2); BR (13); DE (7); KR (6); ES (16); TU (2); US (20); PL (1); TW (3); GB (5); IT (19); JP (10)