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Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

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ClinicalTrials.gov Identifier: NCT04999020
Recruitment Status : Recruiting
First Posted : August 10, 2021
Last Update Posted : June 22, 2022
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Study Description
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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).

Condition or disease Intervention/treatment Phase
Dermatomyositis Drug: Ravulizumab Drug: Placebo Phase 2 Phase 3

Detailed Description:
The study will be conducted in 2 parts: Part A (Phase 2) and Part B (Phase 3). There will be 3 periods in both Part A and Part B of this study: Screening Period, Randomized Controlled Period, and Open-Label Extension Period.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 180 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : December 2, 2021
Estimated Primary Completion Date : August 31, 2022
Estimated Study Completion Date : August 31, 2024

Resource links provided by the National Library of Medicine

Drug Information available for: Ravulizumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Ravulizumab
Participants will receive ravulizumab in both Parts A and B.
 Drug: 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.
Other Names:
  • ALXN1210
  • Ultomiris
Placebo Comparator: Placebo
Participants will receive placebo in both Parts A and B.
 Drug: 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.


Outcome Measures
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Primary Outcome Measures :
  1. Part A: Proportion Of Participants With A ≥ 20-point Improvement Response On International Myositis Assessment And Clinical Studies-Total Improvement Score (IMACS-TIS) (TIS20) At Week 26 Of The Randomized Controlled Period [ Time Frame: Week 26 ]
  2. Part B: Proportion Of Participants With A ≥ 20-point Improvement Response On TIS20 At Week 50 [ Time Frame: Week 50 ]

Secondary Outcome Measures :
  1. Part A: Mean TIS At Week 26 [ Time Frame: Week 26 ]
  2. Part A: Mean Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 26 [ Time Frame: Baseline, Week 26 ]
  3. Part A: Change From Baseline Of Each Of The IMACS Core Set Measures At Week 26 [ Time Frame: Baseline, Week 26 ]
  4. Part A: Time To First CDASI Activity Score Improvement [ Time Frame: Baseline through Week 26 ]
    Minimally clinically important differences (MCID) = 7-point improvement.

  5. Part A: Proportion Of Participants With CDASI MCID Improvement At Week 26 [ Time Frame: Week 26 ]
  6. Part A: Change In Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) At Week 26 [ Time Frame: Baseline, Week 26 ]
  7. Part A: Proportion Of Participants With TIS20 At Each Visit [ Time Frame: Baseline through Week 26 ]
  8. Part A: Proportion Of Participants With A ≥ 40-point Improvement Response On IMACS-TIS (TIS40) At Each Visit [ Time Frame: Baseline through Week 26 ]
  9. Part A: Proportion Of Participants With A ≥ 60-point Improvement Response On IMACS-TIS (TIS60) At Each Visit [ Time Frame: Baseline through Week 26 ]
  10. Part A: Time To First Response Of TIS20, TIS40, Or TIS60 [ Time Frame: Baseline through Week 26 ]
  11. Part A: Time To First IMACS Myositis Core Set Measure Improvements [ Time Frame: Baseline through Week 26 ]
  12. Part B: Mean TIS At Week 50 [ Time Frame: Week 50 ]
  13. Part B: Mean Change From Baseline In Manual Muscle Testing Subset Of 8 Muscles (MMT-8) At Week 50 [ Time Frame: Baseline, Week 50 ]
  14. Part B: Mean Change From Baseline In Extra-muscular Disease Activity Based On Myositis Disease Activity Assessment Tool (MDAAT) At Week 50 [ Time Frame: Baseline, Week 50 ]
  15. Part B: Mean Change From Baseline In CDASI Activity Score At Week 50 [ Time Frame: Baseline, Week 50 ]
  16. Part B: Mean Change From Baseline In Patient Global Activity Assessment At Week 50 [ Time Frame: Baseline, Week 50 ]
  17. Part B: Mean Change From Baseline In Physician Global Activity Assessment At Week 50 [ Time Frame: Baseline, Week 50 ]
  18. Part B: Mean Change From Baseline In Health Assessment Questionnaire (HAQ) At Week 50 [ Time Frame: Baseline, Week 50 ]
  19. Part B: Mean Change From Baseline In Muscle Enzyme Values At Week 50 [ Time Frame: Baseline, Week 50 ]
  20. Part B: Mean TIS At Each Visit From Week 2 Through Week 50 [ Time Frame: Week 2 through Week 50 ]
  21. Part B: Proportion Of Participants With TIS20 At Each Visit [ Time Frame: Baseline through Week 50 ]
  22. Part B: Proportion Of Participants With TIS40 At Each Visit [ Time Frame: Baseline through Week 50 ]
  23. Part B: Proportion Of Participants With TIS60 At Each Visit [ Time Frame: Baseline through Week 50 ]
  24. Part B: Time To First Response Of TIS20, TIS40, Or TIS60 [ Time Frame: Baseline through Week 50 ]
  25. Part B: Time To First IMACS Myositis Core Set Measure Improvements [ Time Frame: Baseline through Week 50 ]
  26. Part B: Time To First CDASI Activity Score Improvement [ Time Frame: Baseline through Week 50 ]
    MCID = 7-point improvement

  27. Part B: Proportion Of Participants With CDASI MCID Improvement At Week 50 [ Time Frame: Week 50 ]
  28. Part B: Change In CDA-IGA At Week 50 [ Time Frame: Week 50 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • 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 American College of Rheumatology/European League Against Rheumatism classification criteria for definite or probable DM.
  • Participants who have an inadequate response (that is, continued impairment by medical doctor report) or are intolerant to 2 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 Exclusion Criteria:

  • Cancer-associated myositis, defined as the diagnosis of myositis within 3 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured and 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.
  • Participants with significant muscle damage (for example, severe muscle atrophy, end stage muscle disease) as per investigator opinion.
  • 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04999020


Contacts
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Contact: Alexion Pharmaceuticals, Inc. 1-855-752-2356 clinicaltrials@alexion.com

Locations
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United States, California
Clinical Trial Site Recruiting
Orange, California, United States, 92868
United States, Florida
Clinical Trial Site Recruiting
Tampa, Florida, United States, 33612
United States, Kansas
Clinical Trial Site Recruiting
Kansas City, Kansas, United States, 66160
United States, Maryland
Clinical Trial Site Recruiting
Baltimore, Maryland, United States, 21224
United States, Pennsylvania
Clinical Trial Site Recruiting
Pittsburgh, Pennsylvania, United States, 15213
France
Clinical Trial Site Recruiting
Lille Cedex, France, 59037
Clinical Trial Site Recruiting
Paris, France, 75010
Clinical Trial Site Recruiting
Strasbourg, France, 67098
Clinical Trial Site Recruiting
Toulouse Cedex 9, France, 31059
Germany
Clinical Trial Site Recruiting
Dusseldorf, Germany, 40225
Clinical Trial Site Recruiting
Erlangen, Germany, 91054
Clinical Trial Site Recruiting
Essen, Germany, 45147
Clinical Trial Site Recruiting
Freiburg, Germany, 79106
Italy
Clinical Trial Site Recruiting
Bari, BA, Italy, 70124
Clinical Trial Site Recruiting
Brescia, BS, Italy, 25123
Clinical Trial Site Recruiting
Pisa, PI, Italy, 56126
Clinical Trial Site Recruiting
Pavia, PV, Italy, 27100
Clinical Trial Site Recruiting
Roma, Italy, 00168
Japan
Clinical Trial Site Recruiting
Bunkyo-ku, Tokyo, Japan, 113-8655
Korea, Republic of
Clinical Trial Site Recruiting
Incheon, Korea, Republic of, 22332
Clinical Trial Site Recruiting
Seoul, Korea, Republic of, 03080
Clinical Trial Site Recruiting
Seoul, Korea, Republic of, 04763
Clinical Trial Site Recruiting
Seoul, Korea, Republic of, 06591
Spain
Clinical Trial Site Recruiting
L'Hospitalet de Llobregat, Barcelona, Spain, 8907
Clinical Trial Site Recruiting
Bilbao, Vizcaya, Spain, 48013
Clinical Trial Site Recruiting
Barcelona, Spain, 08035
Clinical Trial Site Recruiting
Barcelona, Spain, 08036
Clinical Trial Site Recruiting
Madrid, Spain, 28034
Clinical Trial Site Recruiting
Madrid, Spain, 28041
United Kingdom
Clinical Trial Site Recruiting
Liverpool, United Kingdom, L9 7AL
Clinical Trial Site Recruiting
Salford, United Kingdom, M55AP
Sponsors and Collaborators
Alexion Pharmaceuticals
More Information
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04999020    
Other Study ID Numbers: ALXN1210-DM-310
2021-001200-15 ( EudraCT Number )
First Posted: August 10, 2021    Key Record Dates
Last Update Posted: June 22, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Ravulizumab
ALXN1210
Ultomiris
Pharmacokinetics
 Pharmacodynamics
Efficacy
DM
Additional relevant MeSH terms:
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Dermatomyositis
Polymyositis
Myositis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
 Connective Tissue Diseases
Skin Diseases
Ravulizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs