Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

May 9, 2024 updated by: Alexion Pharmaceuticals, Inc.

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

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).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

38

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75010
        • Research Site
      • Strasbourg, France, 67098
        • Research Site
      • Toulouse, France, 31059
        • Research Site
  • Germany
      • Erlangen, Germany, 91054
        • Research Site
      • Essen, Germany, 45147
        • Research Site
      • Freiburg im Breisgau, Germany, 79106
        • Research Site
      • Halle, Germany, 06120
        • Research Site
  • Italy
      • Bari, Italy, 70124
        • Research Site
      • Brescia, Italy, 25123
        • Research Site
      • Firenze, Italy, 50134
        • Research Site
      • Milano, Italy, 20132
        • Research Site
      • Pavia, Italy, 27100
        • Research Site
      • Pisa, Italy, 56126
        • Research Site
      • Roma, Italy, 00168
        • Research Site
      • Rome, Italy, 00189
        • Research Site
      • Torino, Italy, 10126
        • Research Site
  • Japan
      • Bunkyo-ku, Japan, 113-8655
        • Research Site
      • Iruma-Gun, Japan, 350-0495
        • Research Site
      • Osaka, Japan, 565-0871
        • Research Site
  • Korea, Republic of
      • Jung-gu, Korea, Republic of, 22332
        • Research Site
      • Seoul, Korea, Republic of, 03080
        • Research Site
      • Seoul, Korea, Republic of, 02447
        • Research Site
      • Seoul, Korea, Republic of, 04763
        • Research Site
  • Poland
      • Warszawa, Poland, 02-637
        • Research Site
  • Spain
      • Barcelona, Spain, 08035
        • Research Site
      • Barcelona, Spain, 08036
        • Research Site
      • Bilbao (Vizcaya), Spain, 48013
        • Research Site
      • L'Hospitalet de Llobregat, Spain, 08907
        • Research Site
      • Madrid, Spain, 28034
        • Research Site
      • Madrid, Spain, 28041
        • Research Site
  • United Kingdom
      • Liverpool, United Kingdom, L9 7AL
        • Research Site
      • London, United Kingdom, SE5 9RS
        • Research Site
      • Salford, United Kingdom, M6 8HD
        • Research Site
  • United States
    • California
      • Irvine, California, United States, 92617
        • Research Site
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Research Site
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Research Site
    • Kansas
      • Fairway, Kansas, United States, 66205
        • Research Site
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Research Site
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 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 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / 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:
  • Ultomiris
  • ALXN1210
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part A: IMACS-TIS (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand
Time Frame: Week 26
Week 26
Part B: IMACS-TIS (TIS40) response at Week 50 of the Randomized Controlled Period as per defined composite estimand
Time Frame: Week 50
Week 50

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Part A: TIS At Week 26
Time Frame: Week 26
Week 26
Part A: Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 26
Time Frame: Baseline, Week 26
Baseline, Week 26
Part A: Response Related to Muscle Enzymes: Normalization Of Most Abnormal Baseline Enzyme At Week 26
Time Frame: Week 26
Week 26
Part A: Change From Baseline In Five IMACS Core Set Measures (extra-muscular disease activity based on MDAAT, physician global activity assessment, patient global activity assessment, MMT-8, HAQ) At Week 26
Time Frame: Baseline, Week 26
Baseline, Week 26
Part A: Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response At Week 26
Time Frame: Baseline, Week 26
Baseline, Week 26
Part A: TIS20 Response at Week 26
Time Frame: Week 26
Week 26
Part A: TIS60 Response at Week 26
Time Frame: Week 26
Week 26
Part A: Time To First Response Of TIS20, TIS40, Or TIS60
Time Frame: Baseline through Week 26
Baseline through Week 26
Part A: Clinical Worsening During Randomized Controlled Period At Two Consecutive Visits
Time Frame: Baseline through Week 26
Baseline through Week 26
Part A: Receipt of Rescue Therapy
Time Frame: Baseline through Week 26
Baseline through Week 26
Part B: TIS At Week 50
Time Frame: Week 50
Week 50
Part B: Change From Baseline In Five IMACS Core Set Measures (extra-muscular disease activity based on MDAAT, physician global activity assessment, patient global activity assessment, MMT-8, HAQ) At Week 50
Time Frame: Baseline, Week 50
Baseline, Week 50
Part B: Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 50
Time Frame: Baseline, Week 50
Baseline, Week 50
Part B: Response Related to Muscle Enzymes: Normalization Of Most Abnormal Baseline Enzyme At Week 50
Time Frame: Week 50
Week 50
Part B: Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response At Week 50
Time Frame: Week 50
Week 50
Part B: TIS20 Response at Week 50
Time Frame: Week 50
Week 50
Part B: TIS60 Response at Week 50
Time Frame: Week 50
Week 50
Part B: Time To First Response Of TIS20, TIS40, Or TIS60
Time Frame: Baseline through Week 50
Baseline through Week 50
Part B: Clinical Worsening During Randomized Controlled Period At Two Consecutive Visits
Time Frame: Baseline through Week 50
Baseline through Week 50
Part B: Receipt of Rescue Therapy
Time Frame: Baseline through Week 50
Baseline through Week 50
Part A: CDASI Response (7-point improvement) at Week 26
Time Frame: Baseline, Week 26
Minimally clinically important differences (MCID) = 7-point improvement.
Baseline, Week 26
Part B: CDASI Response (7-point improvement) at Week 50
Time Frame: Baseline, Week 50
Minimally clinically important differences (MCID) = 7-point improvement.
Baseline, Week 50

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 19, 2021

Primary Completion (Actual)

October 26, 2023

Study Completion (Estimated)

May 8, 2024

Study Registration Dates

First Submitted

August 4, 2021

First Submitted That Met QC Criteria

August 4, 2021

First Posted (Actual)

August 10, 2021

Study Record Updates

Last Update Posted (Estimated)

May 10, 2024

Last Update Submitted That Met QC Criteria

May 9, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN1210-DM-310
  • 2021-001200-15 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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