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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 30, 2022
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE August 4, 2021
First Posted Date  ICMJE August 10, 2021
Last Update Posted Date June 30, 2022
Actual Study Start Date  ICMJE December 2, 2021
Estimated Primary Completion Date August 31, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 4, 2021)
  • 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 ]
  • Part B: Proportion Of Participants With A ≥ 20-point Improvement Response On TIS20 At Week 50 [ Time Frame: Week 50 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 2, 2021)
  • Part A: Mean TIS At Week 26 [ Time Frame: Week 26 ]
  • 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 ]
  • Part A: Change From Baseline Of Each Of The IMACS Core Set Measures At Week 26 [ Time Frame: Baseline, Week 26 ]
  • Part A: Time To First CDASI Activity Score Improvement [ Time Frame: Baseline through Week 26 ]
    Minimally clinically important differences (MCID) = 7-point improvement.
  • Part A: Proportion Of Participants With CDASI MCID Improvement At Week 26 [ Time Frame: Week 26 ]
  • Part A: Change In Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) At Week 26 [ Time Frame: Baseline, Week 26 ]
  • Part A: Proportion Of Participants With TIS20 At Each Visit [ Time Frame: Baseline through Week 26 ]
  • Part A: Proportion Of Participants With A ≥ 40-point Improvement Response On IMACS-TIS (TIS40) At Each Visit [ Time Frame: Baseline through Week 26 ]
  • Part A: Proportion Of Participants With A ≥ 60-point Improvement Response On IMACS-TIS (TIS60) At Each Visit [ Time Frame: Baseline through Week 26 ]
  • Part A: Time To First Response Of TIS20, TIS40, Or TIS60 [ Time Frame: Baseline through Week 26 ]
  • Part A: Time To First IMACS Myositis Core Set Measure Improvements [ Time Frame: Baseline through Week 26 ]
  • Part B: Mean TIS At Week 50 [ Time Frame: Week 50 ]
  • Part B: Mean Change From Baseline In Manual Muscle Testing Subset Of 8 Muscles (MMT-8) At Week 50 [ Time Frame: Baseline, Week 50 ]
  • 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 ]
  • Part B: Mean Change From Baseline In CDASI Activity Score At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Patient Global Activity Assessment At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Physician Global Activity Assessment At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Health Assessment Questionnaire (HAQ) At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Muscle Enzyme Values At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean TIS At Each Visit From Week 2 Through Week 50 [ Time Frame: Week 2 through Week 50 ]
  • Part B: Proportion Of Participants With TIS20 At Each Visit [ Time Frame: Baseline through Week 50 ]
  • Part B: Proportion Of Participants With TIS40 At Each Visit [ Time Frame: Baseline through Week 50 ]
  • Part B: Proportion Of Participants With TIS60 At Each Visit [ Time Frame: Baseline through Week 50 ]
  • Part B: Time To First Response Of TIS20, TIS40, Or TIS60 [ Time Frame: Baseline through Week 50 ]
  • Part B: Time To First IMACS Myositis Core Set Measure Improvements [ Time Frame: Baseline through Week 50 ]
  • Part B: Time To First CDASI Activity Score Improvement [ Time Frame: Baseline through Week 50 ]
    MCID = 7-point improvement
  • Part B: Proportion Of Participants With CDASI MCID Improvement At Week 50 [ Time Frame: Week 50 ]
  • Part B: Change In CDA-IGA At Week 50 [ Time Frame: Week 50 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: August 4, 2021)
  • Part A: Mean TIS At Week 26 [ Time Frame: Week 26 ]
  • 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 ]
  • Part A: Change From Baseline Of Each Of The IMACS Core Set Measures At Week 26 [ Time Frame: Baseline, Week 26 ]
  • Part A: Time To First CDASI Activity Score Improvement [ Time Frame: Baseline through Week 26 ]
    Minimally clinically important differences (MCID) = 7-point improvement.
  • Part A: Proportion Of Participants With CDASI MCID Improvement At Week 26 [ Time Frame: Week 26 ]
  • Part A: Change In Investigator Global Assessment Scale For Skin Activity (IGA-SA) At Week 26 [ Time Frame: Baseline, Week 26 ]
  • Part A: Proportion Of Participants With TIS20 At Each Visit [ Time Frame: Baseline through Week 26 ]
  • Part A: Proportion Of Participants With A ≥ 40-point Improvement Response On IMACS-TIS (TIS40) At Each Visit [ Time Frame: Baseline through Week 26 ]
  • Part A: Proportion Of Participants With A ≥ 60-point Improvement Response On IMACS-TIS (TIS60) At Each Visit [ Time Frame: Baseline through Week 26 ]
  • Part A: Time To First Response Of TIS20, TIS40, Or TIS60 [ Time Frame: Baseline through Week 26 ]
  • Part A: Time To First IMACS Myositis Core Set Measure Improvements [ Time Frame: Baseline through Week 26 ]
  • Part B: Mean TIS At Week 50 [ Time Frame: Week 50 ]
  • Part B: Mean Change From Baseline In Manual Muscle Testing Subset Of 8 Muscles (MMT-8) At Week 50 [ Time Frame: Baseline, Week 50 ]
  • 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 ]
  • Part B: Mean Change From Baseline In CDASI Activity Score At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Patient Global Activity Assessment At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Physician Global Activity Assessment At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Health Assessment Questionnaire (HAQ) At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean Change From Baseline In Muscle Enzyme Values At Week 50 [ Time Frame: Baseline, Week 50 ]
  • Part B: Mean TIS At Each Visit From Week 2 Through Week 50 [ Time Frame: Week 2 through Week 50 ]
  • Part B: Proportion Of Participants With TIS20 At Each Visit [ Time Frame: Baseline through Week 50 ]
  • Part B: Proportion Of Participants With TIS40 At Each Visit [ Time Frame: Baseline through Week 50 ]
  • Part B: Proportion Of Participants With TIS60 At Each Visit [ Time Frame: Baseline through Week 50 ]
  • Part B: Time To First Response Of TIS20, TIS40, Or TIS60 [ Time Frame: Baseline through Week 50 ]
  • Part B: Time To First IMACS Myositis Core Set Measure Improvements [ Time Frame: Baseline through Week 50 ]
  • Part B: Time To First CDASI Activity Score Improvement [ Time Frame: Baseline through Week 50 ]
    MCID = 7-point improvement
  • Part B: Proportion Of Participants With CDASI MCID Improvement At Week 50 [ Time Frame: Week 50 ]
  • Part B: Change In IGA-SA At Week 50 [ Time Frame: Week 50 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
Official Title  ICMJE 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
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).
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Dermatomyositis
Intervention  ICMJE
  • 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
  • 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.
Study Arms  ICMJE
  • Experimental: Ravulizumab
    Participants will receive ravulizumab in both Parts A and B.
    Intervention: Drug: Ravulizumab
  • Placebo Comparator: Placebo
    Participants will receive placebo in both Parts A and B.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 4, 2021)
180
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 31, 2024
Estimated Primary Completion Date August 31, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Alexion Pharmaceuticals, Inc. 1-855-752-2356 clinicaltrials@alexion.com
Listed Location Countries  ICMJE France,   Germany,   Italy,   Japan,   Korea, Republic of,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04999020
Other Study ID Numbers  ICMJE ALXN1210-DM-310
2021-001200-15 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Current Responsible Party Alexion Pharmaceuticals
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Alexion Pharmaceuticals
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date June 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP