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Clinical Trials/NCT04999020
NCT04999020TerminatedPhase 2

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

Alexion Pharmaceuticals, Inc.1 site in 1 country38 target enrollmentStarted: November 19, 2021Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Terminated
Enrollment
38
Locations
1
Primary Endpoint
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
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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 Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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.

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
  • •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.

Arms & Interventions

Ravulizumab

Experimental

Participants will receive ravulizumab in both Parts A and B.

Intervention: Ravulizumab (Drug)

Placebo

Placebo Comparator

Participants will receive placebo in both Parts A and B.

Intervention: Placebo (Drug)

Outcomes

Primary Outcomes

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

Time Frame: Week 26

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.

Secondary Outcomes

  • 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)
  • Change From Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26(Baseline, Week 26)
  • Change From Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26(Baseline, Week 26)
  • Change From Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26(Baseline, Week 26)
  • Number of Participants With CDASI Response (>=7-point Improvement) at Week 26(Week 26)
  • Number of Participants With Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26(Week 26)
  • Number of Participants With ≥ 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26(Week 26)
  • Number of Participants With ≥ 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26(Week 26)
  • Time to First Response of TIS20, TIS40, or TIS60(Baseline through Week 26)
  • Number of Participants With Clinical Worsening (CW) During the RCP At 2 Consecutive Visits(Baseline through Week 26)
  • Number of Participants Who Received Acute Rescue Therapy With Standard DM Treatment(Baseline through Week 26)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (1)

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