Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

• Register: ClinicalTrials.gov
• Last refreshed on: 9 December 2024
• Main ID: NCT04999020
• Date of registration: 04/08/2021
• Prospective Registration: Yes
• Primary sponsor: Alexion Pharmaceuticals, Inc.
• Public title: Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis
• Scientific 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
• Date of first enrolment: November 19, 2021
• Target sample size: 38
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT04999020
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2/Phase 3

Countries of recruitment

France, Germany, Italy, Japan, Korea, Republic of, Poland, Spain, United Kingdom, United States

Key inclusion & exclusion 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 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.


Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Dermatomyositis

Intervention(s)

Drug: Placebo

Drug: Ravulizumab

Primary Outcome(s)

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]

Secondary Outcome(s)

Number of Participants With Clinical Worsening (CW) During the RCP At 2 Consecutive Visits [Time Frame: Baseline through Week 26]

Change From Baseline In IMACS CSMs: Health Assessment Questionnaire (HAQ) at Week 26 [Time Frame: Baseline, Week 26]

Change From Baseline In IMACS CSMs: Manual Muscle Testing Subset 8 Muscles (MMT-8) at Week 26 [Time Frame: Baseline, Week 26]

Number of Participants Who Received Acute Rescue Therapy With Standard DM Treatment [Time Frame: Baseline through Week 26]

Number of Participants With Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response at Week 26 [Time Frame: Week 26]

Time to First Response of TIS20, TIS40, or TIS60 [Time Frame: Baseline through Week 26]

Change From Baseline In IMACS CSMs: Physician Global Activity Assessment at Week 26 [Time Frame: Baseline, Week 26]

Number of Participants With = 20-Point Improvement Response on IMACS-TIS (TIS20) Response at Week 26 [Time Frame: Week 26]

Change From Baseline In IMACS CSMs: Extra-Muscular Disease Activity Based on Myositis Disease Activity Assessment Tool (MDAAT) at Week 26 [Time Frame: Baseline, Week 26]

Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score at Week 26 [Time Frame: Baseline, Week 26]

Number of Participants With CDASI Response (>=7-point Improvement) at Week 26 [Time Frame: Week 26]

Change From Baseline In IMACS CSMs: Patient Global Activity Assessment at Week 26 [Time Frame: Baseline, Week 26]

TIS at Week 26 [Time Frame: Week 26]

Number of Participants With = 60-Point Improvement Response on IMACS-TIS (TIS60) Response at Week 26 [Time Frame: Week 26]

Number of Participants With Response Related to Muscle Enzymes: Normalization of Most Abnormal Baseline Enzyme at Week 26 [Time Frame: Baseline, Week 26]

Secondary ID(s)

2021-001200-15

ALXN1210-DM-310

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/12/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04999020