Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

Alexion Pharmaceuticals

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Dermatomyositis

Treatments: Drug: Placebo; Drug: Ravulizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT04999020

2021-001200-15 (EudraCT Number)

ALXN1210-DM-310

Details and patient eligibility

About

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).

Full 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.

Enrollment: 38 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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38 participants in 2 patient groups, including a placebo group

Ravulizumab

Experimental group

Description:

Participants will receive ravulizumab in both Parts A and B.

Treatment:

Drug: Ravulizumab

Placebo

Placebo Comparator group

Description:

Participants will receive placebo in both Parts A and B.

Treatment:

Drug: Placebo