Dermatomyositis MedDRA version: 20.0 Level: PT Classification code 10012503 Term: Dermatomyositis System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for injection
INN or Proposed INN: RAVULIZUMAB
CAS Number: 1803171-55-2
Other desc
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all of the following criteria apply: 1. 18 years of age or older at the time of signing the informed consent. 2.Body weight = 30 kg at the time of Screening. 3. Male or female. 4. Diagnosis: Meet American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification criteria for definite or probable DM (Lundberg, 2017). 5. Participants who have an inadequate response (ie, continued impairment by medical doctor report) or are intolerant to 1 or more DM treatments, including systemic corticosteroids or ISTs (eg, azathioprine, methotrexate, rituximab, IVIg), either in combination or as monotherapy. 6. Vaccinated against N 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. 7. Female participants of childbearing potential and male participants must follow specified contraception guidance as described in the protocol. Detailed inclusion criteria are mentioned in protocol. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 150 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Cancer-associated myositis, defined as the diagnosis of myositis within 2 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 for at least 3 months before Screening). 2. Evidence of active malignant disease or malignancies diagnosed within the previous 3 years including hematological malignancies and solid tumors (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured for at least 3 months before Screening). 3. Participants with other forms of myositis 4. As per Investigator's discretion, participants with significant muscle damage (eg, severe muscle atrophy, end stage muscle disease). 5. History of Neisseria meningitidis infection. 6. Human immunodeficiency virus (HIV) infection (evidenced by HIV Type 1 or Type 2 antibody titer). 7. Active systemic bacterial, viral, or fungal infection within 14 days prior to ravulizumab administration. 8. Presence of fever = 38°C (100.4°F) within 7 days prior to study drug administration on Day 1. 9. History of hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab. 10. Pregnant, breastfeeding, or intending to conceive during the course of the study. 11. Inability or unwillingness to adhere to the protocol requirements Detailed exclusion criteria are mentioned in protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: PART A To determine the effect of ravulizumab compared with placebo in the treatment of DM based on improvement in Total Improvement Score (TIS) IMACS - TIS. PART B To determine the effect of ravulizumab compared with placebo in the treatment of DM based on improvement in Total Improvement Score (TIS) IMACS - TIS.;Secondary Objective: PART A To assess the efficacy of ravulizumab compared with placebo in the treatment of DM based on improvement in efficacy endpoints. To characterize the PK/PD and immunogenicity of ravulizumab in adult participants with DM. To characterize the overall safety of ravulizumab in participants with DM. PART B To assess the efficacy of ravulizumab compared with placebo in the treatment of DM based on improvement in components of IMACS and other myositis activity measures To assess the efficacy of ravulizumab compared with placebo in the treatment of DM based on other efficacy endpoints To characterize the PK/PD and immunogenicity of ravulizumab in adult participants with DM To characterize the overall safety of ravulizumab in participants with DM;Primary end point(s): PART A IMACS-TIS (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand. PART B IMACS-TIS (TIS40) response at Week 50 of the Randomized Controlled as per defined composite estimand. ;Timepoint(s) of evaluation of this end point: Part A: Week 26 Part B: Week 50 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): PART A • Mean TIS at Week 26 • Mean change from baseline in CDASI Activity Score at Week 26 • Change from baseline of IMACS core set measures at Week 26 • CDASI response (7-point improvement from baseline) at Week 26. • CDA-IGA response (almost clear or clear) at Week 26. PART B • Mean TIS at Week 50 • Mean change from baseline in MMT-8 at Week 50 • Mean change from baseline in extra-muscular disease activity based on MDAAT at Week 50 • Mean change from baseline in CDASI Activity Score at Week 50 ;Timepoint(s) of evaluation of this end point: Part A: Week 26 Part B: Week 50 | — |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Mexico, Peru, Poland, Serbia, Spain, Sweden, Taiwan, Türkiye, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed