Dermatomyositis Clinical Trial
Official 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
Verified date | October 2023 |
Source | Alexion Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Active, not recruiting |
Enrollment | 150 |
Est. completion date | May 31, 2028 |
Est. primary completion date | November 30, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
France | Clinical Trial Site | Lille Cedex | |
France | Clinical Trial Site | Paris | |
France | Clinical Trial Site | Strasbourg | |
France | Clinical Trial Site | Toulouse Cedex 9 | |
Germany | Clinical Trial Site | Dusseldorf | |
Germany | Clinical Trial Site | Erlangen | |
Germany | Clinical Trial Site | Essen | |
Germany | Clinical Trial Site | Freiburg | |
Germany | Clinical Trial Site | Gottingen | |
Germany | Clinical Trial Site | Halle | Sachsen-Anhalt |
Italy | Clinical Trial Site | Bari | BA |
Italy | Clinical Trial Site | Brescia | BS |
Italy | Clinical Trial Site | Pavia | PV |
Italy | Clinical Trial Site | Pisa | PI |
Italy | Clinical Trial Site | Roma | |
Italy | Clinical Trial Site | Torino | Piedmont |
Japan | Clinical Trial Site | Bunkyo-ku | Tokyo |
Japan | Clinical Trial Site | Iruma-Gun | |
Japan | Clinical Trial Site | Suita-Shi | |
Korea, Republic of | Clinical Trial Site | Incheon | |
Korea, Republic of | Clinical Trial Site | Seoul | |
Korea, Republic of | Clinical Trial Site | Seoul | |
Korea, Republic of | Clinical Trial Site | Seoul | |
Korea, Republic of | Clinical Trial Site | Seoul | |
Spain | Clinical Trial Site | Barcelona | |
Spain | Clinical Trial Site | Barcelona | |
Spain | Clinical Trial Site | Bilbao | Vizcaya |
Spain | Clinical Trial Site | L'Hospitalet de Llobregat | Barcelona |
Spain | Clinical Trial Site | Madrid | |
Spain | Clinical Trial Site | Madrid | |
United Kingdom | Clinical Trial Site | Edinburgh | |
United Kingdom | Clinical Trial Site | Liverpool | |
United Kingdom | Clinical Trial Site | London | |
United Kingdom | Clinical Trial Site | Salford | |
United Kingdom | Clinical Trial Site | West Bromwich | |
United States | Clinical Trial Site | Atlanta | Georgia |
United States | Clinical Trial Site | Baltimore | Maryland |
United States | Clinical Trial Site | Chicago | Illinois |
United States | Clinical Trial Site | Cleveland | Ohio |
United States | Clinical Trial Site | Kansas City | Kansas |
United States | Clinical Trial Site | Los Angeles | California |
United States | Clinical Trial Site | Mesa | Arizona |
United States | Clinical Trial Site | New York | New York |
United States | Clinical Trial Site | Orange | California |
United States | Clinical Trial Site | Pittsburgh | Pennsylvania |
United States | Clinical Trial Site | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals, Inc. |
United States, France, Germany, Italy, Japan, Korea, Republic of, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Part A: IMACS-TIS (TIS40) response at Week 26 of the Randomized Controlled Period as per defined composite estimand | Week 26 | ||
Primary | Part B: IMACS-TIS (TIS40) response at Week 50 of the Randomized Controlled Period as per defined composite estimand | Week 50 | ||
Secondary | Part A: TIS At Week 26 | Week 26 | ||
Secondary | Part A: Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 26 | Baseline, Week 26 | ||
Secondary | Part A: Response Related to Muscle Enzymes: Normalization Of Most Abnormal Baseline Enzyme At Week 26 | Week 26 | ||
Secondary | Part A: Change From Baseline In Five IMACS Core Set Measures (extra-muscular disease activity based on MDAAT, physician global activity assessment, patient global activity assessment, MMT-8, HAQ) At Week 26 | Baseline, Week 26 | ||
Secondary | Part A: CDASI Response (7-point improvement) at Week 26 | Minimally clinically important differences (MCID) = 7-point improvement. | Baseline through Week 26 | |
Secondary | Part A: Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response At Week 26 | Baseline, Week 26 | ||
Secondary | Part A: TIS20 Response at Week 26 | Week 26 | ||
Secondary | Part A: TIS60 Response at Week 26 | Week 26 | ||
Secondary | Part A: Time To First Response Of TIS20, TIS40, Or TIS60 | Baseline through Week 26 | ||
Secondary | Part A: Clinical Worsening During Randomized Controlled Period At Two Consecutive Visits | Baseline through Week 26 | ||
Secondary | Part A: Receipt of Rescue Therapy | Baseline through Week 26 | ||
Secondary | Part B: TIS At Week 50 | Week 50 | ||
Secondary | Part B: Change From Baseline In Five IMACS Core Set Measures (extra-muscular disease activity based on MDAAT, physician global activity assessment, patient global activity assessment, MMT-8, HAQ) At Week 50 | Baseline, Week 50 | ||
Secondary | Part B: Change From Baseline In Cutaneous Dermatomyositis Disease Area And Severity Index (CDASI) Activity Score At Week 50 | Baseline, Week 50 | ||
Secondary | Part B: Response Related to Muscle Enzymes: Normalization Of Most Abnormal Baseline Enzyme At Week 50 | Week 50 | ||
Secondary | Part B: CDASI Response (7-point improvement) at Week 50 | Minimally clinically important differences (MCID) = 7-point improvement. | Baseline through Week 50 | |
Secondary | Part B: Cutaneous Dermatomyositis Activity Physician's Global Assessment (CDA-IGA) Response At Week 50 | Week 50 | ||
Secondary | Part B: TIS20 Response at Week 50 | Week 50 | ||
Secondary | Part B: TIS60 Response at Week 50 | Week 50 | ||
Secondary | Part B: Time To First Response Of TIS20, TIS40, Or TIS60 | Baseline through Week 50 | ||
Secondary | Part B: Clinical Worsening During Randomized Controlled Period At Two Consecutive Visits | Baseline through Week 50 | ||
Secondary | Part B: Receipt of Rescue Therapy | Baseline through Week 50 |
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