Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04999020
• Other study ID #: ALXN1210-DM-310
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: November 19, 2021
• Est. completion date: May 8, 2024

Study information

• Verified date: April 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 38
• Est. completion date: May 8, 2024
• Est. primary completion date: October 26, 2023
• 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.

Related Conditions & MeSH terms

• Dermatomyositis

Intervention

Drug:
• Ravulizumab
Intravenous dosing will consist of a loading dose followed by maintenance doses administered every 8 weeks (q8w). The maintenance dosing will be initiated 2 weeks after the loading dose is administered.
• Placebo
Intravenous dosing will consist of a loading dose followed by maintenance doses administered q8w. The maintenance dosing will be initiated 2 weeks after the loading dose is administered.

Locations

Country	Name	City	State
France	Research Site	Paris
France	Research Site	Strasbourg
France	Research Site	Toulouse
Germany	Research Site	Erlangen
Germany	Research Site	Essen
Germany	Research Site	Freiburg im Breisgau
Germany	Research Site	Halle
Italy	Research Site	Bari
Italy	Research Site	Brescia
Italy	Research Site	Firenze
Italy	Research Site	Milano
Italy	Research Site	Pavia
Italy	Research Site	Pisa
Italy	Research Site	Roma
Italy	Research Site	Rome
Italy	Research Site	Torino
Japan	Research Site	Bunkyo-ku
Japan	Research Site	Iruma-Gun
Japan	Research Site	Osaka
Korea, Republic of	Research Site	Jung-gu
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Poland	Research Site	Warszawa
Spain	Research Site	Barcelona
Spain	Research Site	Barcelona
Spain	Research Site	Bilbao (Vizcaya)
Spain	Research Site	L'Hospitalet de Llobregat
Spain	Research Site	Madrid
Spain	Research Site	Madrid
United Kingdom	Research Site	Liverpool
United Kingdom	Research Site	London
United Kingdom	Research Site	Salford
United States	Research Site	Atlanta	Georgia
United States	Research Site	Baltimore	Maryland
United States	Research Site	Charleston	South Carolina
United States	Research Site	Chicago	Illinois
United States	Research Site	Fairway	Kansas
United States	Research Site	Irvine	California

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

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

Outcome

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, 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, 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