Ravulizumab for Dermatomyositis

Recruiting at 66 trial locations

Overseen By Alexion Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Alexion Pharmaceuticals, Inc.

Must be taking: Systemic corticosteroids

Disqualifiers: Cancer, HIV, Active infection, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

38 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests ravulizumab, a medication that helps manage immune system-related conditions, in adults with dermatomyositis. The drug aims to reduce inflammation and prevent muscle and skin damage by calming the immune system.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, it mentions that participants should have an inadequate response or intolerance to certain treatments, which suggests you might continue some medications. It's best to discuss this with the trial team.

How is the drug Ravulizumab unique for treating dermatomyositis?

Ravulizumab is unique because it is a long-acting medication that works by inhibiting a part of the immune system called the complement system, which is different from other treatments like prednisone or rituximab that target different immune pathways. This mechanism may offer a new approach for managing dermatomyositis, especially for patients who do not respond well to existing therapies. ^{1 2 3 4 5}

Eligibility Criteria

Adults with dermatomyositis who've had a poor response or can't tolerate at least one standard treatment, weigh over 30 kg, and are vaccinated against Neisseria meningitidis. Excludes those with recent cancer, certain infections, fever before the trial starts, HIV infection, severe muscle damage as per investigator's judgment.

Inclusion Criteria

I weigh at least 30 kilograms.

I have tried 2 or more treatments for my condition without success or could not tolerate them.

You have been diagnosed with dermatomyositis (DM) based on specific guidelines from medical organizations.

Exclusion Criteria

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)

I am HIV positive.

I have not had a serious infection in the last 14 days.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Randomized Controlled Period

Participants receive either ravulizumab or placebo to evaluate efficacy and safety

26 weeks

Regular visits as per study protocol

Open-Label Extension

Participants may continue to receive ravulizumab in an open-label setting

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Placebo
• Ravulizumab

Trial Overview The study is testing Ravulizumab versus a placebo in adults with dermatomyositis to assess its effectiveness and safety. Participants will be randomly assigned to receive either the drug or a placebo without knowing which they're getting (double-blind).

Participant Groups

Group I: Ravulizumab Experimental Treatment 1 Intervention

Participants will receive ravulizumab in both Parts A and B.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo in both Parts A and B.

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Alexion Pharmaceuticals

Lead Sponsor

Trials

231

Recruited

36,700+

Marc Dunoyer

Alexion Pharmaceuticals

Chief Executive Officer since 2021

B.A. in Psychology from the University of New Hampshire

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD

Alexion

Lead Sponsor

Trials

247

Recruited

38,600+

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

Christophe Hotermans

Alexion

Chief Medical Officer since 2021

MD, University of Leuven

Findings from Research

In a 6-month clinical trial involving 36 patients with refractory dermatomyositis and polymyositis, tocilizumab was found to be safe and well tolerated, with no significant safety concerns reported.

However, tocilizumab did not show any significant efficacy compared to placebo in improving disease symptoms, as measured by the Total Improvement Score and other secondary outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized Trial of Tocilizumab in the Treatment of Refractory Adult Polymyositis and Dermatomyositis. Oddis, CV., Rockette, HE., Zhu, L., et al.[2022]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Anti-melanoma differentiation-associated gene 5 (MDA5) antibody-positive dermatomyositis responds to rituximab therapy. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

Chlorambucil. An effective corticosteroid-sparing agent for patients with recalcitrant dermatomyositis. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Randomized Trial of Tocilizumab in the Treatment of Refractory Adult Polymyositis and Dermatomyositis. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Treatment of refractory anti-melanoma differentiation-associated gene 5 anbibody-positive dermatomyositis complicated by rapidly progressing interstitial pulmonary disease: Two case reports. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Current treatment of the inflammatory myopathies. [2019]