Ravulizumab for Dermatomyositis

No longer recruiting at 157 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ravulizumab for people with dermatomyositis, a condition that causes muscle weakness and skin rashes. The study aims to determine if ravulizumab is safe and effective compared to a placebo (a substance with no active treatment). Participants will receive either ravulizumab or the placebo. It suits adults diagnosed with dermatomyositis who haven’t improved with treatments like steroids or immunosuppressants. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important early findings.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ravulizumab is generally safe for people. Studies have found that headaches and mild colds are the most common side effects, while serious side effects are rare. Ravulizumab has already been approved for other uses, indicating that its safety is well understood. However, since everyone is different, it is important to consider this information and consult a doctor before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for dermatomyositis?

Ravulizumab is unique because it offers a novel approach to treating dermatomyositis by targeting the complement system, specifically the C5 protein. Unlike traditional treatments like corticosteroids and immunosuppressants, which broadly dampen the immune response, Ravulizumab precisely blocks a part of the immune system involved in inflammation. This targeted action means it may reduce symptoms more effectively while potentially minimizing side effects. Researchers are excited about Ravulizumab because it represents a more focused and possibly safer treatment option for patients suffering from this challenging condition.

What evidence suggests that ravulizumab might be an effective treatment for dermatomyositis?

Research shows that ravulizumab blocks a part of the immune system called the complement system, which can be overly active in some diseases. Studies with patients who have conditions like PNH, a rare blood disorder, demonstrated that ravulizumab provides strong and lasting benefits. It controlled symptoms better than eculizumab, a similar treatment, over a year. In this trial, some participants will receive ravulizumab to assess its potential in managing dermatomyositis by reducing muscle inflammation and improving symptoms. Ongoing trials will provide more specific information about its effectiveness for this condition. ^{1 3 5 6 7}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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).

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04999020

NCT04999020 | Ravulizumab Versus Placebo in Adult ... This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0003496724297132

AB1287 DESIGN OF A GLOBAL PHASE 2/3 ... Treatment with the long-acting anti-C5 monoclonal antibody ravulizumab is associated with immediate, complete, and sustained inhibition of the complement ...

3. ultomirishcp.com ultomirishcp.com/pnh/efficacy-and-safety/adult-data

PNH | Adult data - ULTOMIRIS® (ravulizumab-cwvz) In clinical trials of adult patients with PNH, ULTOMIRIS showed robust & sustained efficacy across all endpoints vs. eculizumab, through 1+ year of ...

4. uat-alexion-engage.app-trialscope.com uat-alexion-engage.app-trialscope.com/study/?id=ALXN1210-DM-310

Study details - Alexion Clinical Trial Transparency Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis. A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, ...

5. trials.arthritis.org trials.arthritis.org/trials/NCT04999020

Ravulizumab Versus Placebo in Adult Participants With ... This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, ...

6. ard.bmj.com ard.bmj.com/content/81/Suppl_1/1750.3

Abstract Safety will be assessed by analyzing the incidence of treatment-related adverse events (TEAEs), including those classified as serious and/or leading to ...

7. ctv.veeva.com ctv.veeva.com/study/ravulizumab-versus-placebo-in-adult-participants-with-dermatomyositis

Ravulizumab Versus Placebo in Adult Participants With ... This is a Phase 2/3, double-blind, randomized, placebo-controlled, parallel group, multicenter study to evaluate the efficacy, safety, pharmacokinetics, ...