Daxdilimab for Inflammatory Myositis

No longer recruiting at 40 trial locations

Overseen By Amgen Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Amgen

Disqualifiers: Cancer, Cardiac disease, HIV, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called daxdilimab for individuals with inflammatory myositis, a condition that causes muscle inflammation and weakness. The main goal is to determine if daxdilimab can reduce disease activity, improve skin symptoms, and decrease the need for steroid medications. The trial compares the effects of daxdilimab to a placebo (a substance with no active medication). It seeks participants diagnosed with myositis who are experiencing active symptoms, such as muscle weakness and skin issues. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it mentions that participants should be on stable standard of care therapy if tolerated. If you cannot tolerate it or have failed standard care, a washout period (time without taking certain medications) is required.

Is there any evidence suggesting that daxdilimab is likely to be safe for humans?

Research has shown that daxdilimab is under study to determine its safety for treating inflammatory muscle conditions. Daxdilimab has undergone previous testing, and researchers are now assessing its tolerability. Although specific results from earlier studies aren't mentioned, daxdilimab's progression to a Phase 2 trial indicates that initial safety tests in humans have been promising.

In the early stages, treatments are typically tested for safety in small groups. By Phase 2, researchers have gathered enough evidence to test the treatment in larger groups, focusing on its efficacy and side effects. This suggests that daxdilimab has demonstrated a reasonable safety profile so far.

Researchers are closely monitoring for any side effects or unexpected issues. Participants can expect careful oversight to ensure their safety during the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Daxdilimab is unique because it targets a specific immune system component called the CD40 ligand, which plays a significant role in inflammatory myositis. Unlike standard treatments like corticosteroids and immunosuppressants, which broadly suppress the immune system, daxdilimab offers a more targeted approach that could reduce side effects. Additionally, it is administered via subcutaneous injection, which might be more convenient compared to intravenous treatments. Researchers are excited about daxdilimab because it has the potential to provide a more precise treatment option with potentially fewer side effects for people with inflammatory myositis.

What evidence suggests that daxdilimab might be an effective treatment for inflammatory myositis?

Research suggests that daxdilimab, which participants in this trial may receive, can help reduce symptoms for people with inflammatory myositis. Studies have shown it might also improve skin issues and lower the need for corticosteroids, often used to manage inflammation. Daxdilimab targets the immune system to lessen inflammation, a major problem in myositis. While early results are promising, more research is needed to confirm its effectiveness. ^{1 2 4 5 6}

Who Is on the Research Team?

MD

Principal Investigator

Amgen

Are You a Good Fit for This Trial?

This trial is for adults aged 18-75 with a diagnosis of dermatomyositis (DM) or anti-synthetase inflammatory myositis (ASIM), meeting specific criteria like muscle weakness and elevated muscle enzymes. Participants must not have severe infections, other connective tissue diseases that affect assessment, significant organ damage, or be pregnant. They should agree to potentially taper off corticosteroids.

Inclusion Criteria

Diagnosis of definite or probable myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria

I am currently experiencing active muscle inflammation.

I am willing to adjust my corticosteroid dose as per the study's guidelines if my condition is stable or getting better.

Exclusion Criteria

I have myositis with another connective tissue disease, making treatment response hard to assess.

I have had significant heart problems in the last 6 months.

Severe herpes virus family infection at any time prior to randomization

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daxdilimab or placebo by subcutaneous injection during the 24-week treatment period

24 weeks

Safety Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

Open-label Extension (optional)

Participants could enter an open-label extension period from weeks 24-48 to receive daxdilimab

24 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Daxdilimab
• Placebo

Trial Overview The study tests Daxdilimab's effectiveness compared to a placebo in reducing disease activity by week 24. It also looks at its impact on skin symptoms and the possibility of lowering corticosteroid use. Additionally, it will assess how the body processes the drug and any potential immune response to it.

How Is the Trial Designed?

Group I: Daxdilimab Experimental Treatment 1 Intervention

Daxdilimab will be administered by subcutaneous (SC) injection during the 24-week treatment period followed by an 8-week safety follow up. Prior to amendment 2 participants could enter an open-label extension period from weeks 24-48. For those participants already in the open-label extension, they will stop dosing and enter the safety follow up.

Group II: Placebo Placebo Group 2 Interventions

Matching placebo will be administered by SC injection during the 24-week treatment period followed by an 8-week safety follow up. Prior to amendment 2 participants could enter an open-label extension period from weeks 24-48 and receive daxdilimab. For those participants already in the open-label extension, they will stop dosing and enter the safety follow up.

Who Is Running the Clinical Trial?

Amgen

Lead Sponsor

Trials

1,508

Recruited

1,433,000+

Founded

1980

Headquarters

Thousand Oaks, USA

Known For

Human Therapeutics

Top Products

Enbrel, Prolia, Neulasta, Otezla

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Horizon Therapeutics Ireland DAC

Lead Sponsor

Trials

21

Recruited

2,500+

Published Research Related to This Trial

Tofacitinib was found to be effective in improving skin symptoms in patients with classic dermatomyositis (DM) and amyopathic DM, with significant cutaneous improvement reported in those who continued treatment.

However, the drug showed minimal benefit in improving muscle strength across all myositis types, and over half of the patients discontinued treatment due to lack of effectiveness or death.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tofacitinib therapy in refractory inflammatory myositis: a retrospective cohort study of 41 patients. Beckett, M., Tan, J., Bonnardeaux, E., et al.[2023]

In a study of 52 anti-MDA5-positive dermatomyositis patients at Johns Hopkins Myositis Centre, approximately 18% achieved long-term drug-free remission after a median disease duration of 4 years.

The study found no specific demographic or disease characteristics that predicted remission, indicating that the factors leading to remission in these patients remain unclear.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anti-MDA5-positive dermatomyositis and remission in a single referral centre population. Tiniakou, E., Mecoli, CA., Kelly, W., et al.[2023]

In a 12-week open-label study involving 10 subjects with treatment-refractory dermatomyositis, tofacitinib demonstrated strong clinical efficacy, with all participants meeting the primary outcome of improved disease activity.

Half of the subjects showed moderate improvement, while the other half had minimal improvement, and significant reductions in disease severity were observed, particularly in skin symptoms, as indicated by a notable decrease in the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) score.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. Paik, JJ., Casciola-Rosen, L., Shin, JY., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05669014

Study Details | NCT05669014 | A Phase 2 Proof of Concept ... The primary efficacy objective: To evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24.

2. isrctn.com isrctn.com/ISRCTN44828082

A phase II proof of concept study to evaluate the efficacy ... This study is being done to know how well a drug called daxdilimab works and how safe it is in participants with DM or ASIM for whom the usual treatment does ...

3. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05669014/a-phase-2-proof-of-concept-study-to-evaluate-the-efficacy-and-safety-of-daxdilimab-in-participants-with-dermatomyositis-dm-or-anti-synthetase-inflammatory-myositis-asim

A Phase 2 Proof of Concept Study to Evaluate the Efficacy ... The primary efficacy objective: To evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24.

4. tipranks.com tipranks.com/news/company-announcements/amgens-phase-2-study-on-daxdilimab-a-potential-game-changer-for-inflammatory-myositis

Amgen's Phase 2 Study on Daxdilimab: A Potential Game ... The study aimed to assess the efficacy and safety of daxdilimab in reducing disease activity and skin symptoms, as well as its impact on corticosteroid use.

5. myositis.org myositis.org/wp-content/uploads/2024/03/2023-Current-myositis-clinical-trials-and-tribulations-1.pdf

Current myositis clinical trials and tribulations Patient- reported outcomes and adult patients' disease experience in the idiopathic inflammatory myopathies. report from the OMERACT 11 myositis ...

6. mayo.edu mayo.edu/research/clinical-trials/cls-20556630

Clinical Trials The purpose of this study is to evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24.