Daxdilimab for Inflammatory Myositis

Recruiting at 37 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

12 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing daxdilimab, a new medicine, to see if it can help patients with specific muscle and skin diseases by reducing inflammation and symptoms. The study focuses on patients with dermatomyositis or anti-synthetase inflammatory myositis.

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.

How is the drug Daxdilimab different from other treatments for inflammatory myositis?

Daxdilimab is unique because it is a novel treatment option for inflammatory myositis, a condition with limited standard treatment guidelines. Unlike other treatments, Daxdilimab may offer a new mechanism of action, potentially targeting the disease differently than existing therapies like tofacitinib, which is a JAK inhibitor used in refractory cases. ^{1 2 3 4 5}

Research Team

MD

Principal Investigator

Amgen

Eligibility Criteria

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 between 18 and 75 years old.

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Tofacitinib therapy in refractory inflammatory myositis: a retrospective cohort study of 41 patients. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Treatment strategies and safety of rechallenge in the setting of immune checkpoint inhibitors-related myositis: a national multicentre study. [2021]

3. Italy pubmed.ncbi.nlm.nih.gov

Developing standardised treatment for adults with myositis and different phenotypes: an international survey of current prescribing preferences. [2021]

4. Italy pubmed.ncbi.nlm.nih.gov

Anti-MDA5-positive dermatomyositis and remission in a single referral centre population. [2023]

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

Study of Tofacitinib in Refractory Dermatomyositis: An Open-Label Pilot Study of Ten Patients. [2022]