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)

Study Purpose

The primary efficacy objective: To evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24. The secondary efficacy objectives include: 1. To evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24. 2. To evaluate the effect of daxdilimab compared with placebo on skin symptoms at Week 24. 3. To evaluate the effect of daxdilimab on decreasing the use of corticosteroid at Week 24. Other secondary objectives include: 1. To characterize the pharmacokinetics (PK) and immunogenicity of daxdilimab in participants. 2. To evaluate the safety and tolerability of daxdilimab in participants.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.

An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.

Searching Both is inclusive of interventional and observational studies.

Eligible Ages 18 Years - 75 Years
Gender All
More Inclusion & Exclusion Criteria


Inclusion Criteria:

1. Adult men or women 18 and ≤ 75 years of age at the time of signing the informed consent (ICF). 2. A diagnosis of definite or probable myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria: 1. Population 1: DM.
  • - Diagnosis of DM with DM rash current or historical, or.
2. Population 2: ASIM.
  • - Anti-histidyl tRNA synthetase-(Anti-Jo-1) antibodies must be positive during screening by central laboratory testing, or.
  • - One of following antibodies must be positive by historical testing: directed against anti-alanyl- (anti-PL-12), anti-threonyl-(anti PL-7), anti-asparaginyl-(anti-KS), anti-glycyl-(anti-EJ), anti-isoleucyl-(anti-OJ), anti-phenylalanyl-transfer RNA synthetase-(anti-ZO), anti-tyrosil-YRS(HA).
3. Currently active myositis with all the following (a, b, and c) during screening: 1. Manual Muscle Testing (MMT 8) score < 142. 2. At least 2 other abnormal core set measures (CSM) from the following list:
  • - Patient global disease activity (PtGDA) ≥ 2cm in a 10 cm visual analog scale (VAS) - Physician's Global Disease Activity (PhGDA) ≥ 2 cm in a 10 cm VAS.
  • - Extramuscular activity ≥ 2cm in a 10 cm VAS.
  • - At least one muscle enzyme 1.5 times upper limit of normal (ULN) - Health assessment questionnaire-disability index (HAQ-DI) ≥ 0.5.
3. Global muscle damage score ≤ 5 on a 10 cm VAS on the myositis damage index (MDI). 4. Participants should be on stable standard of care therapy if tolerated; if they are not able to tolerate it or have failed standard of care, medications should have a washed out period. 5. Participants should be willing to taper corticosteroid dose per protocol when stable or improving.

Exclusion Criteria:

1. Any condition that, in the opinion of the investigator or sponsor, would interfere with the evaluation of investigational product (IP) or interpretation of participant safety or study results. 2. Weight > 160 kg (352 pounds) at screening. 3. Breastfeeding or pregnant women or women who intend to become pregnant anytime from signing the ICF through 6 months after receiving the last dose of IP. 4. History of clinically meaningful cardiac disease including unstable angina, myocardial infarction, congestive heart failure within 6 months prior to randomization; arrhythmia requiring active therapy, except for clinically insignificant extra systoles, or minor conduction abnormalities; or presence of clinically meaningful abnormality on electrocardiogram (ECG) if, in the opinion of the Investigator, it would increase the risk of study participation. 5. History of cancer within the past 5 years, except as follows:
  • - In situ carcinoma of the cervix treated with apparent success with curative therapy > 12 months prior to screening, or.
  • - Cutaneous basal cell or squamous cell carcinoma treated with curative therapy.
6. Any underlying condition that in the opinion of the Investigator significantly predisposes the participant to infection. 7. Known history of a primary immunodeficiency or an underlying condition, such as known human immunodeficiency virus (HIV) infection, or a positive result for HIV infection per central laboratory. 8. Confirmed positive test for hepatitis B virus serology as defined in the protocol. 9. Active tuberculosis (TB), or a positive interferon gamma (IFN-γ) release assay (IGRA) test at screening, unless documented history of appropriate treatment for active or latent TB according to local guidelines. 10. Any severe herpes virus family infection (including Epstein-Barr virus, cytomegalovirus [CMV]) at any time prior to randomization. 11. Opportunistic infection requiring hospitalization or parenteral antimicrobial treatment within 2 years prior to randomization. 12. Significant organ system involvement or myositis damage (global muscle damage score > 5 on a 10cm VAS scale on the MDI) that poses risks in the study or impedes assessments. 13. Diagnosis of immune-mediated necrotizing myopathy (IMNM) [(positive 3-hydroxy-3-methylglutaryl-coenzyme A reductase (anti-HMGR), anti-signal recognition particle (anti- SRP), or antibody negative)], inclusion body myositis (IBM) (including positive anti-cytosolic 5'-nucleotidase 1A (anti cN1A), or drug-induced myositis. 14. Current musculoskeletal, joint, or inflammatory disease, including significant joint contractures or calcinosis that in the opinion of the investigator, could interfere with the muscle strength assessments and confound the disease activity assessments. 15. Wheelchair bound participants. 16. Current inflammatory skin disease other than DM or ASIM that, in the opinion of the investigator, could interfere with the inflammatory skin assessments or confound the disease activity assessments. 17. Severe interstitial lung disease where respiratory symptoms limit participant function or progressive pulmonary fibrosis. 18. Myositis in overlap with another connective tissue disease that precludes the accurate assessment of a treatment response (for example, difficulty in assessing muscle strength in a scleroderma patient with associated myositis).

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.


Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Horizon Therapeutics Ireland DAC
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Medical Director, MD
Principal Investigator Affiliation Horizon Therapeutics
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Overall Status Recruiting
Countries Australia, United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Idiopathic Inflammatory Myositis
Additional Details

The study will enroll 96 participants with 2 idiopathic inflammatory myositis populations:

  • - Population 1 or dermatomyositis (DM): participants with DM with definite or probable myositis according to the American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria and a DM rash.
  • - Population 2 or anti-synthetase inflammatory myositis (ASIM): participants with ASIM with definite or probable myositis according to ACR/EULAR 2017 criteria and a positive ASIM associated antibody.
Participants will be randomized by population in a 1:1 ratio and receive investigational product (IP) daxdilimab or placebo by subcutaneous injection. The estimated total study duration will be up to 60 weeks.

Arms & Interventions


Experimental: Daxdilimab

Daxdilimab will be administered by subcutaneous (SC) injection over a total of 44 weeks.

Placebo Comparator: Placebo

Matching placebo will be administered by SC injection over a total of 24 weeks, then will be administered active drug by SC injection up to Week 44


Drug: - Daxdilimab

Participants will be administered daxdilimab by subcutaneous (SC) injection

Drug: - Placebo

Participants will be administered identically matching placebo by SC injection over a total of 24 weeks, then participants will be given active treatment for the remainder of the study.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Advanced Research Center, Anaheim, California




Advanced Research Center

Anaheim, California, 92805-5854

Site Contact

Halynn Nguyen

[email protected]


International Sites

Griffith University Austrailia, Spring Hill, Australia




Griffith University Austrailia

Spring Hill, , QLD 4000

Site Contact

Tracey Brumby

[email protected]