A study to evaluate safety and efficacy of Empasiprubart in adults with Dermatomyositis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

18 Mar 2025 → 4 Sep 2025

Decision date (initial)

2024-11-20

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Argenx BV

External identifiers

EU CT number

2023-508337-14-00

ClinicalTrials.gov

NCT06284954

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacodynamic, Efficacy, Therapy, Pharmacokinetic, Others

To evaluate the safety and tolerability of empasiprubart compared with placebo in adult participants with dermatomyositis (DM).

Secondary objectives 1

1. To evaluate the clinical efficacy of empasiprubart as compared with placebo.

Conditions and MedDRA coding

Myositis, Dermatomyositis

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Is at least 18 years of age and the local legal age of consent for clinical studies when signing the Informed Consent Form
2. Is capable of providing signed informed consent and complying with protocol requirements.
3. Agrees to use contraceptive measures consistent with local regulations and women of child-bearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test before receiving the study drug.
4. Has a clinical diagnosis of dermatomyositis or juvenile dermatomyositis. The diagnosis date for juvenile dermatomyositis should be ≤5 years before screening .
5. Has active muscle disease associated with classic dermatomyositis or juvenile dermatomyositis at screening and before the first study drug administration and at least 1 of the following: Elevated levels of creatine kinase, aldolase, lactate dehydrogenase, aspartate aminotransaminase or alanine aminotransferase at screening; or electromyography ≤18 weeks before the first study drug administration; or an MRI depicting active muscle inflammation ≤18 weeks before the first study drug administration; or muscle biopsy demonstrating signs of active inflammation ≤18 weeks before the first study drug administration .
6. Has at least mild skin disease at screening.
7. Complies with the permitted background dermatomyositis treatment requirements at screening.
8. Has had immunization with the first meningococcal, pneumococcal, and the single Haemophilus influenza type B vaccine ≥14 days before the first study drug administration.

Exclusion criteria 19

1. Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of dermatomyositis or puts the participant at undue risk.
2. Naïve to standard dermatomyositis treatment according to local recommendations.
3. History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years before the first study drug administration. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological findings of prostate cancer.
4. Clinically significant active infection that is not sufficiently resolved before the first study drug administration in the investigator’s opinion.
5. Positive serum test at screening for active infection with any of the following: Hepatitis B virus, Hepatitis C virus, HIV.
6. Clinically significant disease, recent major surgery, or intention to have major surgery during the study; or any other medical condition that, in the investigator’s opinion, would confound the results of the study or put the participant at undue risk.
7. Current participation in another interventional clinical study.
8. Known hypersensitivity to the study drug or any of its excipients.
9. History (within 12 months before screening) of or current alcohol, drug, or medication abuse, as assessed by the investigator.
10. Pregnant or lactating state or intending to become pregnant during the study.
11. Previous participation in an empasiprubart clinical study with at least 1 dose of study drug received.
12. Known complement component deficiency as assessed by the investigator.
13. Change in dermatomyositis physical therapy or exercise program from ≤4 weeks before screening.
14. Inflammatory or non-inflammatory myopathies other than dermatomyositis, such as drug-induced or endocrine-induced myositis, infective myositis, polymyositis, immune-mediated necrotizing myopathy, inclusion body myositis, overlap myositis, metabolic myopathies, or muscle dystrophies.
15. Paraneoplastic dermatomyositis secondary to malignancy.
16. Glucocorticoid-induced myopathy.
17. Severe muscle damage.
18. Extensive or severe calcinosis.
19. Interstitial lung disease with at least 1 of the following: forced vital capacity (FVC) ≤60%; supplemental oxygen therapy; rapidly progressing uncontrolled interstitial lung disease; moderate or severe interstitial lung disease.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Incidence of adverse events (AEs) at up to 90 weeks.
2. Percentage of participants discontinuing investigational medicinal product (IMP) due to an adverse event (AE) at up to 25 weeks.

Secondary endpoints 1

1. Total Improvement Score (TIS) at weeks 13 and 25.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Chief Scientific Officer

Public contact point

Organisation

Argenx

Contact name

Vice President Clinical Development

Third parties 11

Locations

4 EU/EEA countries · 14 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 36 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications