Efficacy and Safety of Daxdilimab with Prednisone in Adults with Inadequately Controlled Dermatomyositis or Anti‑synthetase Inflammatory Myositis

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What is this study about?

The study focuses on two rare muscle diseases, Dermatomyositis and anti-synthetase inflammatory myositis. These conditions cause muscle weakness and skin rash. The investigational medicine is daxdilimab, given as a subcutaneous injection (a shot placed under the skin). Participants may also continue their usual dose of prednisone, a steroid taken by mouth to reduce inflammation.

The purpose is to see whether adding daxdilimab can lower disease activity compared with placebo. Adults are randomly assigned to receive either the study drug or placebo and neither the participants nor the investigators know which is given (double‑blind). Over about six months, participants attend regular visits where doctors assess improvement using the Total improvement score (a number that shows how much the disease has gotten better) and the CDASI activity score (a measure of skin involvement). Safety is checked at each visit, and blood samples are taken to monitor drug levels and any immune response called antidrug antibodies.

1 enrollment

you receive a study identification number and written information about the trial.

you sign the consent form and are randomly assigned to receive either daxdilimab or matching placebo.

2 baseline visit (day 1)

clinical assessments and laboratory tests are performed to document your condition at the start of the study.

you receive the first dose of the study medication: a 300 mg subcutaneous injection of daxdilimab or the placebo solution.

you continue your regular prednisone 20 mg oral tablet as prescribed for your condition.

3 subsequent study medication administrations

additional doses of the assigned study medication are given according to the study schedule, continuing until the week 24 assessment.

each dose is a 300 mg subcutaneous injection of daxdilimab or the matching placebo.

you maintain the same dose of prednisone 20 mg oral tablet unless a dose adjustment is instructed by the study protocol.

4 scheduled follow‑up visits

you attend clinic visits at predetermined intervals (for example, weeks 2, 4, 8, 12, and 24).

at each visit, clinical evaluations, safety monitoring, and blood samples are collected.

the study medication is administered during the visits according to the schedule.

5 primary efficacy assessment at week 24

a comprehensive evaluation of disease activity is performed to determine the total improvement score (tis) at week 24.

blood samples are taken to measure the concentration of daxdilimab and to test for antibodies against the drug.

the amount of prednisone you are taking is recorded to assess any reduction in dose.

6 end‑of‑study procedures

final safety assessments and laboratory tests are completed.

you receive information about the overall study results and any further follow‑up that may be recommended.

Who Can Join the Study?

Must be an adult aged 18 to 75 years at the time of signing the consent form.
Must have a confirmed diagnosis of myositis (muscle inflammation) that meets the 2017 ACR/EULAR criteria, either:
- Score of 5.5 or higher without a muscle biopsy, and a characteristic skin rash called heliotrope rash or Gottron’s papules/sign if no biopsy is available.
- Score of 6.7 or higher with a muscle biopsy, and the participant must provide the biopsy sample or documentation of the biopsy.
Must belong to one of the two disease groups:
- Dermatomyositis (DM) with a current or past DM rash.
- Anti‑synthetase inflammatory myositis (ASIM) with positive anti‑Jo‑1 antibodies (or one of the other listed antibodies such as anti‑PL‑12, anti‑PL‑7, anti‑KS, anti‑EJ, anti‑OJ, anti‑ZO, anti‑YRS(HA)).
Must have active disease at screening, shown by:
- MMT8 score (a test of muscle strength) less than 142.
- At least two of the following abnormal findings:
  - Patient‑reported disease activity of 2 cm or more on a 10 cm visual analog scale (VAS).
  - Physician‑reported disease activity of 2 cm or more on a 10 cm VAS.
  - Extramuscular activity of 2 cm or more on a 10 cm VAS.
  - At least one muscle enzyme level that is 1.5 times the upper limit of normal (ULN).
  - HAQ‑DI (a disability questionnaire) score of 0.5 or higher.
  - Global muscle damage score of 5 or higher on a 10 cm VAS in the MDI (muscle disease index).
Must be on a stable standard‑of‑care treatment if it is tolerated; if the medication cannot be tolerated or has failed, it must be stopped (washed out) before joining the study.
If taking corticosteroids, the dose must be 20 mg or less of prednisone (or equivalent) per day, and any other allowed immunosuppressant drugs must have been taken at a stable dose for at least 4 weeks before randomization.
If previously unable to tolerate steroids or did not respond, participants must have stopped steroids and any other immunosuppressants before joining.
Must be willing to reduce (taper) the corticosteroid dose according to the study plan when the disease is stable or improving.

Who Cannot Join the Study?

Any other health problem that the doctor thinks could make it hard to see how the study drug works or to keep you safe.
Body weight greater than 160 kg (352 pounds) when you are screened for the study.
Previous severe allergy (including anaphylaxis, a sudden, life‑threatening reaction) to any part of the study drug or to similar medicines called monoclonal antibodies or human immunoglobulin (a type of protein used to treat some conditions).
Having had a major operation within the past 8 weeks, or planning to have an elective (non‑emergency) operation from the time of screening until the study ends.
History of serious heart problems such as unstable angina (chest pain that could signal a heart attack), myocardial infarction (heart attack), or congestive heart failure (heart that cannot pump blood well) within the 6 months before randomization.
Donating more than 50 mL of blood or plasma in the 30 days before screening, or more than 499 mL in the 56 days before screening, or planning to donate blood or plasma during the study or for 6 months after the last dose of the study drug.
Receiving a blood transfusion, packed red blood cells, platelets, or treatments that filter blood such as plasmapheresis or plasma exchange within 8 weeks before randomization, and any time during the study.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Revmatologicky Ustav	Prague	Czechia
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Ospedale San Raffaele S.r.l.	Milan	Italy
Centre Hospitalier Universitaire De Toulouse	Toulouse	France
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Association Institut De Myologie	Paris	France
Universita Degli Studi Di Brescia	Brescia	Italy
Hannqphr Udtudcbfhhiso dd A Cqzkwo	A Coruna Galicia	Spain
Uzjzwjpfousbdwvfjifqm Dgehppsqpeo Agy	Duesseldorf	Germany
Hlkjwedc Uxdgtivzolzqgm Sprauavqug &ykbfpi Hhmdfid dp Hhhbxuuenfg	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
Czechia	Not recruiting	21.07.2023
France	Not recruiting	21.07.2023
Germany	Not recruiting	21.07.2023
Italy	Not recruiting	21.07.2023
Spain	Not recruiting	21.07.2023

Trial locations

Investigated drugs:

Daxdilimab is an experimental medicine given as a small injection under the skin. In this study it is being tested to see if it can lower the activity of dermatomyositis or anti‑synthetase inflammatory myositis, which are conditions that cause muscle weakness and skin rash.

Prednison Léčiva is a tablet that contains the steroid prednisone. It is taken by mouth and is used as a background treatment to help control inflammation and symptoms while participants receive the study drug.

Prednisona Alonga is another brand of oral prednisone tablet. Like other steroids, it is given to reduce inflammation and serve as standard background therapy during the trial.

Prednison acis is a prednisone tablet taken by mouth. It provides the usual steroid treatment that participants continue to use while the experimental injection is being tested.

CORTANCYL is a split‑tablet form of prednisone. Patients swallow it to help keep inflammation under control as part of the regular background medication.

CORTIREX is a prednisone tablet used orally. It is given as background steroid therapy to manage disease symptoms while the new drug is studied.

Investigated diseases:

Dermatomyositis – Dermatomyositis is an inflammatory disease that causes muscle weakness and a distinctive skin rash. The weakness usually starts in the shoulders and hips and may spread to other muscles over time. Skin changes often appear on the face, neck, and back of the hands. As the condition progresses, everyday activities such as climbing stairs or lifting objects can become harder. The disease tends to flare up and then ease, repeating this pattern over months or years.
Anti‑synthetase inflammatory myositis – Anti‑synthetase inflammatory myositis is a type of muscle inflammation linked to specific immune antibodies. It leads to gradual muscle weakness, joint discomfort, and sometimes lung problems. Symptoms develop slowly and can vary in intensity from one episode to the next. Ongoing inflammation can cause persistent difficulty with movements that require strong muscles. The condition follows a pattern of periods of worsening and partial improvement.

Trial ID:

2022-502810-10-00

Protocol code:

HZNP-DAX-205

NCT ID:

NCT05669014

Trial Phase:

Therapeutic exploratory (Phase II)

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Efficacy and Safety of Daxdilimab with Prednisone in Adults with Inadequately Controlled Dermatomyositis or Anti‑synthetase Inflammatory Myositis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?