Experimental drug shows promise for rare muscle diseases

NCT ID NCT04628936

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested the long-term safety and effectiveness of a drug called KZR-616 in 18 adults with active polymyositis or dermatomyositis, rare diseases that cause muscle inflammation and weakness. Participants who completed an earlier study received weekly injections of KZR-616 for up to 48 weeks. The main goal was to see if the drug improved muscle function and disease activity over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

KZR-616 (zetomipzomib), a drug given as a weekly injection under the skin

What this could lead to

If successful, this could point toward a new treatment option for people with polymyositis or dermatomyositis, helping to control disease activity.

What could go wrong

This is a small, early-phase study with only 18 participants and no placebo comparison, so results may not apply broadly. The drug may cause side effects and may not work for everyone.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

dermatomyositis muscle tissue disorder musculoskeletal system disorder myositis disease polymyositis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • KZR Research Site

    Beverly Hills, California, 90211, United States

  • KZR Research Site

    Orange, California, 92868, United States

  • KZR Research Site

    Miami, Florida, 33136, United States

  • KZR Research Site

    Kansas City, Kansas, 66160, United States

  • KZR Research Site

    Baltimore, Maryland, 21224, United States

  • KZR Research Site

    Ann Arbor, Michigan, 48109, United States

  • KZR Research Site

    Duncansville, Pennsylvania, 16635, United States

  • KZR Research Site

    Pittsburgh, Pennsylvania, 15213, United States

  • KZR Research Site

    Austin, Texas, 78756, United States

  • KZR Research Site

    Prague, Czechia