Experimental drug KZR-616 tested for rare muscle disorders
NCT ID NCT04033926
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 2 trial tested a drug called KZR-616 (zetomipzomib) in 25 adults with active polymyositis or dermatomyositis, two rare diseases that cause muscle inflammation and weakness. Participants received either the drug or a placebo for 16 weeks, then switched to the other treatment for another 16 weeks. The study measured changes in a muscle improvement score to see if the drug helps control symptoms.
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 (also called zetomipzomib)
What this could lead to
If it works, this could point toward a new treatment option for people with polymyositis or dermatomyositis, helping control muscle inflammation and weakness.
What could go wrong
This is a small Phase 2 trial with only 25 participants, so results may not apply to everyone. The drug may not show clear benefit over placebo, and side effects are still being studied.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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KZR Research Site
Beverly Hills, California, 90211, United States
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KZR Research Site
Orange, California, 92868, United States
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KZR Research Site
Miami, Florida, 33136, United States
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KZR Research Site
Atlanta, Georgia, 30322, United States
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KZR Research Site
Kansas City, Kansas, 66160, United States
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KZR Research Site
Baltimore, Maryland, 21224, United States
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KZR Research Site
Ann Arbor, Michigan, 48109, United States
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KZR Research Site
Great Neck, New York, 11021, United States
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KZR Research Site
Duncansville, Pennsylvania, 16635, United States
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KZR Research Site
Pittsburgh, Pennsylvania, 15213, United States
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KZR Research Site
Austin, Texas, 78756, United States
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KZR Research Site
Henrico, Virginia, 23233, United States
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KZR Research Site
Prague, Czechia
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KZR Research Site
Göttingen, Germany