Targeted drug cobimetinib tested for rare immune cell cancers
NCT ID NCT04079179
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This phase 2 study tests the drug cobimetinib in children and adults with Langerhans cell histiocytosis and related disorders that have come back or not responded to standard treatment. Cobimetinib blocks a growth signal that goes haywire in these diseases. The study will enroll 90 participants across four groups and measure how well the drug controls the disease over one year.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Cobimetinib (a targeted cancer drug that blocks a protein called MEK)
- What this could lead to
- If it works, this could offer a new treatment option for people with hard-to-treat histiocytic disorders, potentially controlling the disease and slowing neurodegeneration.
- What could go wrong
- This is a mid-stage trial with only 90 participants, so results may not apply to everyone. The drug may cause side effects and may not work for all types of histiocytosis.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Children's Hospital of Orange County
RECRUITINGOrange, California, 92868, United States
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Children's Medical Center- UTSW
RECRUITINGDallas, Texas, 75235, United States
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Dana Farber Cancer Institute, Boston Children's
RECRUITINGBoston, Massachusetts, 02215, United States
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John Hopkins University School of Medicine
RECRUITINGBaltimore, Maryland, 21287, United States
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Memorial Sloan Kettering Cancer Center
RECRUITINGNew York, New York, 10065, United States
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NACHO Consortium
NOT_YET_RECRUITINGMemphis, Tennessee, 38105, United States
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Phoenix Children's Hospital
RECRUITINGPhoenix, Arizona, 85016, United States
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
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UCSF Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94158, United States
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University of Wisconsin-American Family Children's Hospital
TERMINATEDMadison, Wisconsin, 53792, United States
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