New hope for NF1 patients: experimental drug targets painful nerve tumors
NCT ID NCT06541847
First seen Jun 27, 2026 · Last updated Jul 30, 2026 · Updated 2 times
Summary
This phase 2 trial tests an oral drug called HLX-1502 in people aged 12 and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas—nerve tumors that can cause pain, disfigurement, or other problems. The study will enroll 25 participants and measure whether the drug can shrink tumors by at least 20% on MRI scans. It is open-label, meaning everyone knows they are getting the drug, and there is no placebo group.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- HLX-1502 (an oral drug taken three times daily)
- What this could lead to
- If successful, this could point toward a new treatment option to shrink or control plexiform neurofibromas in people with NF1.
- What could go wrong
- This is an early-phase, small study with no placebo group, so results may not be definitive. The drug may cause side effects or fail to show benefit.
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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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's National Hospital
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
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Indiana University
RECRUITINGIndianapolis, Indiana, 46202, United States
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Johns Hopkins Hospital
RECRUITINGBaltimore, Maryland, 21287, United States
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New York University Langone Health
RECRUITINGNew York, New York, 10016, United States
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35294, United States
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University of Chicago
RECRUITINGChicago, Illinois, 60637, United States
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Real-World study tracks Selumetinib's impact on NF1 tumors in children
- Could a cholesterol drug boost reading skills in kids with NF1?
- Antioxidant drug shows promise for kids with NF1
- Can a simple walk test spot motor trouble in kids with NF1?
- New drug aims to help kids with rare genetic short stature grow taller
- New study tracks Long-Term safety of NF1 drug in kids