New hope for NF1 patients: experimental drug targets painful nerve tumors

NCT ID NCT06541847

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

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.

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.

neurofibromatosis type 1 plexiform neurofibroma

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

  • Ann & Robert H. Lurie Children's Hospital of Chicago

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    Chicago, Illinois, 60611, United States

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  • Children's Hospital Colorado

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    Aurora, Colorado, 80045, United States

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  • Children's Hospital Los Angeles

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    Los Angeles, California, 90027, United States

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  • Children's Hospital of Philadelphia

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    Philadelphia, Pennsylvania, 19104, United States

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  • Children's National Hospital

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    Washington D.C., District of Columbia, 20010, United States

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  • Cincinnati Children's Hospital Medical Center

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    Cincinnati, Ohio, 45229, United States

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  • Indiana University

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    Indianapolis, Indiana, 46202, United States

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  • Johns Hopkins Hospital

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    Baltimore, Maryland, 21287, United States

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  • New York University Langone Health

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    New York, New York, 10016, United States

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  • Stanford University

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    Palo Alto, California, 94304, United States

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  • University of Alabama at Birmingham

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    Birmingham, Alabama, 35294, United States

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  • University of Chicago

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    Chicago, Illinois, 60637, United States

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  • University of Texas Southwestern Medical Center

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    Dallas, Texas, 75390, United States

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  • Washington University School of Medicine

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    St Louis, Missouri, 63110, United States

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