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

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

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    RECRUITING

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Hospital

    RECRUITING

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

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Indiana University

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Johns Hopkins Hospital

    RECRUITING

    Baltimore, Maryland, 21287, United States

  • New York University Langone Health

    RECRUITING

    New York, New York, 10016, United States

  • Stanford University

    RECRUITING

    Palo Alto, California, 94304, United States

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35294, United States

  • University of Chicago

    RECRUITING

    Chicago, Illinois, 60637, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

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