Targeted pill shows promise for rare genetic cancers

NCT ID NCT05199584

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

Summary

This study tested an experimental drug called ENV-101 (taladegib) in 20 adults with advanced solid tumors that have a specific genetic mutation (PTCH1 loss-of-function). The drug works by blocking a growth pathway in cancer cells. Researchers measured how many patients' tumors shrank and monitored side effects.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

ENV-101 (taladegib) tablets

What this could lead to

If successful, this could point toward a targeted treatment option for people with certain hard-to-treat cancers that have a specific genetic change.

What could go wrong

This is an early-phase trial with only 20 participants, so results may not apply broadly. The drug may not shrink tumors or could cause significant side effects.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for SOLID TUMORS WITH PTCH1 LOSS-OF-FUNCTION MUTATIONS are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Adenoma, Islet Cell glioma laryngeal disorder medulloblastoma melanoma mesothelioma neuroblastoma osteosarcoma pituitary gland disorder retinoblastoma rhabdomyosarcoma sarcoma Uveal Melanoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Research Site

    Los Angeles, California, 90095, United States

  • Research Site

    Santa Rosa, California, 95403, United States

  • Research Site

    Tampa, Florida, 33609, United States

  • Research Site

    Zion, Illinois, 60099, United States

  • Research Site

    Covington, Louisiana, 70433, United States

  • Research Site

    Las Vegas, Nevada, 89102, United States

  • Research Site

    New York, New York, 10065, United States

  • Research Site

    Durham, North Carolina, 27710, United States

  • Research Site

    Cincinnati, Ohio, 45229, United States

  • Research Site

    Columbus, Ohio, 43210, United States

  • Research Site

    Pittsburgh, Pennsylvania, 15232, United States

  • Research Site

    Nashville, Tennessee, 37203, United States

  • Research Site

    Houston, Texas, 77030, United States

  • Research Site

    Fredericksburg, Virginia, 22408, United States

  • Research Site

    Lynchburg, Virginia, 24501, United States

  • Research Site

    Madison, Wisconsin, 53792, United States