Targeted pill shows promise for rare genetic cancers

NCT ID NCT05199584

Completed Disease control Sponsor: Endeavor Biomedicines, Inc. Source: ClinicalTrials.gov ↗

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.

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.

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.

SOLID TUMORS WITH PTCH1 LOSS-OF-FUNCTION MUTATIONS

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
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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