New hope for rare cancers: drug targets genetic flaw

NCT ID NCT06357988

First seen Nov 01, 2025 · Last updated Jun 20, 2026 · Updated 31 times

Summary

This phase 2 trial tests the drug vismodegib in 34 people with advanced solid tumors, lymphoma, or multiple myeloma that have specific mutations in the SMO or PTCH1 genes. Vismodegib blocks a protein that can fuel cancer growth. The study aims to see if the drug can shrink or stabilize tumors in these patients.

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 HEMATOPOIETIC AND LYMPHATIC SYSTEM NEOPLASM are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • ECOG-ACRIN Cancer Research Group

    Philadelphia, Pennsylvania, 19103, United States

What this could mean

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

Active substance

Vismodegib (Erivedge)

What this could lead to

If successful, this could provide a new treatment option for people with certain hard-to-treat cancers that have specific genetic mutations.

What could go wrong

This is a small, early-phase trial (34 participants) with no control group, so results may not apply broadly. The drug may cause side effects or fail to shrink tumors.

Conditions

The condition(s) this trial relates to.

cancer hematopoietic and lymphoid system neoplasm lymphoma plasma cell myeloma refractory malignant neoplasm refractory plasma cell neoplasm

As listed by the trial registrant

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