New drug targets rare cancer mutation in early trial

NCT ID NCT02381886

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

Summary

This early-phase study tests a drug called IDH305 in people with advanced cancers that have a specific IDH1R132 mutation. The main goal is to find a safe dose and see if the drug can shrink tumors. About 166 participants will take part.

What this could mean

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

Active substance

IDH305

What this could lead to

If it works, this could point toward a treatment for certain cancers with an IDH1 mutation.

What could go wrong

This is a very early Phase 1 trial, so the drug may not work or could have serious side effects. It is only for people with a specific genetic mutation.

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.

acute myeloid leukemia astrocytoma (excluding glioblastoma) cholangiocarcinoma glioblastoma glioma leukemia Myelodysplastic Syndromes oligodendroglioma

As listed by the trial registrant

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

Contacts and locations

Locations

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Columbia University Medical Center- New York Presbyterian Onc Dept.

    New York, New York, 10032, United States

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Dana Farber Cancer Institute SC (1)

    Boston, Massachusetts, 02115, United States

  • ErasmusMC Cancer lnstitute, Neuro-Oncology, Rm G3-55

    Rotterdam, 3075 EA, Netherlands

  • Novartis Investigative Site

    Melbourne, Victoria, 3004, Australia

  • Novartis Investigative Site

    Parkville, Victoria, 3050, Australia

  • Novartis Investigative Site

    Leuven, 3000, Belgium

  • Novartis Investigative Site

    Toronto, Ontario, M5G 2M9, Canada

  • Novartis Investigative Site

    Cologne, North Rhine-Westphalia, 50937, Germany

  • Novartis Investigative Site

    Jena, Thuringia, 07740, Germany

  • Novartis Investigative Site

    Heidelberg, 69120, Germany

  • Novartis Investigative Site

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Novartis Investigative Site

    Singapore, 168583, Singapore

  • Novartis Investigative Site

    Barcelona, 08035, Spain

  • Uni Of TX MD Anderson Cancer Cntr

    Houston, Texas, 77030, United States