New RNA drug takes aim at tough bladder cancer

NCT ID NCT06351904

First seen Jun 26, 2026 ยท Last updated Jun 26, 2026

Summary

This early-stage trial is testing a new drug called RAG-01 in 72 people with a type of bladder cancer that has not responded to standard therapy. The drug uses a small RNA molecule to try to control the cancer. The main goals are to check safety and find the right dose, while also looking for early signs that it might work.

What this could mean

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

Active substance
RAG-01 (a small activating RNA drug)
What this could lead to
If it works, this could point toward a new treatment option for bladder cancer patients who have not responded to standard therapy.
What could go wrong
This is an early Phase 1 trial with only 72 participants, so safety and effectiveness are not yet proven. 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.

non-invasive bladder urothelial carcinoma Non-Muscle Invasive Bladder Neoplasms

As listed by the trial registrant

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

Contacts and locations

Locations

  • GenesisCare North Shore

    RECRUITING

    St Leonards, New South Wales, 2065, Australia

  • Peninsula & South Eastern Haematology and Oncology Group

    RECRUITING

    Melbourne, Victoria, 3199, Australia

  • The Royal Melbourne Hospital

    RECRUITING

    Melbourne, Victoria, 3050, Australia

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