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Experimental drug tested for rare cancers, but trial ends early

NCT ID NCT03165721

First seen Feb 18, 2026 · Last updated Jun 22, 2026 · Updated 21 times

Summary

This phase 2 trial tested the drug guadecitabine (SGI-110) in people aged 12 and older with certain rare cancers—wild-type GIST, pheochromocytoma, paraganglioma, and HLRCC-related kidney cancer—that had not responded to other treatments. Participants received daily injections for 5 days every 28 days. The study was terminated early after enrolling only 9 people, so it is unclear if the drug helps shrink tumors or slow disease.

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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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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, 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

Guadecitabine (SGI-110)

What this could lead to

If it works, this could point toward a treatment for rare cancers that don't respond to standard therapies.

What could go wrong

This trial was terminated early with only 9 participants, so results are very limited. The drug may not shrink tumors or may cause side effects like injection reactions or low blood counts.

Conditions

The condition(s) this trial relates to.

gastrointestinal stromal tumor kidney cancer paraganglioma pheochromocytoma renal cell carcinoma

As listed by the trial registrant

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