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Kidney drug trial halted: venglustat fails to slow disease

NCT ID NCT04705051

Terminated Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

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

Summary

This study looked at whether taking venglustat earlier versus later could help people with a fast-progressing form of polycystic kidney disease. The trial was stopped early because the main study found the drug did not work well enough. Only 24 people took part, so no clear conclusions could be drawn about its long-term effects.

What this could mean

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

Active substance
venglustat (a drug taken orally as a capsule)
What this could lead to
If it had worked, this could have pointed toward a treatment to slow kidney growth and preserve kidney function in people with rapidly progressive ADPKD.
What could go wrong
The trial was terminated early because the parent study showed venglustat was not effective for ADPKD. Only 24 people enrolled, so results are very limited.

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.

autosomal dominant polycystic kidney disease cystic kidney disease

As listed by the trial registrant

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

CONGENITAL CYSTIC KIDNEY DISEASE

Contacts and locations

Locations

  • Investigational Site Number :0360001

    Westmead, New South Wales, 2145, Australia

  • Investigational Site Number :0560002

    Leuven, 3000, Belgium

  • Investigational Site Number :2760001

    Berlin, 10117, Germany

  • Investigational Site Number :3920001

    Sapporo, Hokkaido, 060-8648, Japan

  • Investigational Site Number :3920002

    Bunkyo-ku, Tokyo, 113-8431, Japan

  • Investigational Site Number :3920004

    Shinjuku-ku, Tokyo, 162-8666, Japan

  • Investigational Site Number :3920007

    Osaka, Osaka, 545-8586, Japan

  • Investigational Site Number :4100002

    Seoul, Seoul-teukbyeolsi, 07061, South Korea

  • Investigational Site Number :5280002

    Nijmegen, 6525GA, Netherlands

  • Investigational Site Number :7240003

    Barcelona, Barcelona [Barcelona], 08003, Spain

  • Investigational Site Number :8400019

    Morgantown, West Virginia, 26506-9180, United States

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