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Hope fades: trial of Tay-Sachs drug venglustat terminated early

NCT ID NCT04221451

Terminated Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 trial tested an oral drug called venglustat in 75 adults and children with late-onset Tay-Sachs or Sandhoff disease, rare genetic disorders that cause progressive nerve damage. The drug aimed to lower toxic fat buildup in the brain and slow disease worsening. However, the study was terminated early, so we don't have clear results on whether it worked.

What this could mean

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

Active substance

venglustat (oral tablet)

What this could lead to

If it had succeeded, venglustat could have slowed or stabilized symptoms of Tay-Sachs and Sandhoff diseases by reducing harmful fat buildup in the brain.

What could go wrong

The trial was terminated early, so results are limited. It was a small study (75 people) and the drug may not have worked or caused side effects. These are ultra-rare diseases, so findings may not apply broadly.

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.

galactosialidosis Gaucher disease due to saposin C deficiency GM1 gangliosidosis Sandhoff disease Sandhoff disease, adult form sialidosis type 1 Tay-Sachs disease Tay-Sachs disease, B variant, adult form Tay-Sachs disease, b variant, juvenile form

As listed by the trial registrant

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

SANDHOFF DISEASE TAY-SACHS DISEASE

Contacts and locations

Locations

  • APHP - Centre Hospitalier la Pitié Salpetrière, Service de Neurologie, 47-83 Boulevard De l'Hôpital_Investigational site number 2500001

    Paris, 75013, France

  • Ataxia Center and HD Center of Excellence, 300 UCLA Med Plaza, Suite B200 - Investigational site number 8400004

    Los Angeles, California, 90095, United States

  • Cambridge University Hospitals NHS Foundation Trust Addenbrookes Hospital Hills Road_investigational site number 8260001

    Cambridge, Cambridgeshire, CB2 OQQ, United Kingdom

  • Centro Hospitalar Universitário Lisboa Norte- Hospital Santa Maria Avenida Professor Egas Moniz_investigational site number 6200002

    Lisbon, 1649-035, Portugal

  • Clínica Universitaria Reina Fabiola Córdoba_investigational site number 0320001

    Córdoba, X5004FHP, Argentina

  • Emory Genetics - Investigational site number 8400006

    Atlanta, Georgia, 30322, United States

  • Gazi Universitesi Tip Fakultesi Emniyet Street Mevlana Blv Besevler Yenimahalle_investigational site number 7920001

    Ankara, 06500, Turkey (Türkiye)

  • Hospital Clínico Universitario de Santiago-CHUS. Travesia de Chopuana, s/n_investigational site number 7240002

    Santiago de Compostela, Galicia [Galicia], 15706, Spain

  • Hospital Maternoinfantil Sant Joan de Déu Paseo de Sant Joan de Déu, 2_investigational site number 7240001

    Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain

  • Hospital Universitari de Bellvitge. Feixa Llarga, S / N_investigational site number 7240004

    L'Hospitalet de Llobregat, Barcelona [Barcelona], 08907, Spain

  • Hospital Universitario Austral Pilar, Buenos Aires_Unidad de Investigación Clínica_investigational site number 0320002

    Pilar, Buenos Aires, B1629ODT, Argentina

  • Hospital de Clinicas de Porto Alegre _investigational site number 0760001

    Porto Alegre, Rio Grande do Sul, 90035 003, Brazil

  • Investigational Site Number : 3800001

    Milan, 20133, Italy

  • Investigational Site Number : 8260003

    Manchester, M13 9WL, United Kingdom

  • Japanese Red Cross Akita Hospital 222-1 Nawashirosawa, Kamikitatesaruta_investigational site number 3920001

    Akita, Akita, 010-1495, Japan

  • Massachusetts General Hospital - Charles River Plaza 175 Cambridge St. Ste 340 - Investigational site number 8400002

    Boston, Massachusetts, 02114, United States

  • NIH National Human Genome Research Institute - 10 Center Dr - Bldg. 10 CRC3-2551 MSC 1205 - Investigational site number 8400005

    Bethesda, Maryland, 20892, United States

  • NYU Langone - 550 First Avenue-Investigational site number 8400001

    New York, New York, 10016, United States

  • Research Center Of Neurology 80, Volokolamskoye shosse_investigational site number 6430001

    Moscow, 125367, Russia

  • Salford Royal NHS Foundation Trust Salford Royal Hospital Stott Lane_investigational site number 8260002

    Salford, M6 8HD, United Kingdom

  • Tohoku Medical and Pharmaceutical University Hospital_investigation site number 3920002

    Sendai, Miyagi, 983-8512, Japan

  • Universitätsklinikum der Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Feulgenstraße 10-12_investigational site number 2760001

    Giessen, 35390, Germany

  • Vseobecna Fakultni Nemocnice V Praze Metabolicke centrum U Nemocnice 2_investigational site number 2030001

    Prague, 12808, Czechia