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Promising new pill for rare childhood brain diseases enters final testing

NCT ID NCT07082543

Ongoing Disease control Sponsor: Azafaros B.V. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 24, 2026 · Updated 6 times

Summary

This study tests an oral drug called nizubaglustat in children and teens with rare genetic disorders (GM1, GM2 gangliosidosis) that damage the brain and nerves. The goal is to see if the drug can slow disease progression and improve movement and coordination. About 75 participants will receive either the drug or a placebo for 18 months.

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.

GM1 gangliosidosis GM2 gangliosidosis Sandhoff disease Tay-Sachs disease

As listed by the trial registrant

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

GANGLIOSIDOSES, GM2 GANGLIOSIDOSIS, GM1

Contacts and locations

Locations

  • AP-HP - Hôpital Armand Trousseau

    Paris, 75012, France

  • All India Institute of Medical Sciences (AIIMS) - New Delhi

    New Delhi, National Capital Territory of Delhi, 110029, India

  • Amrita Institute of Medical Sciences and Research Centre

    Ernākulam, Kerala, 682041, India

  • Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

    Adana, Adana, 1250, Turkey (Türkiye)

  • Children's Medical Center Dallas

    Dallas, Texas, 75235, United States

  • Ege Universitesi Tip Fakultesi

    Bornova, İzmir, 35100, Turkey (Türkiye)

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi

    Çankaya, Ankara, 6500, Turkey (Türkiye)

  • Hospital Infantil Universitario Niño Jesus - PIN

    Madrid, Madrid, 28009, Spain

  • Hospital Universitario Austral

    Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina

  • Hospital Universitario Vall d'Hebron - PPDS

    Barcelona, Barcelona, 8035, Spain

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

  • Hospital de Niños de La Santisima Trinidad

    Córdoba, Córdoba Province, X5004 ASL, Argentina

  • Inselspital - Universitätsspital Bern

    Bern, Canton of Bern, 3010, Switzerland

  • Instituto Fernandes Figueira

    Rio de Janeiro, Rio de Janeiro, 22250-020, Brazil

  • JK Lone Hospital

    Jaipur, Rajasthan, 302004, India

  • Lysosomal Rare Disorders Research and Treatment Center

    Fairfax, Virginia, 22030-7404, United States

  • M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary

    Calgary, Alberta, T3B 6A8, Canada

  • Mayo Clinic Children's Center - PIN

    Rochester, Minnesota, 55905, United States

  • Sahlgrenska universitetssjukhuset Östra

    Gothenburg, Västra Götaland County, 416 50, Sweden

  • SphinCS GmbH

    Höchheim, 65239, Germany

  • UCSF Children's Hospital and Research Center at Oakland

    Oakland, California, 94609, United States

  • ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS

    Lisbon, 1649-035, Portugal

  • ULS de Santo António, EPE - Centro Materno Infantil Norte

    Porto, Porto District, 4050-651, Portugal

  • University of Minnesota Medical School

    Minneapolis, Minnesota, 55455, United States

  • Women's and Children's Hospital

    North Adelaide, South Australia, 5006, Australia

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