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Studies

Interventional Recruiting Phase 3

NCT07054515 on ClinicalTrials.gov

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

Official title: 18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

  • Sponsor: Azafaros
  • Enrollment: 147 (Estimated)
  • Study start: 2025-06-30 (Actual)
  • Primary completion: 2028-05-14 (Estimated)
  • Study completion: 2028-06-14 (Estimated)
  • Allocation: Randomized
  • InterventionModel: Parallel
  • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • PrimaryPurpose: Treatment

Brief Summary

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

Conditions

  • Niemann-Pick Type C Disease
  • GM1 Gangliosidosis
  • GM2 Gangliosidosis

Intervention / Treatment

  • Drug: AZ-3102
  • Drug: Placebo

Primary Outcome Measures

  • Number of participants allocated to subprotocol AZA-001-301-NPC (Baseline to month 18)
  • Number of participants allocated to subprotocol AZA-001-301-GMx (Baseline to month 18)

Countries

Argentina, Australia, Brazil, Canada, France, Germany, India, Italy, Mexico, Pakistan, Portugal, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom, United States

Locations and Investigators

  • Queen Silvia Childrens Hospital - Sahlgrenska University Hospital - Vaestra Goetalandsregionen, Gothenburg, Sweden
    • Niklas, Darin
  • Assistance Publique Hopitaux De Paris, Paris, France
    • Bénédicte, HERON
  • Hospital Infantil Universitario Nino Jesus, Madrid, Spain
    • Beatriz, Bernardino Cuesta
  • Hospital Universitari Vall D Hebron, Barcelona, Spain
    • Mireia, del Toro
  • SphinCS GmbH, Hochheim Am Main, Germany
    • Eugen, Mengel
  • Unidade Local De Saude De Santo Antonio E.P.E., Porto, Portugal
    • Anabela, Bandeira
  • Unidade Local De Saude De Santa Maria E.P.E., Lisbon, Portugal
    • Tiago, Proença dos Santos
  • IRCCS Foundation Istituto Neurologico Carlo Besta, Milan, Italy
    • Anna, Ardissone