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

June 17, 2026 updated by: Azafaros B.V.

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.

Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.

For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT07082725 and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543.

Study Type

Interventional

Enrollment (Estimated)

147

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Contact for Healthcare Professionals
  • Phone Number: Please reach out by email
  • Email: medinfo@azafaros.com

Study Contact Backup

  • Name: Patient Advocacy Representative
  • Phone Number: Please reach out by email
  • Email: info@azafaros.com

Study Locations

  • Argentina
    • Buenos Aires
      • Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina, B1629AHJ
        • Recruiting
        • Hospital Universitario Austral
    • Córdoba Province
      • Córdoba, Córdoba Province, Argentina, X5004 ASL
        • Recruiting
        • Hospital de Niños de La Santisima Trinidad
  • Australia
    • South Australia
      • North Adelaide, South Australia, Australia, 5006
        • Recruiting
        • Women's and Children's Hospital
        • Contact:
    • Victoria
      • Parkville, Victoria, Australia, 3050
      • Parkville, Victoria, Australia, 3052
        • Not yet recruiting
        • Royal Children's Hospital Melbourne - PIN
        • Contact:
  • Brazil
      • Curitiba, Brazil, 80250-060
        • Not yet recruiting
        • Hospital Pequeno Príncipe
        • Contact:
    • Rio Grande do Sul
      • Porto Alegre, Rio Grande do Sul, Brazil, 90560-030
        • Recruiting
        • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
        • Principal Investigator:
          • Roberto Giugliani
        • Contact:
    • Rio de Janeiro
      • Rio de Janeiro, Rio de Janeiro, Brazil, 22250-020
        • Recruiting
        • Instituto Fernandes Figueira
        • Contact:
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Not yet recruiting
        • M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
      • Edmonton, Alberta, Canada, T6G 2B7
    • Quebec
      • Montreal, Quebec, Canada, H2X 0C1
        • Not yet recruiting
        • Centre Hospitalier de l'Universite de Montreal-1000 rue Saint-Denis
  • France
      • Paris, France, 75012
  • Germany
      • Höchheim, Germany, 65239
        • Recruiting
        • SphinCS GmbH
  • India
    • Kerala
      • Ernākulam, Kerala, India, 682041
        • Recruiting
        • Amrita Institute Of Medical Sciences and Research Centre
        • Contact:
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, India, 110029
        • Recruiting
        • All India Institute of Medical Sciences (AIIMS) - New Delhi
        • Contact:
    • Rajasthan
      • Jaipur, Rajasthan, India, 302004
        • Recruiting
        • JK Lone Hospital
        • Contact:
    • Tamil Nadu
      • Vellore, Tamil Nadu, India, 632004
        • Not yet recruiting
        • Christian Medical College and Hospital
  • Italy
      • Milan, Italy, 20133
  • Mexico
      • Aguascalientes, Mexico, 20000
        • Not yet recruiting
        • Centenario Hospital Miguel Hidalgo
    • Nuevo León
      • Monterrey, Nuevo León, Mexico, 64460
        • Not yet recruiting
        • Hospital Universitario Dr. Jose Eleuterio Gonzalez
  • Pakistan
    • Punjab Province
      • Lahore, Punjab Province, Pakistan
        • Not yet recruiting
        • The Children's Hospital & the University of Child Health Lahore
  • Portugal
      • Lisbon, Portugal, 1649-035
        • Recruiting
        • ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
        • Contact:
    • Porto District
      • Porto, Porto District, Portugal, 4050-651
  • Spain
    • Barcelona
      • Barcelona, Barcelona, Spain, 8035
        • Recruiting
        • Hospital Universitario Vall d'Hebron - PPDS
        • Contact:
    • Madrid
      • Madrid, Madrid, Spain, 28009
  • Sweden
    • Västra Götaland County
      • Gothenburg, Västra Götaland County, Sweden, 416 50
        • Recruiting
        • Sahlgrenska universitetssjukhuset Östra
        • Contact:
  • Switzerland
    • Canton of Bern
      • Bern, Canton of Bern, Switzerland, 3010
        • Recruiting
        • Inselspital - Universitätsspital Bern
        • Contact:
  • Turkey (Türkiye)
    • Adana
      • Adana, Adana, Turkey (Türkiye), 1250
        • Not yet recruiting
        • Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
    • Ankara
      • Çankaya, Ankara, Turkey (Türkiye), 6500
        • Recruiting
        • Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
    • İzmir
      • Bornova, İzmir, Turkey (Türkiye), 35100
        • Recruiting
        • Ege Universitesi Tip Fakultesi
  • United Kingdom
      • Manchester, United Kingdom, M13 9WL
        • Not yet recruiting
        • Royal Manchester Children's Hospital
    • London
      • London, London, United Kingdom, WC1N 3JH
        • Not yet recruiting
        • Great Ormond Street Hospital
    • Middlesex
      • London, Middlesex, United Kingdom, WC1N 3BG
        • Not yet recruiting
        • University College London Hospitals (UCLH)
  • United States
    • California
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF Children's Hospital and Research Center at Oakland
        • Principal Investigator:
          • Caroline Hastings, MD
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Not yet recruiting
        • University of Minnesota Medical School
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic Childrens Center - PIN
    • Texas
      • Dallas, Texas, United States, 75235
        • Recruiting
        • Children's Medical Center Dallas
    • Virginia
      • Fairfax, Virginia, United States, 22030-7404
        • Recruiting
        • Lysosomal Rare Disorders Research and Treatment Center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
  • Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).

Exclusion Criteria:

  • Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
  • Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Subprotocol NCT07082725: Intervention Group NPC
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Drug: AZ-3102
Oral dispersible tablets
Other Names:
  • nizubaglustat
Drug: Placebo
A matching placebo will be administered in the same regimen as the intervention
Experimental: Subprotocol NCT07082543: Intervention Group GM1/GM2
Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)
Drug: AZ-3102
Oral dispersible tablets
Other Names:
  • nizubaglustat
Drug: Placebo
A matching placebo will be administered in the same regimen as the intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants allocated to subprotocol AZA-001-301-NPC
Time Frame: Baseline to month 18
Baseline to month 18
Number of participants allocated to subprotocol AZA-001-301-GMx
Time Frame: Baseline to month 18
Baseline to month 18

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Azafaros B.V.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 30, 2025

Primary Completion (Estimated)

November 4, 2027

Study Completion (Estimated)

November 4, 2027

Study Registration Dates

First Submitted

June 27, 2025

First Submitted That Met QC Criteria

June 27, 2025

First Posted (Actual)

July 8, 2025

Study Record Updates

Last Update Posted (Actual)

June 18, 2026

Last Update Submitted That Met QC Criteria

June 17, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AZA-001-301
  • 2024-515778-28-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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