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

June 2, 2026 updated by: Azafaros A.G.

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 GM1 gangliosidosis or GM2 gangliosidosis

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Please see NCT #07054515 for information on the AZA-001-301 Master Protocol

PRIMARY OBJECTIVE

The primary objective of this study is to demonstrate superior efficacy on ataxic manifestations with oral nizubaglustat dosing compared with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis

SECONDARY OBJECTIVES

I. To assess additional efficacy in ataxic and non-ataxic manifestations comparing nizubaglustat dosing with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis

II. To assess the pharmacokinetic (PK) properties of nizubaglustat after administration of the first dose (Visit 1) and at steady state after multiple once daily doses

III. To assess the pharmacodynamic (PD) effects of nizubaglustat

IV. To assess the safety and tolerability of daily oral nizubaglustat dosing compared with placebo, when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis

Study Type

Interventional

Enrollment (Estimated)

75

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, 3052
        • Not yet recruiting
        • Royal Children's Hospital Melbourne - PIN
        • Contact:
  • Brazil
      • Curitiba, Brazil
        • 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
        • Not yet 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 González
  • 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
        • Not yet recruiting
        • Ege Universitesi Tip Fakultesi
  • United Kingdom
      • London, United Kingdom
        • Not yet recruiting
        • Great Ormond Street Hospital NHSFT
      • Manchester, United Kingdom, M13 9WL
        • Not yet recruiting
        • Royal Manchester Children's 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 Children's Center - PIN
    • Texas
      • Dallas, Texas, United States, 75235
        • Not yet 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:

  • Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant
  • Male and female participants aged 4 years and older at the time of informed consent
  • Onset of neurological symptoms from 1 to 10 years
  • Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
  • Females of childbearing potential who are sexually active willing to follow the contraceptive guidance
  • Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance

Exclusion Criteria:

  • A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results
  • Body weight of <10 kg
  • The presence of another neurologic disease
  • The presence of moderate or severe hepatic impairment
  • The presence of moderate or severe renal impairment
  • Platelet count of <100x10^9/L
  • The dose of any anti-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and/or a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline
  • Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening
  • A positive serum pregnancy test (for women of childbearing potential)

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
Placebo Comparator: Placebo
Drug: Placebo
Placebo
Experimental: Nizubaglustat
Once daily oral dispersible tablets
Drug: AZ-3102
Nizubaglustat

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Time Frame: Baseline to month 18
Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)
Baseline to month 18
Change from baseline in functional SARA score
Time Frame: Baseline to month 18
Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
Baseline to month 18

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in SARA score for gait/posture
Time Frame: Baseline to months 6, 12, and 18
Assessed on a 9-point scale with higher scores indicating inability to complete the task
Baseline to months 6, 12, and 18
Change from baseline in SARA score for speech
Time Frame: Baseline to months 6, 12, and 18
Assessed on a 7-point scale with higher scores indicating unintelligible speech/anarthria
Baseline to months 6, 12, and 18
Change from baseline in SARA score for kinetics
Time Frame: Baseline to months 6, 12, and 18
Assessed on a 5-point scale with higher scores indicating more severe impairment
Baseline to months 6, 12, and 18
Change from baseline in Vineland Adaptive Behavior Scale (VABS)
Time Frame: Baseline to months 6, 12, and 18
Assesses four domains of function: communication, daily living skills, socialization, and motor skills
Baseline to months 6, 12, and 18
Change from baseline in Penetration-Aspiration Scale (PAS)
Time Frame: Baseline to months 6, 12, and 18
Assessed on an 8-point ordinal scale, with 1 representing the lowest and 8 the highest/most severe score
Baseline to months 6, 12, and 18
Change from baseline in 9-Hole Peg Test (9-HPT-D)
Time Frame: Baseline to months 6, 12, and 18
Assessed by the number of pegs per second placed in a 50-second, 9-HPT using the dominant hand
Baseline to months 6, 12, and 18
Change from baseline in Clinician Global Impression of Change (CGI-C)
Time Frame: Baseline to months 6, 12, and 18
Assessed on a 7-point scale with higher scores indicating lower improvement
Baseline to months 6, 12, and 18
Change from baseline in Participant/Caregiver Global Impression of Change (PGI-C)
Time Frame: Baseline to months 6, 12, and 18
Assessed on a 7-point scale with higher scores indicating lower improvement
Baseline to months 6, 12, and 18
Change from baseline in Seizure frequency and duration, as per the seizure diary
Time Frame: Baseline to months 6, 12, and 18
Baseline to months 6, 12, and 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of an increase in total SARA score of ≥2 points
Time Frame: Baseline to month 18
Baseline to month 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of a decrease in PAS of ≥1 point
Time Frame: Baseline to month 18
Baseline to month 18
Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of participants leaving the study due to participant/caregiver perception of disease progression/lack of efficacy
Time Frame: Baseline to month 18
Baseline to month 18
Maximum observed plasma concentration (Cmax)
Time Frame: Baseline and months 1,18, and 21
Baseline and months 1,18, and 21
Time to Cmax (Tmax)
Time Frame: Baseline and months 1,18, and 21
Baseline and months 1,18, and 21
Plasma trough concentration (Ctrough)
Time Frame: Baseline and month 1
Baseline and month 1
Area under the plasma concentration-time curve from time of dosing (zero) to 24 hours post-dose (AUC0-24) at baseline
Time Frame: Baseline (Day 1)
Baseline (Day 1)
Accumulation ratio for Cmax
Time Frame: Baseline and months 1,18, and 21
Baseline and months 1,18, and 21
Change from baseline in Goal Attainment Scale (GAS)
Time Frame: Baseline to months 6, 12, and 18
Assesses individual goals achieved during the study
Baseline to months 6, 12, and 18
Time-to-event comparison between nizubaglustat and placebo over the course of the study for pre-defined detrimental events of an increase in functional SARA of ≥1.5 points
Time Frame: Baseline to month 18
Baseline to month 18
Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; C18:0
Time Frame: Baseline to months 1, 6, 12, and 18
Baseline to months 1, 6, 12, and 18

Collaborators and Investigators

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

Sponsor

Azafaros A.G.

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

July 16, 2025

First Posted (Actual)

July 24, 2025

Study Record Updates

Last Update Posted (Actual)

June 4, 2026

Last Update Submitted That Met QC Criteria

June 2, 2026

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AZA-001-301-GMx
  • 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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