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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT07054515




Registration number
NCT07054515
Ethics application status
Date submitted
27/06/2025
Date registered
8/07/2025
Date last updated
8/07/2025

Titles & IDs
Public title
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
Scientific 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
Secondary ID [1] 0 0
2024-515778-28-00
Secondary ID [2] 0 0
AZA-001-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Niemann-Pick Type C Disease 0 0
GM1 Gangliosidosis 0 0
GM2 Gangliosidosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AZ-3102
Treatment: Drugs - Placebo

Experimental: Subprotocol NCTXXXXXXXX: Intervention Group NPC - Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)

Experimental: Subprotocol NCTXXXXXXXX: Intervention Group GM1/GM2 - Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo)


Treatment: Drugs: AZ-3102
Oral dispersible tablets

Treatment: Drugs: Placebo
A matching placebo will be administered in the same regimen as the intervention
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants allocated to subprotocol AZA-001-301-NPC
Timepoint [1] 0 0
Baseline to month 18
Primary outcome [2] 0 0
Number of participants allocated to subprotocol AZA-001-301-GMx
Timepoint [2] 0 0
Baseline to month 18

Eligibility
Key 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 (NCTXXX).
* 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 (NCTXXX).
Minimum age
4 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
30/06/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
4/11/2027
Actual
Sample size
Target
147
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment hospital [2] 0 0
Royal Melbourne Hospital - Parkville
Recruitment hospital [3] 0 0
Royal Children's Hospital Melbourne - PIN - Parkville
Recruitment postcode(s) [1] 0 0
5006 - North Adelaide
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Virginia
Country [4] 0 0
Argentina
State/province [4] 0 0
Buenos Aires
Country [5] 0 0
Argentina
State/province [5] 0 0
Córdoba
Country [6] 0 0
Brazil
State/province [6] 0 0
Rio Grande Do Sul
Country [7] 0 0
Brazil
State/province [7] 0 0
Curitiba
Country [8] 0 0
Brazil
State/province [8] 0 0
Rio De Janeiro
Country [9] 0 0
Canada
State/province [9] 0 0
Alberta
Country [10] 0 0
Canada
State/province [10] 0 0
Quebec
Country [11] 0 0
France
State/province [11] 0 0
Bouches-du-Rhône
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
India
State/province [13] 0 0
Delhi
Country [14] 0 0
India
State/province [14] 0 0
Kerala
Country [15] 0 0
India
State/province [15] 0 0
Maharashtra
Country [16] 0 0
India
State/province [16] 0 0
Rajasthan
Country [17] 0 0
Italy
State/province [17] 0 0
Milano
Country [18] 0 0
Portugal
State/province [18] 0 0
Lisbon
Country [19] 0 0
Portugal
State/province [19] 0 0
Porto
Country [20] 0 0
Spain
State/province [20] 0 0
Barcelona
Country [21] 0 0
Spain
State/province [21] 0 0
Madrid
Country [22] 0 0
Sweden
State/province [22] 0 0
Vastra Gotalands Lan
Country [23] 0 0
Switzerland
State/province [23] 0 0
Bern
Country [24] 0 0
Turkey
State/province [24] 0 0
Ankara
Country [25] 0 0
Turkey
State/province [25] 0 0
Izmir
Country [26] 0 0
Turkey
State/province [26] 0 0
Adana
Country [27] 0 0
United Kingdom
State/province [27] 0 0
Middlesex
Country [28] 0 0
United Kingdom
State/province [28] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Azafaros A.G.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT07054515