A Phase 3 interventional study of AZ-3102 and Placebo in Gangliosidoses, GM2 and Gangliosidosis, GM1, sponsored by Azafaros B.V.. Active, not recruiting at 27 sites in 14 countries. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Azafaros B.V. · Phase 3, Interventional, and Treatment
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
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
Exclusion Criteria:
Once daily oral dispersible tablets
Drug: AZ-3102
Drug: Placebo
Nizubaglustat
Placebo
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)
Time frame: Baseline to month 18
Change from baseline in functional SARA score
Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
Time frame: Baseline to month 18
Change from baseline in SARA score for gait/posture
Assessed on a 9-point scale with higher scores indicating inability to complete the task
Time frame: Baseline to months 6, 12, and 18
Change from baseline in SARA score for speech
Assessed on a 7-point scale with higher scores indicating unintelligible speech/anarthria
Time frame: Baseline to months 6, 12, and 18
Change from baseline in SARA score for kinetics
Assessed on a 5-point scale with higher scores indicating more severe impairment
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Vineland Adaptive Behavior Scale (VABS)
Assesses four domains of function: communication, daily living skills, socialization, and motor skills
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Penetration-Aspiration Scale (PAS)
Assessed on an 8-point ordinal scale, with 1 representing the lowest and 8 the highest/most severe score
Time frame: Baseline to months 6, 12, and 18
Change from baseline in 9-Hole Peg Test (9-HPT-D)
Assessed by the number of pegs per second placed in a 50-second, 9-HPT using the dominant hand
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Goal Attainment Scale (GAS)
Assesses individual goals achieved during the study
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Clinician Global Impression of Change (CGI-C)
Assessed on a 7-point scale with higher scores indicating lower improvement
Time frame: Baseline to months 6, 12, and 18
Change from baseline in Participant/Caregiver Global Impression of Change (PGI-C)
Assessed on a 7-point scale with higher scores indicating lower improvement
Time frame: 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
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
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
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
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
Maximum observed plasma concentration (Cmax)
Time frame: Baseline and months 1,18, and 21
Time to Cmax (Tmax)
Time frame: Baseline and months 1,18, and 21
Plasma trough concentration (Ctrough)
Time frame: 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)
Accumulation ratio for Cmax
Time frame: Baseline and months 1,18, and 21
Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; C18:0
Time frame: Baseline to months 1, 6, 12, and 18
Plan to share: No
No publications or documents are linked to this record.
This study is active, not recruiting, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Azafaros B.V.