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
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Among 17 studies in Gangliosidoses, GM2 with a site in United States — 4 open to participants now, 6 under way, 2 of them phase 3. Gangliosidoses, GM2 · United States.
Among the 17 studies in Gangliosidoses, GM2 with a site in United States, this one is one of 2 that started in 2025, is one of 3 in phase 3, and was the 16th registered.
26 studies on the registry are indexed under Gangliosidoses, GM2; 6 are open to participants now.
Browse Gangliosidoses, GM2 studies →4 of the 17 studies in Gangliosidoses, GM2 with a site in United States are open now. Recruiting first, the newest first:
Azafaros B.V. · Sites in United States, Argentina, Australia and 14 more countries
Ages 4+
Children's Hospital of Philadelphia · Sites in United States
University of Minnesota · Sites in United States
National Human Genome Research Institute (NHGRI) · Sites in United States
Ages 1 days–100 yrs
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 5 Gangliosidoses, GM2 studies recruiting now, 4 set no upper age limit and none stop at 75 or younger; 5 take children under 18; none take healthy volunteers.
This study: 4 Years and older · all sexes · no healthy volunteers.
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17 studies; the count open now beside each. This study's row is marked.
First in 2002; this study’s year is marked. 2 started in 2025.
This study's planned enrollment of 75 is above the median of 21 across 14 interventional studies indexed under Gangliosidoses, GM2.
Median enrollment 13 across the 8 interventional studies, 52 across the 9 observational. Of the 9 that completed or were terminated, 4 (44%) have results posted.
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6 of 17 have an industry lead sponsor. The 10 most frequent:
Azafaros B.V. is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
Azafaros B.V. has 6 studies filed under Gangliosidoses, GM2 — fewer than ten, so there is no comparison to draw.
1 of the studies in Gangliosidoses, GM2 with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT00668187University of MinnesotaA Natural History Study of the Gangliosidoses | 2027-03-01 |
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Among the 17 studies in Gangliosidoses, GM2 with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Started Jun 2025 (actual). Primary completion is estimated for Feb 2028, 31 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nizubaglustat
Azafaros B.V.