Gangliosidoses, GM2Phase 3NCT07082543
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
A Phase 3 study testing AZ-3102 in people with Gangliosidoses, GM2 and Gangliosidosis, GM1, run by Azafaros B.V. at 27 sites in 14 countries.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Gangliosidoses, GM2, Gangliosidosis, GM1
- What is being tested?
- AZ-3102
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 75 participants
- Where
- 27 sitesin 14 countries
- Study dates
- 30 Jun 2025to 6 Mar 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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)
Trial locations and contacts
27 sites in 14 countries.
27 sites in 14 countries
United States5 sites
Children's Medical Center Dallas
Dallas, Texas, 75235
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-7404
University of Minnesota Medical School
Minneapolis, Minnesota, 55455
UCSF Children's Hospital and Research Center at Oakland
Oakland, California, 94609
Mayo Clinic Children's Center - PIN
Rochester, Minnesota, 55905
India3 sites
Amrita Institute of Medical Sciences and Research Centre
Ernākulam, Kerala, 682041
JK Lone Hospital
Jaipur, Rajasthan, 302004
All India Institute of Medical Sciences (AIIMS) - New Delhi
New Delhi, National Capital Territory of Delhi, 110029
Turkey (Türkiye)3 sites
Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi
Adana, Adana, 1250
Ege Universitesi Tip Fakultesi
Bornova, İzmir, 35100
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi
Çankaya, Ankara, 6500
Argentina2 sites
Hospital Universitario Austral
Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ
Hospital de Niños de La Santisima Trinidad
Córdoba, Córdoba Province, X5004 ASL
Brazil2 sites
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, 90560-030
Instituto Fernandes Figueira
Rio de Janeiro, Rio de Janeiro, 22250-020
France2 sites
AP-HP - Hôpital Armand Trousseau
Paris, 75012
AP-HP - Hôpital de la Pitié Salpétrière
Paris
Portugal2 sites
ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS
Lisbon, 1649-035
ULS de Santo António, EPE - Centro Materno Infantil Norte
Porto, Porto District, 4050-651
Spain2 sites
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, 8035
Hospital Infantil Universitario Niño Jesus - PIN
Madrid, Madrid, 28009
Australia1 site
Women's and Children's Hospital
North Adelaide, South Australia, 5006
Canada1 site
M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
Calgary, Alberta, T3B 6A8
Sweden1 site
Sahlgrenska universitetssjukhuset Östra
Gothenburg, Västra Götaland County, 416 50
Switzerland1 site
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, 3010
About this study
Official 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
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
Read the 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
- 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
- Conditions
- Gangliosidoses, GM2, Gangliosidosis, GM1
- Being tested
- AZ-3102, Placebo
- Keywords
- Nizubaglustat
- Medical subject headings
- Gangliosidoses, GM2, Gangliosidosis, GM1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AZ-3102Drug
Nizubaglustat
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Nizubaglustat | Experimental | Once daily oral dispersible tablets Receives: Drug: AZ-3102 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
Measured: Baseline to month 18
Secondary outcomes (20)
Change from baseline in SARA score for gait/posture
Assessed on a 9-point scale with higher scores indicating inability to complete the task
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Baseline to months 6, 12, and 18
Change from baseline in Goal Attainment Scale (GAS)
Assesses individual goals achieved during the study
Measured: 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
Measured: 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
Measured: Baseline to months 6, 12, and 18
Change from baseline in Seizure frequency and duration, as per the seizure diary
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: Baseline to month 18
Maximum observed plasma concentration (Cmax)
Measured: Baseline and months 1,18, and 21
Time to Cmax (Tmax)
Measured: Baseline and months 1,18, and 21
Plasma trough concentration (Ctrough)
Measured: 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
Measured: Baseline (Day 1)
Accumulation ratio for Cmax
Measured: Baseline and months 1,18, and 21
Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; C18:0
Measured: Baseline to months 1, 6, 12, and 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2025
- Study start30 Jun 2025
- Primary completion6 Feb 2028 (estimated)
- Study completion6 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gangliosidoses, GM2Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Azafaros B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07082543
- Sponsor's protocol ID
- AZA-001-301-GMx
- Other IDs
- 2024-515778-28-00
- Status last verified
- Sep 2025
- First submitted
- 27 Jun 2025
- Last updated
- 22 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07082543
- Acronym / sponsor ID
- AZA-001-301-GMx
- Conditions
- Gangliosidoses, GM2, Gangliosidosis, GM1
- Keywords
- Nizubaglustat
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Azafaros B.V.Industry (pharma, biotech, devices)
- Study start
- 30 Jun 2025 (actual)
- Primary completion
- 6 Feb 2028 (estimated)
- Study completion
- 6 Mar 2028 (estimated)
- First posted
- 24 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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
- 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Nizubaglustat | Experimental | Drug: AZ-3102Once daily oral dispersible tablets |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AZ-3102Nizubaglustat | Drug | Nizubaglustat |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
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)
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
20 secondary outcomes
Change from baseline in SARA score for gait/posture
Time frame: Baseline to months 6, 12, and 18
Change from baseline in SARA score for speech
Time frame: Baseline to months 6, 12, and 18
Change from baseline in SARA score for kinetics
Time frame: 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
Change from baseline in Penetration-Aspiration Scale (PAS)
Time frame: 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
Change from baseline in Goal Attainment Scale (GAS)
Time frame: 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
Change from baseline in Participant/Caregiver Global Impression of Change (PGI-C)
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
27 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AZ-3102 in people with Gangliosidoses, GM2 and Gangliosidosis, GM1, run by Azafaros B.V. at 27 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, India, Turkey (Türkiye), Argentina, Brazil and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score (time frame: Baseline to month 18). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2026.