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Study of Nizubaglustat (AZ-3102) for GM1 and GM2 Gangliosidosis

This study is testing a new oral medication called nizubaglustat (AZ-3102) for people with late-infantile and juvenile forms of GM1 or GM2 gangliosidosis. We want to see if nizubaglustat is safe and if it can improve problems with coordination and balance (ataxia) compared to a placebo (a pill with no medicine). You could be eligible if you are 4 years or older, have a confirmed diagnosis of GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant, and started having neurological symptoms between ages 1 and 10. The study will measure changes in your ataxia scores over 18 months to see if the treatment is successful. The current status of this study is unclear.

Study design
This is an 18-month, double-blind, randomized, placebo-controlled study involving 75 participants. This means some participants will receive nizubaglustat and others will receive a placebo, and neither you nor your doctors will know which you are receiving.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for changes in their ataxia scores from baseline to month 18.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07082543

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

Active, Not Recruiting
PHASE3Ages 4+InterventionalTreatment
Azafaros B.V.
~75 participants
Updated 2026-07-24 on ClinicalTrials.gov
What's tested:AZ-3102Placebo

At a glance

Recruiting sites
0 of 26 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Measured over Baseline to month 18
+1 more outcome measured

Conditions

Gangliosidoses, GM2
Gangliosidosis, GM1

Explore related trials

GM1 GangliosidosisGM2 Gangliosidosis

NCT07082543

Where you'd take part

This study runs at 26 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • All India Institute of Medical Sciences (AIIMS) - New Delhi

    New Delhi, National Capital Territory of Delhi, Indiano site contact published

  • Amrita Institute of Medical Sciences and Research Centre

    Ernākulam, Kerala, Indiano site contact published

  • AP-HP - Hôpital Armand Trousseau

    Paris, Franceno site contact published

  • Balcali Hastanesi Saglik Uygulama ve Arastirma Merkezi

    Adana, Adana, Turkey (Türkiye)no site contact published

  • Children's Medical Center Dallas

    Dallas, Texasno site contact published

  • Ege Universitesi Tip Fakultesi

    Bornova, İzmir, Turkey (Türkiye)no site contact published

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, Italyno site contact published

  • Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi

    Çankaya, Ankara, Turkey (Türkiye)no site contact published

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

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Eligibility criteria

Inclusion

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

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)

What this trial measures

  • Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) scoreBaseline 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 scoreBaseline to month 18

    Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment