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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

Sponsored by Azafaros A.G.

Quick Self-Assessment

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Current Status

Recruiting

Auto-verified from ClinicalTrials.gov

Verified Feb 9, 2026

Phase

Clinical Trial

Sponsor

Azafaros A.G.

Enrollment Target

75

Start Date

Jun 2025

📊
1 of 75 participants interested
1% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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

Who Can Participate

Inclusion Criteria

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 Criteria

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-ep

❓ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

📋

Study Type

INTERVENTIONAL

👥

Target Enrollment

75 participants

Gender

ALL

🏢

Sponsor

Azafaros A.G.

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Oakland

California

Location available
NOT_YET_RECRUITING

Minneapolis

Minnesota

Location available
NOT_YET_RECRUITING

Rochester

Minnesota

Location available
NOT_YET_RECRUITING

Dallas

Texas

Location available
RECRUITING

Fairfax

Virginia

Location available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

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Looking for GM1 Gangliosidosis Treatment in Oakland?

Join others in California exploring innovative treatment options through clinical research

GM1 Gangliosidosis Treatment Options in Oakland, California

If you're searching for GM1 Gangliosidosis treatment in Oakland, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Oakland, Rochester, Fairfax and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with GM1 Gangliosidosis. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 75 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for GM1 Gangliosidosis?

Potential Benefits

  • Access to new treatment approaches before public availability
  • Close monitoring by experienced medical professionals
  • Study-related care provided at no cost
  • Contribute to medical research for GM1 Gangliosidosis

What to Expect

  • Initial screening to determine eligibility
  • Regular check-ups and monitoring visits
  • Possible compensation for time and travel
  • You can withdraw at any time

Frequently Asked Questions About This GM1 Gangliosidosis Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT07082543. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.