Nizubaglustat for GM1 and GM2 Gangliosidosis

Recruiting at 34 trial locations

Overseen By Contact for Healthcare Professionals

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Azafaros A.G.

Must not be taking: Anti-epileptics, Investigational drugs

Disqualifiers: Neurologic disease, Hepatic impairment, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

Duration: 18 months

75 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests an oral treatment called nizubaglustat (AZ-3102) to determine its safety and effectiveness for individuals with certain rare genetic disorders, GM1 and GM2 gangliosidosis. These disorders can cause serious nerve problems, and the study aims to assess whether the treatment can help. Participants will join one of two groups: one receiving the treatment and the other a placebo (a harmless pill resembling the treatment). Suitable candidates have GM1 gangliosidosis or specific types of GM2 gangliosidosis and began experiencing nerve problems between ages 1 and 10. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but if you are on anti-epileptic treatments, the dose must be stable for at least 3 months before joining the trial.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that nizubaglustat (AZ-3102) is safe and well-tolerated. In one study, participants who took nizubaglustat experienced few side effects, and their bodies generally accepted the treatment well, supporting further testing and development. This treatment has been tested for conditions like GM2 gangliosidosis and Niemann-Pick disease type C, showing a positive safety profile in these cases. Since this trial is in a later phase, nizubaglustat has demonstrated good safety in earlier tests. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Nizubaglustat is unique because it offers a novel approach to treating GM1 and GM2 gangliosidosis by being an oral dispersible tablet, making it potentially more convenient for patients compared to existing therapies like enzyme replacement or gene therapy, which often involve more invasive administration methods. Researchers are excited about Nizubaglustat because it targets the disease at a biochemical level by inhibiting glucosylceramide synthase, which might help reduce the accumulation of harmful substances in the brain and improve symptoms. This mechanism could provide a more effective and accessible treatment option for those affected by these rare genetic disorders.

What evidence suggests that nizubaglustat might be an effective treatment for GM1 and GM2 gangliosidosis?

This trial will compare Nizubaglustat (AZ-3102) with a placebo to evaluate its potential to treat GM1 and GM2 gangliosidosis, genetic disorders affecting the brain and nervous system. Studies have shown that Nizubaglustat reduces harmful substances that accumulate in the cells of individuals with these conditions. Early results suggest it may slow the progression of symptoms. Although more research is needed, the treatment has received special recognition for its potential benefits in these rare diseases. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals aged 4 and older with late-infantile or juvenile GM1 or GM2 gangliosidosis, such as Tay-Sachs or Sandhoff disease. Participants must have certain levels of ataxic disturbances and agree to use contraception. Excluded are those with other neurological diseases, severe liver/renal impairment, recent investigational drug use, unstable epilepsy treatments, or a body weight under 10 kg.

Inclusion Criteria

I have been diagnosed with GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant.

I am a man willing to follow birth control advice if my partner can have children.

My balance and coordination issues score between 3 and 30.

Exclusion Criteria

Presence of another neurologic disease

Positive serum pregnancy test for women of childbearing potential

History of medical conditions other than GM1 or GM2 gangliosidosis that would confound scientific rigor or interpretation of results

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral nizubaglustat or placebo once daily for 18 months

18 months

Visits at baseline, months 6, 12, and 18

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AZ-3102

Trial Overview The study tests the safety and effectiveness of an oral medication called Nizubaglustat (AZ-3102) compared to a placebo over 18 months in patients with specific forms of gangliosidosis. The participants will be randomly assigned to either the treatment group or the placebo group in a double-blind manner.

How Is the Trial Designed?

Group I: Nizubaglustat Experimental Treatment 1 Intervention

Once daily oral dispersible tablets

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Azafaros A.G.

Lead Sponsor

Trials

2

Recruited

40+

Citations

1. azafaros.com azafaros.com/news/azafaros-announces-initiation-of-two-global-phase-3-studies-with-nizubaglustat-in-niemann-pick-disease-type-c-npc-and-gm1-gm2-gangliosidoses-respectively/l233c14

Azafaros Announces Initiation of two Global Phase 3 ... Nizubaglustat has received Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07082543

NCT07082543 | A Study to Evaluate the Safety and ... 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.

3. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT07082543

Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile ... 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.

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-nizubaglustat-in-patients-with-niemann-pick-type-c-gm1-or-gm2-gangliosidosis/

Study on the Effectiveness and Safety of Nizubaglustat in ... This clinical trial investigates the effectiveness and safety of nizubaglustat in treating Niemann-Pick Type C, GM1, and GM2 Gangliosidosis, ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT07054515

NCT07054515 | A Study to Evaluate the Safety and ... This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple ...

6. azafaros.com azafaros.com/news/azafaros-announces-positive-topline-phase-2-study-data-with-nizubaglustat-in-gm2-gangliosidosis-and-niemann-pick-disease-type-c/l187c14

Azafaros announces positive topline Phase 2 study data ... Study designed to support safety profile showed nizubaglustat was safe and well tolerated; Results support continued development of ...

7. delta.larvol.com delta.larvol.com/Products/?ProductId=2ce9d9b5-8b41-4625-bffb-2804bbc589cb

nizubaglustat (AZ-3102) / Azafaros BLINDED SAFETY DATA OF NIZUBAGLUSTAT PHASE 2 STUDY FOR GM2 GANGLIOSIDOSIS AND NPC DISEASE (SSIEM 2024) - "Background: Nizubaglustat (AZ-3102) is an orally ...