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Tests treatment safety and results for Gangliosidoses, GM2
Official title 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
ClinicalTrials.gov ID: NCT07082543
What this study is testing
What is AZ-3102?
AZ-3102 is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for gangliosidoses, gm2.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 3: a large, late-stage study
- Time commitment: about 18 months
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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
You likely can't join if
- A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific...
- 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
See the full eligibility 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
- 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)
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Minneapolis, Minnesota, United States
- Rochester, Minnesota, United States
- Dallas, Texas, United States
- Fairfax, Virginia, United States
- Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
- Córdoba, Córdoba Province, Argentina
- North Adelaide, South Australia, Australia
- Parkville, Victoria, Australia
- Rio de Janeiro, Rio de Janeiro, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- Curitiba, Brazil
- Calgary, Alberta, Canada
- Edmonton, Alberta, Canada
- Montreal, Quebec, Canada
- Paris, France
- Höchheim, Germany
- Ernākulam, Kerala, India
- New Delhi, National Capital Territory of Delhi, India
- Jaipur, Rajasthan, India
+ 16 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 18 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Minneapolis, Minnesota, United States; Rochester, Minnesota, United States; Dallas, Texas, United States; Fairfax, Virginia, United States; Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina and 30 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.