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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
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

NCT07082543

Active Not RecruitingPhase 3

Sponsor: Azafaros B.V.

Conditions: Gangliosidoses, GM2, Gangliosidosis, GM1

Interventions: AZ-3102, Placebo

Countries: United States, Argentina, Australia, Brazil, Canada, France, Germany, India

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

Eligibility overview

Sex: ALL

Age: 4 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
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-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)
Locations (26)
  • 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
  • Rio de Janeiro, Rio de Janeiro, Brazil
  • Porto Alegre, Rio Grande do Sul, Brazil
  • Calgary, Alberta, Canada
  • Paris, France
  • Höchheim, Germany
  • Ernākulam, Kerala, India
  • New Delhi, National Capital Territory of Delhi, India
  • Jaipur, Rajasthan, India
  • Milan, Italy
  • Porto, Porto District, Portugal
  • Lisbon, Portugal
  • Barcelona, Barcelona, Spain
  • Madrid, Madrid, Spain
  • Gothenburg, Västra Götaland County, Sweden
  • Bern, Canton of Bern, Switzerland
  • Adana, Adana, Turkey (Türkiye)
  • Çankaya, Ankara, Turkey (Türkiye)
  • Bornova, İzmir, Turkey (Türkiye)