Trial Radar
Sign in

NCT lookup

Pull any trial record directly from ClinicalTrials.gov.

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, 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 Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515

RecruitingPhase 3

Sponsor: Azafaros B.V.

Conditions: Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis

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 Niemann-Pick type C disease and in late-infantile and juvenile-onset 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:

* Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
* Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).

Exclusion Criteria:

* Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
* Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
Locations (38)
  • 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
  • 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
  • Vellore, Tamil Nadu, India
  • Milan, Italy
  • Monterrey, Nuevo León, Mexico
  • Aguascalientes, Mexico
  • Lahore, Punjab Province, Pakistan
  • Porto, Porto District, Portugal
  • Lisbon, Portugal
  • Barcelona, Barcelona, Spain
  • Madrid, Madrid, Spain
  • + 8 more on CT.gov