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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 on Niemann-Pick Disease GM1 Gangliosidosis GM2 Gangliosidosis Tay-Sachs Disease

Summary

Eligibility
for people ages 4 years and up (full criteria)
Location
at Oakland, California and other locations
Dates
study started
study ends around

Description

Summary

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

Official Title

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

Details

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 disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.

Treatment specific procedures will be described in the disease-specific subprotocol. Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols.

For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials.gov records: Niemann-Pick type C disease NCT######## and GM1 gangliosidosis or GM2 gangliosidosis NCT########.

Keywords

Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis, Nizubaglustat, Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Type C Niemann-Pick Disease, Niemann-Pick Diseases, Type A Niemann-Pick Disease

Eligibility

You can join if…

Open to people ages 4 years and up

  • 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 (NCTXXX).
  • 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 (NCTXXX).

You CAN'T join if...

  • 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

  • Children's Hospital and Research Center at Oakland
    Oakland California 94609 United States
  • M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary
    Calgary Alberta T3B 6A8 Canada

Details

Status
not yet accepting patients
Start Date
Completion Date
(estimated)
Sponsor
Azafaros A.G.
ID
NCT07054515
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 147 study participants
Last Updated