Not currently recruiting at UCSF

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

Summary

Eligibility
for people ages 4 years and up (full criteria)
Location
at Oakland 5378538, California 5332921 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 NCT07082725 and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543.

Keywords

Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis, Nizubaglustat, Type C Niemann-Pick Disease, GM2 Gangliosidoses, Counterfeit Drugs

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 (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).

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 not yet accepting patients
    Oakland 5378538 California 5332921 94609 United States
  • M.A.G.I.C. Clinic Ltd. Metabolics and Genetics in Calgary not yet accepting patients
    Calgary 5913490 Alberta 5883102 T3B 6A8 Canada
  • ULS de Santo António, EPE - Centro Materno Infantil Norte accepting new patients
    Porto 2735943 Porto District 2735941 4050-651 Portugal
  • ULS de Santa Maria,EPE - Hospital de Santa Maria - PPDS not yet accepting patients
    Lisbon 2267057 1649-035 Portugal
  • Hospital Infantil Universitario Niño Jesus - PIN accepting new patients
    Madrid 3117735 Madrid 3117732 28009 Spain

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
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