RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2025-06-30
Study Completion:2027-11-04
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT07054515

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * 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).
  2. * 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).
  1. * Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
  2. * Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Contacts and Locations

Study Contact

Patient Advocacy Representative
CONTACT
Please reach out by email
patientadvocacy@azafaros.com
Contact for Healthcare Professionals
CONTACT
Please reach out by email
medinfo@azafaros.com

Study Locations (Sites)

Children's Hospital and Research Center at Oakland
Oakland, California, 94609
United States
Mayo Clinic Childrens Center - PIN
Rochester, Minnesota, 55905
United States
Lysosomal Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030-7404
United States

Collaborators and Investigators

Sponsor: Azafaros A.G.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06-30
Study Completion Date2027-11-04

Study Record Updates

Study Start Date2025-06-30
Study Completion Date2027-11-04

Terms related to this study

Keywords Provided by Researchers

  • Nizubaglustat

Additional Relevant MeSH Terms

  • Niemann-Pick Type C Disease
  • GM1 Gangliosidosis
  • GM2 Gangliosidosis