Last updated 2 months ago
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
21 patients around the world
Available in Brazil
This is a multicenter, open-label study to assess the safety, tolerability, PK, PD, and efficacy of Nizubaglustat in male or female patients with late-infantile or juvenile onset GM2 gangliosidosis or NPC disease in two cohorts:
- Cohort 1: Patients who previously took part in Phase 2 Study AZA-001-5A2-01 (RAINBOW) and wish to continue in this open-label study
- Cohort 2: Approximately 10 patients with NPC disease, aged ≥12 years who received full-dose Miglustat for more than 12 months, have stable or worsening disease over the 2 previous clinic visits, and who wish to stop Miglustat treatment and transition to Nizubaglustat.
Azafaros A.G.
3Research sites
21Patients around the world
This study is for people with
Lysosomal Storage Disease
GM2 Gangliosidosis
Niemann-Pick Type C Disease
Requirements for the patient
From 12 Years
All Gender
Medical requirements
- Cohort 1 (NPC and GM2 patients): Have been randomized into Phase 2 Study AZA-001-5A2-01.Cohort 2 (NPC patients): Be male or female aged ≥12 years.Cohort 2 (NPC patients): Have a genetically-confirmed diagnosis of NPC disease.Cohort 2 (NPC patients): Have received full-dose Miglustat treatment for at least 12 months and experienced disease stabilization or worsening with treatment over the 2 previous clinic visits.Cohort 2 (NPC patients): Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.Cohort 2 (NPC patients): Wish to change treatment to Nizubaglustat for their NPC disease.Cohort 2 (NPC patients): Participants from Phase 2 Study AZA-001-5A2-01 (RAINBOW) who transitioned to Miglustat may be eligible for Cohort 2 if they meet all other criteria.Participation is supported and deemed beneficial by the Principal Investigator.Be willing and able to be evaluated for all protocol assessments.The participant, parent, and/or legal guardian can read, understand, and sign the informed consent form.Where appropriate, assent will also be sought for participants who have not reached the age of majority.
- A positive serum pregnancy test (only tested for women of childbearing potential).Female planning to breastfeed during the study.Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.Participation in another interventional or non-interventional study or early access program.
Sites
Instituto Fernandes Figueira
Instituto de Proteção à Infância Dr. Raul Carneiro
Hospital de Clínicas de Porto Alegre (HCPA)
StudyPRISMA
SponsorAzafaros A.G.
Study typeInterventional
Conditions
GM2 GangliosidosisNiemann-Pick Type C Disease
Requirements
From 12 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT07399704
