Actively Recruiting
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Led by Azafaros A.G. · Updated on 2026-05-07
21
Participants Needed
3
Research Sites
235 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study (Cohort 1). In addition, the study aims to assess safety, clinical, and biochemical impact of transitioning NPC disease patients to Nizubaglustat after prior treatment with stable, full-dose Miglustat (Cohort 2).
CONDITIONS
Official Title
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Have been randomized into Phase 2 Study AZA-001-5A2-01 (RAINBOW) for Cohort 1
- Be male or female aged 12 years or older for Cohort 2
- Have genetically confirmed Niemann-Pick Type C disease for Cohort 2
- Have received full-dose Miglustat treatment for at least 12 months for Cohort 2
- Have stable or worsening disease over the 2 previous clinic visits while on Miglustat for Cohort 2
- Wish to change treatment to Nizubaglustat for NPC disease for Cohort 2
- Be willing and able to complete all protocol assessments
- Participant, parent, or legal guardian can read, understand, and sign informed consent; assent obtained if applicable
You will not qualify if you...
- Positive serum pregnancy test (for women of childbearing potential)
- Female planning to breastfeed during the study
- Any medical condition or event that prevents participation as judged by the Principal Investigator
- Participation in another interventional or non-interventional study or early access program
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Associação Hospitalar de Prot à Infância Dr. Raul Carneiro
Água Verde, Curitiba, Brazil, 80250-060
Actively Recruiting
2
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
3
Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira
Rio de Janeiro, Brazil, 22250
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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