NCT07399704Active, not recruitingPhase 2
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Open-label Study to Evaluate the Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease, With or Without Previous Administration of Miglustat
Source: ClinicalTrials.gov · Last updated 21 Oct 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 21 (estimated)
- First submitted
- 17 Dec 2025
- Start date
- 4 Feb 2026
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- Azafaros B.V.
- NCT number
- NCT07399704
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 21
- From 4 Feb 2026 to the primary completion date, 30 Jun 2027
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Any medical event/condition that prevents participation in the study based on the judgment of the Principal Investigator.
Lab values
- A positive serum pregnancy test (only tested for women of childbearing potential).
Other
- Female planning to breastfeed during the study.
- Participation in another interventional or non-interventional study or early access program.
Who the study is looking for
- Cohort 1 (NPC and GM2 patients):
- Have been randomized into Phase 2 Study AZA-001-5A2-01.
- OR
- Cohort 2 (NPC patients):
- Be male or female aged ≥12 years
- Have a genetically-confirmed diagnosis of NPC disease
- 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. Patients experiencing clinical improvement with Miglustat over the preceding 3 months should not be considered for this study.
- Wish to change treatment to Nizubaglustat for their NPC disease.
- 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.
Age in the registry: 12 years to - · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Associação Hospitalar de Prot à Infância Dr. Raul Carneiro Coordinator of this site? Confirm its status | Água Verde | Brazil | Unknown status |
| Hospital de Clinicas de Porto Alegre Coordinator of this site? Confirm its status | Porto Alegre | Brazil | Unknown status |
| Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Coordinator of this site? Confirm its status | Rio de Janeiro | Brazil | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.