# A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

> **NCT07399704** · PHASE2 · RECRUITING · sponsor: **Azafaros A.G.** · enrollment: 21 (estimated)

## Conditions studied

- GM2 Gangliosidosis
- Niemann-Pick Type C Disease

## Interventions

- **DRUG:** AZ-3102

## Key facts

- **NCT ID:** NCT07399704
- **Lead sponsor:** Azafaros A.G.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2026-02-04
- **Primary completion:** 2030-04-15
- **Final completion:** 2030-08-07
- **Target enrollment:** 21 (ESTIMATED)
- **Last updated:** 2026-05-07


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07399704

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07399704, "A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease". Retrieved via AI Analytics 2026-07-19 from https://api.ai-analytics.org/clinical/NCT07399704. Licensed CC0.

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