A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Recruiting
Phase 2
Interventional Study
GM2 Gangliosidosis
Niemann-Pick Type C Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 12 and older
- Sex
- Any
- Trial phase
- Phase 2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 21 (estimated)
- Sponsor
- Azafaros B.V. · Industry
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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 s…
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
* 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…
Contacts
No contact information available.