Phase 3 study for people with Niemann-Pick Disease Type C
Recruiting Phase 3
This trial at a glance
Where this trial is running
| Site | City | State or country | Status at this site |
|---|---|---|---|
| Women's and Children's Hospital | North Adelaide | SA | Recruiting |
| Royal Melbourne Hospital | Parkville | VIC | Recruiting |
| Royal Children's Hospital Melbourne - PIN | Parkville | VIC | Recruiting |
voxsanity.com.au · trial sites from public registries · registry record synced 11 October 2026 · not medical advice
Who can join
The official eligibility criteria
Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.
Inclusion Criteria:
* Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
* Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).
Exclusion Criteria:
* Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
* Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx
Dates on the record
Results, publications and the official record
- No results are posted on the official record yet.
- No publications are listed on the official record yet.
- Official record and contacts on ClinicalTrials.gov – contact details for each site are kept there, and are not copied onto this page.
Sources and dates
- ClinicalTrials.gov – the record this page reformats
- ClinicalTrials.gov processing date: 24 September 2026.
- This page last checked by Voxsanity: 11 October 2026.
Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.
Not medical advice.
Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.
Letter for your doctor
A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.