Rare disease
Niemann-Pick Disease Type C
Clinical trials recruiting in Australia for Niemann-Pick Disease Type C, a rare disease, as the public registries list them.
Also called: NPC; Niemann Pick disease type C
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At a glance
3 trials recruiting in Australia
5 trials recruiting overseas
1-9 / 100 000 (Point prevalence, Europe, Validated) prevalence
All ages age of onset
11 October 2026 date last checked
Trials in Australia
This Australian count is likely to be on the low side. It counts the trials this site files under this condition; the registry’s own search is broader and also returns closely related conditions, some of which have their own page here. If you are searching widely, check the registry as well — every trial below links to its own record.
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Recruiting Phase 3
Phase 3 study for people with Niemann-Pick Disease Type C
WhereNo Australian site on this record is listed as recruiting right now, even though the trial itself is open. Check with the site or the official record before travelling.
- The Royal Melbourne Hospital, Parkville (VIC) — Active not recruiting
Who can join Age 4 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor IntraBio IncTimelineStarted 30 June 2022 Today 16 October 2026 Estimated completion 12 June 2030NCT05163288 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Niemann-Pick Disease Type C
Where- 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
Who can join Age 4 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Azafaros B.V.TimelineStarted 30 June 2025 Today 16 October 2026 Estimated completion 14 May 2028NCT07054515 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Niemann-Pick Disease Type C
Where- 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
Who can join Age 4 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Azafaros B.V.TimelineStarted 30 June 2025 Today 16 October 2026 Estimated completion 14 May 2028NCT07082725 · Checked 11 October 2026
Trials overseas
5 trials for this disease are open to new participants outside Australia, as ClinicalTrials.gov listed them on 11 October 2026. Voxsanity does not publish a page for any of them; the searches below reach them at the registry.
These overseas counts are likely too high. They come from the registry’s own search, which expands a condition name to related conditions, so some of the trials counted are for a different disease in the same family.
Search ClinicalTrials.gov Search the WHO registry portal Joining a trial overseas
Trials not shown here
Some Australian trials are registered only on the Australian New Zealand Clinical Trials Registry and are not shown here. Search them at Australian Clinical Trials.
Key facts
Orphadata Science: Free access data from Orphanet. © INSERM 1999. Available on https://www.orphadata.com. Used under CC BY 4.0. Source: Orphanet / Orphadata (orphadata.com).
About Niemann-Pick Disease Type C
The text below is reproduced exactly as its source published it. It has not been edited, shortened or rewritten.
Niemann-Pick disease is a condition that affects many body systems. It has a wide range of symptoms that vary in severity. Niemann-Pick disease is divided into four main types: type A, type B, type C1, and type C2. These types are classified on the basis of genetic cause and the signs and symptoms of the condition. Infants with Niemann-Pick disease type A usually develop an enlarged liver and spleen (hepatosplenomegaly) by age 3 months and fail to gain weight and grow at the expected rate (failure to thrive). The affected children develop normally until around age 1 year when they experience a progressive loss of mental abilities and movement (psychomotor regression). Children with Niemann-Pick disease type A also develop widespread lung damage (interstitial lung disease) that can cause recurrent lung infections and eventually lead to respiratory failure. All affected children have an eye abnormality called a cherry-red spot, which can be identified with an eye examination. Children with Niemann-Pick disease type A generally do not survive past early childhood. Niemann-Pick disease type B usually presents in mid-childhood. The signs and symptoms of this type are similar to type A, but not as severe. People with Niemann-Pick disease type B often have hepatosplenomegaly, recurrent lung infections, and a low number of platelets in the blood (thrombocytopenia). They also have short stature and slowed mineralization of bone (delayed bone age). About one-third of affected individuals have the cherry-red spot eye abnormality or neurological impairment. People with Niemann-Pick disease type B usually survive into adulthood. The signs and symptoms of Niemann-Pick disease types C1 and C2 are very similar; these types differ only in their genetic cause. Niemann-Pick disease types C1 and C2 usually become apparent in childhood, although signs and symptoms can develop at any time. People with these types usually develop difficulty coordinating movements (ataxia), an inability to move the eyes vertically (vertical supranuclear gaze palsy), poor muscle tone (dystonia), severe liver disease, and interstitial lung disease. Individuals with Niemann-Pick disease types C1 and C2 have problems with speech and swallowing that worsen over time, eventually interfering with feeding. Affected individuals often experience progressive decline in intellectual function and about one-third have seizures. People with these types may survive into adulthood.
US source; availability in Australia may differ.
Source: MedlinePlus Genetics (medlineplus.gov), public domain (U.S. National Library of Medicine). Read the full source.
Sources and dates
- ClinicalTrials.gov – trial records, statuses and site lists
- MedlinePlus – the About text above, reproduced exactly
- Orphanet / Orphadata – prevalence, age of onset, inheritance, genes and alternative names, used under CC BY 4.0
- ClinicalTrials.gov processing date: 28 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.
Orphanet citation
Orphadata Science: Free access data from Orphanet. © INSERM 1999. Available on https://www.orphadata.com. Used under CC BY 4.0. Source: Orphanet / Orphadata (orphadata.com). Accessed 20 September 2026.
Niemann-Pick Disease Type C is one of the rare diseases tracked on the Voxsanity rare disease hub, where you can compare it with every other rare condition we follow and filter trials by country.
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