Rare Disease
Clinical trials recruiting in Australia for Rare Disease, as the public registries list them.
At a glance
4 trials recruiting in Australia
3 states or territories with a listed site
0 started recruiting in the last 30 days
281 trials recruiting overseas
11 October 2026 date last checked
Where the trials are
Listed sites in each state and territory. Choose one to show only the trials with a site there, and choose it again to show them all.
1 listed site on this page does not say which state it is in, so the counts above are lower than the real number of sites in some states. The trials themselves are all listed below.
Trials for Rare Disease
4 of 4 trials shown.
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 Not a phased study
Study for people with Rare Disease
Where- Online Patient Enrollment System, Sydney — Recruiting
Who can join Age child, adult, older adult. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Sanford HealthTimelineStarted 1 July 2010 Today 16 October 2026 Estimated completion 1 December 2100NCT01793168 · Checked 11 October 2026
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Recruiting Not a phased study
Study for people with Rare Disease
Where- Murdoch Childrens Research Institute, Parkville (VIC) — Recruiting
Who can join Age child, adult, older adult. Sex Anyone, regardless of sex. Healthy volunteers Accepted.Sponsor Friedreich's Ataxia Research AllianceTimelineStarted 28 June 2023 Today 16 October 2026 Estimated completion 28 January 2048NCT06016946 · Checked 11 October 2026
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By invitation Not a phased study
Study for people with Rare Disease
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.
- Royal Children's Hospital Melbourne, Parkville (VIC) — this site's status is not stated on the record
Who can join Age 1 Day and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Prof. Valérie Mc LinTimelineStarted 26 April 2018 Today 16 October 2026 Estimated completion 26 October 2026NCT06041906 · Checked 11 October 2026
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Recruiting Phase 4
Phase 4 study for people with Rare Disease
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.
- Nepean Hospital, Kingswood (NSW) — Not yet recruiting
- Princess Alexandra Hospital, Woolloongabba (QLD) — Not yet recruiting
Who can join Age child, adult, older adult. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Swedish Orphan BiovitrumTimelineStarted 5 August 2026 Today 16 October 2026 Estimated completion 31 December 2031NCT07746895 · Checked 11 October 2026
No trials match those filters. Clear one to see more.
voxsanity.com.au · trial list from public registries · page built 16 October 2026 · not medical advice
0 selected. Choose 2 or 3 trials to compare them side by side.
Compare selected trialsTrials 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.
Trials overseas
281 trials for this condition 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
About Rare Disease
The text below is reproduced exactly as its source published it. It has not been edited, shortened or rewritten.
A rare disease is one that affects fewer than 200,000 people in the United States. There are nearly 7,000 rare diseases. More than 25 million Americans have one. Rare diseases: - May involve chronic illness, disability, and often premature death - Often have no treatment or not very effective treatment - Are frequently not diagnosed correctly - Are often very complex - Are often caused by changes in genes It can be hard to find a specialist who knows how to treat your rare disease. Disease advocacy groups, rare disease organizations, and genetics clinics may help you to find one. NIH: National Institutes of Health
US source; availability in Australia may differ.
Source: MedlinePlus Health Topics (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
- ClinicalTrials.gov processing date: 9 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.
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