Rare disease
Systemic Mastocytosis
Clinical trials recruiting in Australia for Systemic Mastocytosis, a rare disease, as the public registries list them.
Also called: Systemic mast cell disease; Systemic tissue mast cell disease; Systemic mast-cell disease
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At a glance
2 trials recruiting in Australia
11 trials recruiting overseas
1-5 / 10 000 (Point prevalence, Worldwide, Validated) prevalence
All ages age of onset
14 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 Systemic Mastocytosis
Where- Princess Alexandra Hospital, Woolloongabba (QLD) — Recruiting
- The Alfred Hospital, Melbourne (VIC) — Recruiting
- The Canberra Hospital, Garran (ACT) — Recruiting
Who can join Age 18 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Blueprint Medicines CorporationTimelineStarted 30 November 2021 Today 16 October 2026 Estimated completion 30 September 2032NCT04910685 · Checked 14 October 2026
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Recruiting Phase 2
Phase 2 study for people with Systemic Mastocytosis
Where- Nepean Hospital, Kingswood (NSW) — Recruiting
- Gold Coast University Hospital, Southport (QLD) — Recruiting
- Peter MacCallum Cancer Centre, Melbourne N. (VIC) — Recruiting
Who can join Age 18 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Cogent Biosciences, Inc.TimelineStarted 9 November 2021 Today 16 October 2026 Estimated completion 19 September 2025Past estimated completion. The registry still lists this trial as open, and the date it gave for finishing has been and gone. Confirm with the official record before you act on it.
NCT04996875 · Checked 11 October 2026
Trials overseas
11 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 Systemic Mastocytosis
The text below is reproduced exactly as its source published it. It has not been edited, shortened or rewritten.
Systemic mastocytosis is a blood disorder that can affect many different body systems. Individuals with the condition can develop signs and symptoms at any age, but it usually appears after adolescence. Signs and symptoms of systemic mastocytosis often include extreme tiredness (fatigue), skin redness and warmth (flushing), nausea, abdominal pain, bloating, diarrhea, the backflow of stomach acids into the esophagus (gastroesophageal reflux), nasal congestion, shortness of breath, low blood pressure (hypotension), lightheadedness, and headache. Some affected individuals have attention or memory problems, anxiety, or depression. Many individuals with systemic mastocytosis develop a skin condition called urticaria pigmentosa, which is characterized by raised patches of brownish skin that sting or itch with contact or changes in temperature. Nearly half of individuals with systemic mastocytosis will experience severe allergic reactions (anaphylaxis). There are five subtypes of systemic mastocytosis, which are differentiated by their severity and the signs and symptoms. The mildest forms of systemic mastocytosis are the indolent and smoldering types. Individuals with these types tend to have only the general signs and symptoms of systemic mastocytosis described above. Individuals with smoldering mastocytosis may have more organs affected and more severe features than those with indolent mastocytosis. The indolent type is the most common type of systemic mastocytosis. The severe types include aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, and mast cell leukemia. These types are associated with a reduced life span, which varies among the types and affected individuals. In addition to the general signs and symptoms of systemic mastocytosis, these types typically involve impaired function of an organ, such as the liver, spleen, or lymph nodes. The organ dysfunction can result in an abnormal buildup of fluid in the abdominal cavity (ascites). Aggressive systemic mastocytosis is associated with a loss of bone tissue (osteoporosis and osteopenia) and multiple bone fractures. Systemic mastocytosis with an associated hematologic neoplasm and mast cell leukemia both involve blood cell disorders or blood cell cancer (leukemia). Mast cell leukemia is the rarest and most severe type of systemic mastocytosis. Individuals with the milder forms of the condition generally have a normal or near normal life expectancy, while those with the more severe forms typically survive months or a few years after diagnosis.
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: 7 October 2026.
- This page last checked by Voxsanity: 14 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.
Systemic Mastocytosis 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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