Rare disease
Achondroplasia
Clinical trials recruiting in Australia for Achondroplasia, a rare disease, as the public registries list them.
Also called: ACH; Achondroplastic dwarfism
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At a glance
6 trials recruiting in Australia
7 trials recruiting overseas
Unknown (Point prevalence, Worldwide, Validated) prevalence
Antenatal; Neonatal 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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By invitation Phase 2
Phase 2 study for people with Achondroplasia
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.
- Murdoch Children's Hospital, Parkville (VIC) — this site's status is not stated on the record
Who can join Age 3 Years to 18 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor QED Therapeutics, a BridgeBio companyTimelineStarted 6 December 2021 Today 16 October 2026 Estimated completion 1 December 2031NCT05145010 · Checked 11 October 2026
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By invitation Phase 3
Phase 3 study for people with Achondroplasia
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.
- Ascendis Pharma Investigational Site, Parkville (VIC) — this site's status is not stated on the record
Who can join Age 3 Years to 15 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Ascendis Pharma Growth Disorders A/STimelineStarted 21 June 2023 Today 16 October 2026 Estimated completion 1 January 2039NCT05929807 · Checked 11 October 2026
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Recruiting Phase 2
Phase 2 study for people with Achondroplasia
Where- Ascendis Investigational Site, Parkville (VIC) — Recruiting
Who can join Age 0 Years to 2 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Ascendis Pharma A/STimelineStarted 23 January 2024 Today 16 October 2026 Estimated completion 1 December 2027NCT06079398 · Checked 11 October 2026
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Recruiting Phase 2
Phase 2 study for people with Achondroplasia
Where- The Children's Hospital at Westmead, Westmead (NSW) — Recruiting
- Royal Children's Hospital, Parkville (VIC) — Recruiting
Who can join Age 3 Years to 10 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Tyra Biosciences, IncTimelineStarted 4 March 2025 Today 16 October 2026 Estimated completion 1 January 2030NCT06842355 · Checked 11 October 2026
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Recruiting Phase 2
Phase 2 study for people with Achondroplasia
Where- Murdoch Children's Research Institute, Parkville (VIC) — Recruiting
Who can join Age 0 Years to 32 Months. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor QED Therapeutics, a BridgeBio companyTimelineStarted 19 November 2025 Today 16 October 2026 Estimated completion 1 March 2030NCT07169279 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Achondroplasia
Where- Murdoch Children's Research Institute, Parkville (VIC) — Recruiting
Who can join Age 2 Years to 17 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor BioMarin PharmaceuticalTimelineStarted 20 April 2026 Today 16 October 2026 Estimated completion 1 June 2029NCT07441876 · Checked 11 October 2026
Trials overseas
7 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 Achondroplasia
The text below is reproduced exactly as its source published it. It has not been edited, shortened or rewritten.
Achondroplasia is the most common form of short-limbed dwarfism. The word achondroplasia means "without cartilage formation." Cartilage is a tough but flexible tissue that makes up much of the skeleton during early development. However, in people with achondroplasia the problem is not forming cartilage but converting it to bone (a process called ossification), particularly in the long bones of the arms and legs. Achondroplasia is similar to another skeletal disorder called hypochondroplasia, but the features of achondroplasia tend to be more severe. All people with achondroplasia have short stature. Without treatment, the average height of an adult male with achondroplasia is 131 centimeters (4 feet, 4 inches), and the average height for adult females is 124 centimeters (4 feet, 1 inch). Characteristic features of achondroplasia include an average-size trunk, short arms and legs with particularly short upper arms and thighs, limited range of motion at the elbows, and an enlarged head (macrocephaly) with a prominent forehead. Fingers are typically short and the ring finger and middle finger may diverge, giving the hand a three-pronged (trident) appearance. Health problems commonly associated with achondroplasia include obesity and recurrent ear infections. People with achondroplasia are generally of normal intelligence. In childhood, individuals with the condition usually develop a pronounced and permanent sway of the lower back (lordosis) and bowed legs. Some affected people also develop abnormal front-to-back curvature of the spine (kyphosis) and back pain. As affected individuals age, they may experience a potentially serious complication of achondroplasia called spinal stenosis. Spinal stenosis is a narrowing of the spinal canal that can pinch (compress) the upper part of the spinal cord. Spinal stenosis causes with pain, tingling, and weakness in the legs that can make walking difficult. An uncommon but serious complication of achondroplasia in early childhood is stenosis of the hole at the base of the skull where the spinal cord comes out of brain (foramen magnum). This complication can cause compression of the brain stem, which can lead to pauses in breathing during sleep (sleep apnea) or a condition known as hydrocephalus. Hydrocephalus is a buildup of fluid in the brain that can lead to increased head size and related brain abnormalities.
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: 1 October 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.
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