Rare disease
Haemophilia
Clinical trials recruiting in Australia for Haemophilia, a rare disease, as the public registries list them.
Also called: Congenital F9 deficiency; Congenital factor IX deficiency; Christmas disease; Hemophilia; Familial hemophilia; Hereditary hemophilia
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At a glance
10 trials recruiting in Australia
132 trials recruiting overseas
1-9 / 100 000 (Point prevalence, Europe, Validated) prevalence
Childhood; Infancy; Neonatal age of onset
15 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 Haemophilia
Where- Royal Children's Hospital, Melbourne (VIC) — Recruiting
Who can join Age 1 Year to 74 Years. Sex Male participants only. Healthy volunteers Not accepted.Sponsor PfizerTimelineStarted 17 November 2021 Today 16 October 2026 Estimated completion 31 July 2030NCT05145127 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Haemophilia
Where- Royal Prince Alfred Hospital, Camperdown (NSW) — Recruiting
- Sydney Local Health District (RPAH Zone), Camperdown (NSW) — Recruiting
- The Alfred Hospital, Melbourne (VIC) — Recruiting
Who can join Age 18 Years and over. Sex Male participants only. Healthy volunteers Not accepted.Sponsor PfizerTimelineStarted 28 December 2022 Today 16 October 2026 Estimated completion 25 February 2040NCT05568719 · Checked 14 October 2026
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Recruiting Phase 3
Phase 3 study for people with Haemophilia
Where- Murdoch Children's Research Institute, Parkville (VIC) — Recruiting
Who can join Age 1 Year to 17 Years. Sex Male participants only. Healthy volunteers Not accepted.Sponsor PfizerTimelineStarted 9 December 2022 Today 16 October 2026 Estimated completion 10 September 2028NCT05611801 · Checked 15 October 2026
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By invitation Not a phased study
Study for people with Haemophilia
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 Prince Alfred Hospital, Institute of Haematology, Sydney (NSW) — this site's status is not stated on the record
- Royal Brisbane and Women's Hospital, Herston (QLD) — this site's status is not stated on the record
- Royal Adelaide Hospital, Adelaide (SA) — this site's status is not stated on the record
- The Alfred Hospital, Melbourne (VIC) — this site's status is not stated on the record
- Fiona Stanley Hospital, Murdoch (WA) — this site's status is not stated on the record
Who can join Age 18 Years and over. Sex Male participants only. Healthy volunteers Not accepted.Sponsor BioMarin PharmaceuticalTimelineStarted 1 January 2023 Today 16 October 2026 Estimated completion 1 January 2040NCT05768386 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Haemophilia
Where- Royal Prince Alfred Hospital, Camperdown (NSW) — Recruiting
- Royal Brisbane Hospital, Herston (QLD) — Recruiting
- Royal Adelaide Hospital, Adelaide (SA) — Recruiting
- The Alfred Hospital, Melbourne (VIC) — Recruiting
Who can join Age 18 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor CSL BehringTimelineStarted 30 January 2024 Today 16 October 2026 Estimated completion 4 October 2028NCT06003387 · Checked 11 October 2026
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Recruiting Phase 2
Phase 2 study for people with Haemophilia
Where- Royal Prince Alfred Hospital, Haemophilia Treatment Centre, Camperdown (NSW) — Recruiting
- Royal Brisbane and Women's Hospital, Herston (QLD) — Recruiting
- Royal Adelaide Hospital, Adelaide (SA) — Recruiting
- Alfred Hospital, Melbourne (VIC) — Recruiting
Who can join Age 2 Years and over. Sex Male participants only. Healthy volunteers Not accepted.Sponsor Regeneron PharmaceuticalsTimelineStarted 11 September 2024 Today 16 October 2026 Estimated completion 14 August 2034NCT06379789 · Checked 15 October 2026
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Recruiting Phase 3
Phase 3 study for people with Haemophilia
Where- The Children's Hospital at Westmead, Westmead (NSW) — Recruiting
- Queensland Children's Hospital, Brisbane (QLD) — Recruiting
Who can join Age 138 Months to 206 Months. Sex Male participants only. Healthy volunteers Not accepted.Sponsor CSL BehringTimelineStarted 28 July 2025 Today 16 October 2026 Estimated completion 24 October 2033NCT07080905 · Checked 13 October 2026
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Recruiting Phase 3
Phase 3 study for people with Haemophilia
Where- Royal Brisbane and Women's Hospital (RBWH), Herston (QLD) — Recruiting
- Mater Private Hospital, South Brisbane (QLD) — Recruiting
- Gold Coast University Hospital, Southport (QLD) — Recruiting
- St Vincent Hospital, Melbourne, Fitzroy (VIC) — Not yet recruiting
- St Vincent Hospital, Melbourne, Fitzroy (VIC) — Recruiting
- Alfred Health, Melbourne, Melbourne (VIC) — Recruiting
- Fiona Stanley Hospital, Murdoch (WA) — Recruiting
Who can join Age 18 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor VarmX B.V.TimelineStarted 18 August 2026 Today 16 October 2026 Estimated completion 1 January 2029NCT07288489 · Checked 15 October 2026
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Recruiting Phase 3
Phase 3 study for people with Haemophilia
Where- The Alfred Hospital, Melbourne (VIC) — Recruiting
- Royal Children's Hospital Melbourne - PIN, Parkville (VIC) — Recruiting
Who can join Age 12 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Hoffmann-La RocheTimelineStarted 27 April 2026 Today 16 October 2026 Estimated completion 29 February 2028NCT07416604 · Checked 13 October 2026
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Not yet recruiting Phase 3
Phase 3 study for people with Haemophilia
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 Prince Alfred Hospital - Department of Haematology, Camperdown (NSW) — this site's status is not stated on the record
- Royal Brisbane and Women's Hospital - Integrated Cancer Research (I-CARE), Herston (QLD) — this site's status is not stated on the record
- The Alfred - Cancer Trials, Melbourne (VIC) — this site's status is not stated on the record
Who can join Age 18 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Novo Nordisk A/STimelineStarted 9 October 2026 Today 16 October 2026 Estimated completion 23 March 2029NCT07830732 · Checked 11 October 2026
Trials overseas
132 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 Haemophilia
The text below is reproduced exactly as its source published it. It has not been edited, shortened or rewritten.
What is hemophilia? Hemophilia is a rare bleeding disorder in which the blood does not clot properly. This can lead to problems with bleeding too much after an injury or surgery. You can also have sudden bleeding inside your body, such as in your joints, muscles, and organs. Your blood contains many proteins called clotting factors that can help form clots to stop bleeding. People with hemophilia have low levels of one of these factors, usually either factor VIII (8) or factor IX (9). How severe the hemophilia is depends on the amount of factor in the blood. The lower the amount of the factor, the more likely it is that bleeding could happen and might lead to serious health problems. What are the types of hemophilia? There are several different types of hemophilia. The most common are: - Hemophilia A (classic hemophilia), which is caused by a lack or decrease of clotting factor VIII (8) - Hemophilia B (Christmas disease), which is caused by a lack or decrease of clotting factor IX (9) What causes hemophilia? Most types of hemophilia are inherited. They are caused by change in one of the genes (also called a mutation) that provides instructions for making the clotting factor proteins. The change may mean that the clotting proteins don't work properly or that they are missing altogether. These genes are on the X chromosome. You may have one or two X chromosomes: - People who are born male have one X chromosome (from the mother) and one Y chromosome (from the father). They can get hemophilia if their one X chromosome has the gene change. - People who are born female have two X chromosomes, one from the father and one from the mother. They usually only get hemophilia if: - Both X chromosomes have the gene change OR - One X chromosome has the gene change and the other X chromosome is missing or inactive. People who are born female who have the gene change on one X chromosome are a "carrier" of hemophilia. Sometimes they may have some symptoms of hemophilia. They can pass the gene change on to their children. Hemophilia that is not inherited is called acquired hemophilia. It is rare. It happens when your body makes specialized proteins called autoantibodies that attack and disable a clotting factor. This can happen because of pregnancy, immune system disorders, cancer, or allergic reactions to certain medicines. Sometimes the cause is unknown. Who is at risk for hemophilia? Hemophilia is much more common in people who were born male since they can get it with a change to the gene on one X chromosome. People who have a family history of hemophilia are also at higher risk. What are the symptoms of hemophilia? The signs and symptoms of hemophilia are: - Bleeding into the joints. This can cause swelling and pain or tightness in the joints. It often affects the knees, elbows, and ankles. - Bleeding into the skin (which is bruising). - Bleeding into the muscle and soft tissue, which can cause a build-up of blood in the area (called a hematoma). - Bleeding of the mouth and gums, including bleeding that is hard to stop after you lose a tooth. - Bleeding after circumcision. - Bleeding after having shots, such as vaccinations. - Bleeding in the head of an infant after a difficult delivery. - Blood in the urine or stool. - Frequent and hard-to-stop nosebleeds. In some cases, severe hemophilia may cause bleeding in the brain. This may cause brain damage and can be life-threatening. How is hemophilia diagnosed? To find out if you have hemophilia, your health care provider will: - Ask about your medical history, including your symptoms and other health conditions you may have. - Ask about your family history, to find out if you have relatives who have or had hemophilia. - Do a physical exam to look for signs of hemophilia, such as bruising. - Do certain blood tests to show if your blood is clotting properly. If it does not, then you will have clotting factor tests to diagnose the cause of the bleeding disorder. These blood tests would show the type of hemophilia and the severity. There is genetic testing for the factor VIII (8) and factor IX (9) genes. This testing may be used in people who have a family history of hemophilia to: - Identify people who are carriers before they make decisions about pregnancy - Test a fetus for hemophilia during pregnancy - Test a newborn for hemophilia What are the treatments for hemophilia? The best way to treat hemophilia is to replace the missing clotting factor so that your blood can clot properly. This is usually done by injecting replacement clotting factor into a vein. The replacement clotting factor may be made from donated human blood. Or it may be made in a lab; this kind is called a recombinant clotting factor. Replacement clotting factor can help treat a bleeding episode. In more severe cases of hemophilia, you might get the factor on a regular basis to prevent bleeding. You can learn how to inject the factor so that you can do it yourself at home. There are other medicines to treat hemophilia. They may work by releasing factor VIII (8) from where it is stored in the body tissues, replacing the function of factor VIII (8), or preventing clots from breaking down. If bleeding has damaged your joints, physical therapy may help them function better. Good quality medical care from healthcare professionals who know a lot about the disorder can help prevent some serious problems. Often the best choice for care is to visit a hemophilia treatment center (HTC). Centers for Disease Control and Prevention
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
- Orphanet / Orphadata – prevalence, age of onset, inheritance, genes and alternative names, used under CC BY 4.0
- ClinicalTrials.gov processing date: 14 October 2026.
- This page last checked by Voxsanity: 15 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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