Haemophilia Trial, By Invitation (NCT05768386)

Haemophilia Trial, By Invitation NCT05768386 Sponsor: BioMarin Pharmaceutical Condition: Haemophilia

Printed from Voxsanity (voxsanity.com.au) — NCT05768386 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Haemophilia

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

You must have either already finished a related main treatment study, or be currently enrolled in that main study when it closes — and you can still join even if you have gone back to using regular hemophilia A treatments.
You must be able to understand and agree to the study terms by signing a consent form, or have a legally authorized person (such as a guardian) sign on your behalf.

Who may not be able to join:

If you did not join this follow-up study at the time you finished the main study, you must enroll within 4 months of that completion date — if more than 4 months have passed, you would need special approval from the study's Medical Monitor before joining.
You must be in generally good health, with no condition that the study doctor believes would stop you from fully taking part in the study or affect the study results (this includes, where relevant, advanced HIV disease).
In countries where this rule applies, you must not be legally held or confined in an institution by order of a court or government authority (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: Medical Director, MD, BioMarin Pharmaceutical

Australian sites

Royal Prince Alfred Hospital, Institute of Haematology, Sydney, New South Wales

Royal Brisbane and Women's Hospital, Herston, Queensland

Royal Adelaide Hospital, Adelaide, South Australia

The Alfred Hospital, Melbourne, Victoria

Fiona Stanley Hospital, Murdoch, Western Australia

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT05768386

Sponsor: BioMarin Pharmaceutical

Phase: Not Applicable

Status: By Invitation

Condition: Haemophilia

More information: https://clinicaltrials.gov/study/NCT05768386

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

You must have either already finished a related main treatment study, or be currently enrolled in that main study when it closes — and you can still join even if you have gone back to using regular hemophilia A treatments.
You must be able to understand and agree to the study terms by signing a consent form, or have a legally authorized person (such as a guardian) sign on your behalf.

Who may not be able to join:

If you did not join this follow-up study at the time you finished the main study, you must enroll within 4 months of that completion date — if more than 4 months have passed, you would need special approval from the study's Medical Monitor before joining.
You must be in generally good health, with no condition that the study doctor believes would stop you from fully taking part in the study or affect the study results (this includes, where relevant, advanced HIV disease).
In countries where this rule applies, you must not be legally held or confined in an institution by order of a court or government authority (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

By Invitation

Phase

Not Applicable

Sponsor

BioMarin Pharmaceutical

Registry

ClinicalTrials.gov

Start date

1 January 2023

Est. completion

1 January 2040

Where this trial is recruiting

🇦🇺 Australia 🇧🇪 Belgium 🇧🇷 Brazil 🇫🇷 France 🇩🇪 Germany 🇮🇱 Israel 🇮🇹 Italy 🇿🇦 South Africa 🇰🇷 South Korea 🇪🇸 Spain 🇬🇧 United Kingdom 🇺🇸 United States

5 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Evaluate the long-term safety of BMN 270

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov