Study for people with Haemophilia

By invitation Not a phased study

Sources: ClinicalTrials.gov Checked: 11 October 2026 Not medical advice

This trial at a glance

Status By invitation
Phase Not a phased study
Sponsor BioMarin Pharmaceutical
Registry number NCT05768386
Started 1 January 2023 Today 16 October 2026 Estimated completion 1 January 2040

Where this trial is running

Trial sites and the status recorded for each
SiteCityState or countryStatus at this site
Royal Prince Alfred Hospital, Institute of Haematology Sydney NSW Status not stated
Royal Brisbane and Women's Hospital Herston QLD Status not stated
Royal Adelaide Hospital Adelaide SA Status not stated
The Alfred Hospital Melbourne VIC Status not stated
Fiona Stanley Hospital Murdoch WA Status not stated

voxsanity.com.au · trial sites from public registries · registry record synced 11 October 2026 · not medical advice

Who can join

Age 18 Years and over
Sex Male participants only
Healthy volunteers Not accepted
Enrolment target 172

The official eligibility criteria

Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.

Inclusion Criteria:

* Participants must have completed their primary treatment study or be currently enrolled in the primary treatment study at the time of closure by the Sponsor. Participants may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.

* Subjects must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. If the subject is unable to provide consent, a legally authorized representative may provide written informed consent.

Exclusion Criteria:

* Participants who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study should enroll in 270-401 within 4 months of the date of that study completion visit. If a participant wishes to enroll in 270-401 after 4 months, they must receive prior approval from the Medical Monitor.

* Participants must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the participant from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease).

* Where applicable, per country regulation, the participant must not currently be committed to an institution by virtue of an order issued either by judicial or administrative authorities.

Dates on the record

Started 1 January 2023
Estimated completion 1 January 2040
Registry last updated 12 January 2026
Checked by Voxsanity 11 October 2026

Results, publications and the official record

Sources and dates

  • ClinicalTrials.gov – the record this page reformats
  • ClinicalTrials.gov processing date: 12 January 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.

Letter for your doctor

A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.