Phase 3 study for people with Haemophilia
Recruiting Phase 3
This trial at a glance
Where this trial is running
| Site | City | State or country | Status at this site |
|---|---|---|---|
| The Children's Hospital at Westmead | Westmead | NSW | Recruiting |
| Queensland Children's Hospital | Brisbane | QLD | Recruiting |
voxsanity.com.au · trial sites from public registries · registry record synced 13 October 2026 · not medical advice
Who can join
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:
* Key Inclusion Criteria for the Lead-in Period:
Assigned male sex at birth
* Aged \>=138 months (11 years and 6 months) to less than (\<) 206 months (17 years and 2 months) at the time of informed consent / assent.
* Congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to \[\<=\] 2% of normal circulating FIX) for which the participant has been on continuous FIX prophylaxis.
* On stable continuous FIX prophylaxis for at least 2 months before Screening.
* Minimum of 75 previous exposure days of treatment with FIX protein before Screening.
* Additional Key Inclusion Criteria for the Treatment Period:
Completed the Lead-in Period: minimum of 6 months (26 weeks) of lead-in data collected and eligibility has been confirmed.
* Aged \>= 12 to \< 18 years at the time of CSL222 treatment.
Exclusion Criteria:
* Key Exclusion Criteria for the Lead-in Period:
History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results).
* Screening laboratory values (based on central laboratory results):
* Total bilirubin \> 2 × the upper limit of normal (ULN).
* Alanine aminotransferase (ALT) \> 2 × the ULN.
* Aspartate aminotransferase (AST) \> 2 × the ULN.
* Alkaline phosphatase (ALP) \> 2 × the ULN.
* Serum creatinine \> 2 × the ULN.
* Hemoglobin \< 8 g/dL.
* Any condition other than hemophilia B resulting in an increased bleeding tendency.
* Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at screening (based on central laboratory results).
* Any uncontrolled or untreated infection (human immunodeficiency virus, hepatitis C, etc) or any other significant concurrent, uncontrolled medical condition, as evaluated by the investigator, including, but not limited to renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral, or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder evaluated by the investigator to interfere with adherence to the Clinical Study Protocol procedures or with the degree of tolerance to CSL222.
* Additional Key Exclusion Criteria for the Treatment Period:
Positive FIX inhibitor test at Visit L-Final (based on central laboratory results)
* AAV5 NAb titer \> 1:900 as assessed at Visit LX (last visit before Visit L-Final).
* Visit L-Final laboratory values (based on central laboratory results) of:
* Total bilirubin \> 2 × the ULN
* ALT \> 2 × the ULN.
* AST \> 2 × the ULN.
* ALP \> 2 × the ULN.
* Serum creatinine \> 2 × the ULN.
* Hemoglobin \< 8 g/dL.
* Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at Visit L-Final (based on central laboratory results).
Dates on the record
Results, publications and the official record
- No results are posted on the official record yet.
- No publications are listed on the official record yet.
- Official record and contacts on ClinicalTrials.gov – contact details for each site are kept there, and are not copied onto this page.
Sources and dates
- ClinicalTrials.gov – the record this page reformats
- ClinicalTrials.gov processing date: 6 October 2026.
- This page last checked by Voxsanity: 13 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.