Hemophilia BPhase 3NCT06003387
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
A Phase 3 study testing CSL222 (AAV5-hFIXco-Padua) in people with Hemophilia B, run by CSL Behring at 32 sites in 15 countries.
Trial summary
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophilia B
- What is being tested?
- CSL222 (AAV5-hFIXco-Padua)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 32 sitesin 15 countries
- Study dates
- 30 Jan 2024to 2 Apr 2032
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Considered legally an adult, as defined by country regulations.
- Has 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 is on continuous routine FIX prophylaxis.
- Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results).
- Has greater than (>) 150 previous exposure days to FIX replacement therapy.
- Has been on stable FIX prophylaxis for at least 2 months before Screening.
- Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator.
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- Acceptance to adhere to contraception guidelines.
- Able to provide informed consent after receipt of verbal and written information about the study.
- Investigator believes that the participant (or the participant's legally acceptable representative[s]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures.
Who cannot join
- History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results).
- Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin > 2 × the upper limit of normal (ULN) (except if caused by Gilbert's syndrome).
- Screening or Visit L-Final laboratory values (based on central laboratory results) of any of the following laboratory abnormalities:
- ALT > 2 × the ULN
- AST > 2 × the ULN
- Alkaline phosphatase > 2 × the ULN
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- Serum creatinine > 2 × the ULN
- Hemoglobin less than (<) 8 g/dL
- Any condition other than hemophilia B resulting in an increased bleeding tendency.
- Thrombocytopenia, defined as a platelet count <50 × 10^9/L, at Screening or Visit L Final (based on central laboratory results).
- Any uncontrolled or untreated infection (human immunodeficiency virus [HIV], hepatitis B virus [HBV] and hepatitis C virus [HCV], or any other significant concurrent, uncontrolled medical condition 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.
- Known history of allergy to corticosteroids or known medical condition that would require chronic administration of oral corticosteroids.
- Known uncontrolled allergic conditions or allergy / hypersensitivity to any component of the CSL222 excipients (ie, sucrose, potassium chloride, potassium dihydrogen phosphate, sodium chloride, and disodium hydrogen phosphate).
- Previous AAV5 gene therapy treatment.
- Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
Trial locations and contacts
32 sites in 15 countries. Contact the study team or a site near you to ask about taking part.
32 sites in 15 countries
Taiwan5 sites, 5 recruiting
- Recruiting
Changhua Christian Hospital (CCH)
Chang-hua, 500
Use Central Contact
- Recruiting
Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)
Kaohsiung City, Sanmin District, 80756
Use Central Contact
- Recruiting
Taichung Veterans General Hospital -
Taichung, 40705
Use Central Contact
- Recruiting
Tri-Service General Hospital
Taipei, Neihu District, 114
Use Central Contact
- Recruiting
National Taiwan University Hospital
Taipei, 100225
United States4 sites, 4 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Use Central Contact
- Recruiting
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
- Recruiting
Hemophilia Center of Western Pennsylvania (HCWP)
Pittsburgh, Pennsylvania, 15260
- Recruiting
University of California, San Diego (UCSD)
San Diego, California, 92121
Use Central Contact
Australia4 sites, 4 recruiting
- Recruiting
Royal Adelaide Hospital
Adelaide, South Australia, 5000
- Recruiting
Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050
Use Central Contact
- Recruiting
Royal Brisbane Hospital
Herston, Queensland, 4029
Use Central Contact
- Recruiting
The Alfred Hospital
Melbourne, Victoria, 3004
Use Central Contact
South Korea3 sites, 3 recruiting
- Recruiting
Kyungpook National University Hospital
Daegu, 41944
Use Central Contact
- Recruiting
Kyung Hee University Hospital at Gangdong
Seoul, 05278
Use Central Contact
- Recruiting
Severance Hospital, Yonsei University Health System
Seoul, 3722
Use Central Contact
Turkey (Türkiye)3 sites, 3 recruiting
- Recruiting
Ege University Medical Faculty
Bornova, 35100
Use Central Contact
- Recruiting
Gaziantep University Sahinbey Research and Practice Hospital
Gaziantep, 27310
Use Central Contact
- Recruiting
Özel Acibadem Adana Hastanesi
Seyhan, 01130
Use Central Contact
Brazil2 sites, 2 recruiting
- Recruiting
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, 05403-010
- Recruiting
UNICAMP Universidade Estadual de Campinas
São Paulo, 13083-878
Hong Kong2 sites, 2 recruiting
- Recruiting
Queen Mary Hospital
Hong Kong, 999077
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- Recruiting
Prince of Wales Hospital Chinese University of Hong Kong
Shatin, 999077
Use Central Contact
Singapore2 sites, 2 recruiting
- Recruiting
National University Hospital
Singapore, 110974
Use Central Contact
- Recruiting
Singapore General Hospital
Singapore, 169608
Bulgaria1 site, 1 recruiting
- Recruiting
Specialized Hospital for Active Treatment of Hematological Diseases
Sofia, 1756
Canada1 site, 1 recruiting
- Recruiting
McMaster University - Hamilton
Hamilton, Ontario, L8N 3Z5
Use Central Contact
Israel1 site, 1 recruiting
- Recruiting
Sheba Medical Center
Tel HaShomer, 5265601
Use Central Contact
Mexico1 site, 1 recruiting
- Recruiting
Centro de Investigacion Clinica GRAMEL S.C.
Mexico City, Mexico City, 3720
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Poland1 site, 1 recruiting
- Recruiting
Klinika Zaburzen Hemostazy i Chorob Wewnetrznych
Warsaw, 02776
Saudi Arabia1 site, 1 recruiting
- Recruiting
King Faisal Specialist Hospital and Research Center
Riyadh, 11471
Use Central Contact
South Africa1 site, 1 recruiting
- Recruiting
Haemophilia Comprehensive Care Centre
Johannesburg, 2193
Use Central Contact
About this study
Official title: Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
- Conditions
- Hemophilia B
- Being tested
- CSL222 (AAV5-hFIXco-Padua)
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CSL222 (AAV5-hFIXco-Padua)Genetic
Also called Etranacogene dezaparvovec
Administered as a single IV infusion.
| Study group | Role | What they receive |
|---|---|---|
| CSL222 | Experimental | Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram (gc/kg) on Day 1. Receives: Genetic: CSL222 (AAV5-hFIXco-Padua) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Bleeding Rate (ABR)
The total bleeding episodes will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk.
Measured: Months 7 to 18 after CSL222 treatment
Secondary outcomes (30)
Number of participants with Treatment Emergent Adverse Events (TEAEs)
Measured: Up to 60 months after CSL222 treatment
Percentage of participants with TEAEs
Measured: Up to 60 months after CSL222 treatment
Number of TEAEs
Measured: Up to 60 months after CSL222 treatment
Change in Liver ultrasound
Measured: Up to 60 months after CSL222 treatment
Number of participants who develop Factor IX (FIX) Inhibitors
Measured: Up to 60 months after CSL222 treatment
Percentage of participants who develop FIX Inhibitors
Measured: Up to 60 months after CSL222 treatment
Change in hematology and biochemistry parameters
Measured: Up to 60 months after CSL222 treatment
Number of participants with clinically significant increase in Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST)
Measured: Up to 60 months after CSL222 treatment
Percentage of participants with clinically significant increase in ALT or AST
Measured: Up to 60 months after CSL222 treatment
Corticosteroid use for ALT or AST increases after CSL222 treatment
Measured: Up to 60 months after CSL222 treatment
Number of participants with clinically significant Alpha-fetoprotein (AFP)
Measured: Baseline and up to 60 months after CSL222 treatment
Percentage of participants with clinically significant AFP
Measured: Baseline and up to 60 months after CSL222 treatment
Number of participants with infusion related reactions or hypersensitivity reactions
Measured: Throughout CSL222 infusion period and up to 60 months after CSL222 treatment
Percentage of participants with infusion related reactions or hypersensitivity reactions
Measured: Throughout CSL222 infusion period and up to 60 months after CSL222 treatment
Change in the Uncontaminated Endogenous FIX activity
Measured: Baseline and up to Months 6, 12, and 18 after CSL222 treatment
Annualized consumption of FIX replacement therapy
Measured: Months 7 to 18 after CSL222 treatment
Annualized infusion rate of FIX replacement therapy
Measured: Months 7 to 18 after CSL222 treatment
Number of participants remaining free of continuous FIX prophylaxis
Measured: Months 7 to 18 after CSL222 treatment
Percentage of participants remaining free of continuous FIX prophylaxis
Measured: Months 7 to 18 after CSL222 treatment
ABR for spontaneous bleeding episodes
Measured: Months 7 to 18 after CSL222 treatment
ABR for joint bleeding episodes
Measured: Months 7 to 18 after CSL222 treatment
ABR for FIX-treated bleeding episodes
Measured: Months 7 to 18 after CSL222 treatment
Correlation analysis of FIX activity levels with baseline AAV5 NAb titers
Measured: Months 7 to 18 after CSL222 treatment
Number of participants with new target joints and resolved pre-existing target joints
Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint.
Measured: Months 7 to 18 after CSL222 treatment
Number of participants with zero bleeding episodes and zero FIX-treated bleeding episodes
Measured: Months 7 to 18 after CSL222 treatment
Percentage of participants with zero bleeding episodes and zero FIX-treated bleeding episodes
Measured: Months 7 to 18 after CSL222 treatment
Change in the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score
The EQ-5D-5L questionnaire visual analogue scale (VAS) measures overall health status on a vertical VAS ranging from 0 to 100. A higher score indicates better quality of life. The change from baseline in the EQ-5D-5L VAS score will be determined.
Measured: Baseline and up to 18 months after CSL222 treatment
Change in the EQ-5D-5L Index Scores
The EQ-5D-5L questionnaire descriptive system of health-related quality of life consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single index utility score (typically between -0.6 and 1). A higher score indicates better quality of life. The change from baseline in the EQ-5D-5L index score will be determined.
Measured: Baseline and up to 18 months after CSL222 treatment
Number of Participants with Uncontaminated Endogenous FIX Activity of Greater than or Equal to (>=) 5%
Measured: Months 7 to 18 after CSL222 treatment
Percentage of Participants with Uncontaminated Endogenous FIX Activity of >= 5%
Measured: Months 7 to 18 after CSL222 treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2023
- Study start30 Jan 2024
- Primary completion4 Oct 2028 (estimated)
- Study completion2 Apr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06003387
- Sponsor's protocol ID
- CSL222_3005
- Other IDs
- 2023-509590-23-00, 2023
- Status last verified
- Aug 2026
- First submitted
- 1 Aug 2023
- Last updated
- 27 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
- ClinicalTrials.gov ID
- NCT06003387
- Acronym / sponsor ID
- CSL222_3005
- Conditions
- Hemophilia B
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 30 Jan 2024 (actual)
- Primary completion
- 4 Oct 2028 (estimated)
- Study completion
- 2 Apr 2032 (estimated)
- First posted
- 22 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CSL222 | Experimental | Genetic: CSL222 (AAV5-hFIXco-Padua)Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram (gc/kg) on Day 1. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Considered legally an adult, as defined by country regulations.
- Has 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 is on continuous routine FIX prophylaxis.
- Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results).
- Has greater than (>) 150 previous exposure days to FIX replacement therapy.
- Has been on stable FIX prophylaxis for at least 2 months before Screening.
Key exclusion criteria (15 listed)
- History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results).
- Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin > 2 × the upper limit of normal (ULN) (except if caused by Gilbert's syndrome).
- Screening or Visit L-Final laboratory values (based on central laboratory results) of any of the following laboratory abnormalities:
- Any condition other than hemophilia B resulting in an increased bleeding tendency.
- Thrombocytopenia, defined as a platelet count <50 × 10^9/L, at Screening or Visit L Final (based on central laboratory results).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CSL222 (AAV5-hFIXco-Padua)Also: Etranacogene dezaparvovecAdministered as a single IV infusion. | Genetic | CSL222 |
Primary outcome
Annualized Bleeding Rate (ABR)
Time frame: Months 7 to 18 after CSL222 treatment
The total bleeding episodes will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk.
30 secondary outcomes
Number of participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 60 months after CSL222 treatment
Percentage of participants with TEAEs
Time frame: Up to 60 months after CSL222 treatment
Number of TEAEs
Time frame: Up to 60 months after CSL222 treatment
Change in Liver ultrasound
Time frame: Up to 60 months after CSL222 treatment
Number of participants who develop Factor IX (FIX) Inhibitors
Time frame: Up to 60 months after CSL222 treatment
Percentage of participants who develop FIX Inhibitors
Time frame: Up to 60 months after CSL222 treatment
Change in hematology and biochemistry parameters
Time frame: Up to 60 months after CSL222 treatment
Number of participants with clinically significant increase in Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST)
Time frame: Up to 60 months after CSL222 treatment
Percentage of participants with clinically significant increase in ALT or AST
Time frame: Up to 60 months after CSL222 treatment
Corticosteroid use for ALT or AST increases after CSL222 treatment
Time frame: Up to 60 months after CSL222 treatment
Number of participants with clinically significant Alpha-fetoprotein (AFP)
Time frame: Baseline and up to 60 months after CSL222 treatment
Percentage of participants with clinically significant AFP
Time frame: Baseline and up to 60 months after CSL222 treatment
Number of participants with infusion related reactions or hypersensitivity reactions
Time frame: Throughout CSL222 infusion period and up to 60 months after CSL222 treatment
Percentage of participants with infusion related reactions or hypersensitivity reactions
Time frame: Throughout CSL222 infusion period and up to 60 months after CSL222 treatment
Change in the Uncontaminated Endogenous FIX activity
Time frame: Baseline and up to Months 6, 12, and 18 after CSL222 treatment
Annualized consumption of FIX replacement therapy
Time frame: Months 7 to 18 after CSL222 treatment
Annualized infusion rate of FIX replacement therapy
Time frame: Months 7 to 18 after CSL222 treatment
Number of participants remaining free of continuous FIX prophylaxis
Time frame: Months 7 to 18 after CSL222 treatment
Percentage of participants remaining free of continuous FIX prophylaxis
Time frame: Months 7 to 18 after CSL222 treatment
ABR for spontaneous bleeding episodes
Time frame: Months 7 to 18 after CSL222 treatment
ABR for joint bleeding episodes
Time frame: Months 7 to 18 after CSL222 treatment
ABR for FIX-treated bleeding episodes
Time frame: Months 7 to 18 after CSL222 treatment
Correlation analysis of FIX activity levels with baseline AAV5 NAb titers
Time frame: Months 7 to 18 after CSL222 treatment
Number of participants with new target joints and resolved pre-existing target joints
Time frame: Months 7 to 18 after CSL222 treatment
Number of participants with zero bleeding episodes and zero FIX-treated bleeding episodes
Time frame: Months 7 to 18 after CSL222 treatment
Percentage of participants with zero bleeding episodes and zero FIX-treated bleeding episodes
Time frame: Months 7 to 18 after CSL222 treatment
Change in the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score
Time frame: Baseline and up to 18 months after CSL222 treatment
Change in the EQ-5D-5L Index Scores
Time frame: Baseline and up to 18 months after CSL222 treatment
Number of Participants with Uncontaminated Endogenous FIX Activity of Greater than or Equal to (>=) 5%
Time frame: Months 7 to 18 after CSL222 treatment
Percentage of Participants with Uncontaminated Endogenous FIX Activity of >= 5%
Time frame: Months 7 to 18 after CSL222 treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2026.
- Individual participant dataSharing planned: Yes. CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Study Director study director
CSL Behring
32 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing CSL222 (AAV5-hFIXco-Padua) in people with Hemophilia B, run by CSL Behring at 32 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in Taiwan, United States, Australia, South Korea, Turkey (Türkiye) and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Registration Coordinator (1-610-878-4697, clinicaltrials@cslbehring.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Bleeding Rate (ABR) (time frame: Months 7 to 18 after CSL222 treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2026.