NCT06003387 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)
| NCT ID | NCT06003387 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | CSL Behring |
| Condition | Hemophilia B |
| Study Type | INTERVENTIONAL |
| Enrollment | 35 participants |
| Start Date | 2024-01-30 |
| Primary Completion | 2028-10-04 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 35 participants in total. It began in 2024-01-30 with a primary completion date of 2028-10-04.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * 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. * 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. Exclusion Criteria: * • 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: * a) ALT \> 2 × the ULN * b) AST \> 2 × the ULN * c) Alkaline phosphatase \> 2 × the ULN * d) Serum creatinine \> 2 × the ULN * e) 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.
Contact & Investigator
Study Director
STUDY DIRECTOR
CSL Behring
Frequently Asked Questions
Who can join the NCT06003387 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Hemophilia B. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06003387 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 35 participants.
Is NCT06003387 currently recruiting?
Yes, NCT06003387 is actively recruiting participants. Contact the research team at clinicaltrials@cslbehring.com for enrollment information.
Where is the NCT06003387 trial being conducted?
This trial is being conducted at San Diego, United States, Ann Arbor, United States, Pittsburgh, United States, Camperdown, Australia and 11 additional locations.
Who is sponsoring the NCT06003387 clinical trial?
NCT06003387 is sponsored by CSL Behring. The principal investigator is Study Director at CSL Behring. The trial plans to enroll 35 participants.