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Tests treatment safety and results for Hemophilia B
Official title 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)
ClinicalTrials.gov ID: NCT06003387
What this study is testing
What is CSL222 (AAV5-hFIXco-Padua)?
CSL222 (AAV5-hFIXco-Padua) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for hemophilia b.
Also referred to as Etranacogene dezaparvovec.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory...
- Has greater than (\>) 150 previous exposure days to FIX replacement therapy.
- Has been on stable FIX prophylaxis for at least 2 months before Screening.
You likely can't join if
- 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...
- 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
See the full eligibility 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.
- 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...
- 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...
- Previous AAV5 gene therapy treatment.
- Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
The study team makes the final eligibility decision.
Where it's taking place
- San Diego, California, United States
- Ann Arbor, Michigan, United States
- Pittsburgh, Pennsylvania, United States
- Camperdown, New South Wales, Australia
- Herston, Queensland, Australia
- Adelaide, South Australia, Australia
- Melbourne, Victoria, Australia
- Hamilton, Ontario, Canada
- Hong Kong, Hong Kong
- Shatin, Hong Kong
- Tel Litwinsky, Israel
- Mexico City, Mexico City, Mexico
- Riyadh, Saudi Arabia
- Singapore, Singapore
- Johannesburg, South Africa
- Daegu, South Korea
- Seoul, South Korea
- Taipei, Neihu District, Taiwan
- Kaohsiung City, Sanmin District, Taiwan
- Chang-hua, Taiwan
+ 5 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Diego, California, United States; Ann Arbor, Michigan, United States; Pittsburgh, Pennsylvania, United States; Camperdown, New South Wales, Australia; Herston, Queensland, Australia; Adelaide, South Australia, Australia and 19 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.