AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

โ† Trials
๐Ÿงช

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (โ‰ฅ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

RecruitingPhase 3Ages 138 Months - 206 MonthsMaleCSL Behring
๐Ÿ’ฌ

In plain words

What the trial is, and whether it could suit you

This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Key Inclusion Criteria for the Lead-in Period: Assigned male sex at birth * Aged โ‰ฅ138 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 \[โ‰ค\] 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 โ‰ฅ 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 ร— the upper limit of normal (ULN). * Alanine aminotransferase (ALT) \> 2 ร— the ULN. * Aspartate aminotransferase (AST) \> 2 ร— the ULN. * Alkaline phosphatase (ALP) \> 2 ร— the ULN. * Serum creatinine \> 2 ร— 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 ร— 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 ร— the ULN * ALT \> 2 ร— the ULN. * AST \> 2 ร— the ULN. * ALP \> 2 ร— the ULN. * Serum creatinine \> 2 ร— the ULN. * Hemoglobin \< 8 g/dL. * Thrombocytopenia, defined as a platelet count below 50 ร— 10\^9/L, at Visit L-Final (based on central laboratory results).
๐Ÿ’Š

What it tests

  • CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
๐Ÿ“

Where

17 sites
Center for Inherited Blood Disorders
Orange, United States
Rady Children's Hospital San Diego
San Diego, United States
University of Florida
Gainesville, United States
Arthur M. Blank Hospital - Children's Healthcare of Atlanta
Atlanta, United States
University of Michigan Medical Center
Ann Arbor, United States
St. Jude Children's Research Hospital
Memphis, United States
The Children's Hospital at Westmead
Westmead, Australia
Queensland Children's Hospital
Brisbane, Australia

โ€ฆand 9 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
๐Ÿ›ก๏ธ

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it โ€” not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use ยท Privacy