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临床试验/NCT07080905
NCT07080905招募中3 期

Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

CSL Behring14 个研究点 分布在 8 个国家目标入组 20 人开始时间: 2025年7月28日最近更新:
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适应症
干预措施

试验速览

阶段
3 期
状态
招募中
发起方
CSL Behring
入组人数
20
试验地点
14
主要终点
Annualized Bleeding Rate (ABR)
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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.

研究设计

研究类型
Interventional
分配方式
Na
干预模型
Single Group
主要目的
Treatment
盲法
None

入排标准

年龄范围
138 Months 至 206 Months（Child）
性别
Male
接受健康志愿者
否

入选标准

  • •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.

排除标准

  • •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 CSL
  • •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).

研究组 & 干预措施

CSL222

Experimental

Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram of body weight (gc/kg).

干预措施: CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua) (Genetic)

结局指标

主要结局

Annualized Bleeding Rate (ABR)

时间窗: For the Lead-In Period (at least 6 months) and for the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222)

次要结局

  • Endogenous FIX activity(At baseline, and at months 6, 12, and 18 after treatment with CSL222)
  • Change from baseline in endogenous FIX activity(At baseline, and at months 6, 12, and 18 after treatment with CSL222)
  • Number of participants with endogenous FIX activity of ≥ 5%(Up to 5 years after treatment with CSL222)
  • Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) total score and change from baseline(At baseline and Month 18 post treatment)
  • Annualized consumption of FIX replacement therapy(For the Lead-In Period (at least 6 months), for the 52 weeks following stable FIX expression (months 7 to 18 after treatment with CSL222), and annually in the Posttreatment Follow-up Period (up to 5 years))
  • Annualized infusion rate of FIX replacement therapy(For the Lead-In Period (at least 6 months), for the 52 weeks following stable FIX expression (months 7 to 18 after treatment with CSL222), and annually in the Posttreatment Follow-up Period (up to 5 years))
  • Percentage of participants with zero bleeds and zero FIX-treated bleeds(During the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222), and during months 7 to: 24, 36, 48, and 60 after treatment with CSL222)
  • Change in the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Change in the EQ-5D-5L Index Scores(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Change in the Patient-Reported Outcomes Measurement Information System (PROMIS)-25 total score(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Percentage of participants with endogenous FIX activity of ≥ 5%(Up to 5 years after treatment with CSL222)
  • Change from baseline in Inflammatory markers(At baseline and up to 5 years after treatment with CSL222)
  • Change from baseline in PROMIS-29 total score(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Adverse events - percentage of participants(During the Lead-in Period (at least 6 months) and during the Posttreatment Follow-up Period (up to 5 years).)
  • Number of participants with zero bleeds and zero FIX-treated bleeds(During the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222), and during months 7 to: 24, 36, 48, and 60 after treatment with CSL222)
  • Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) total score and change from baseline(At baseline and Month 18 post treatment)
  • Endogenous FIX activity(At baseline, and at months 6, 12, and 18 after treatment with CSL222)
  • Change from baseline in endogenous FIX activity(At baseline, and at months 6, 12, and 18 after treatment with CSL222)
  • Annualized consumption of FIX replacement therapy(For the Lead-In Period (at least 6 months), for the 52 weeks following stable FIX expression (months 7 to 18 after treatment with CSL222), and annually in the Posttreatment Follow-up Period (up to 5 years))
  • Annualized infusion rate of FIX replacement therapy(For the Lead-In Period (at least 6 months), for the 52 weeks following stable FIX expression (months 7 to 18 after treatment with CSL222), and annually in the Posttreatment Follow-up Period (up to 5 years))
  • Number of participants remaining free of continuous FIX prophylaxis(During the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222), and during months 7 to: 24, 36, 48, and 60 after treatment with CSL222)
  • Percentage of participants remaining free of continuous FIX prophylaxis(During the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222), and during months 7 to: 24, 36, 48, and 60 after treatment with CSL222)
  • ABR for spontaneous, joint, and FIX-treated bleeding episodes(For the Lead-In Period (at least 6 months) and during the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222))
  • Correlation of FIX activity levels and pre-CSL222 AAV5 NAb titer(At the end of the Lead-in Period and during 6 to 18 months after treatment with CSL222)
  • Number of new target joints and resolution of preexisting target joints(During the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222), and then annually during the Posttreatment Follow-up Period (up to 5 years))
  • Percentage of participants with zero bleeds and zero FIX-treated bleeds(During the 52 weeks after stable FIX expression (months 7 to 18 after treatment with CSL222), and during months 7 to: 24, 36, 48, and 60 after treatment with CSL222)
  • Change in the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Change in the EQ-5D-5L Index Scores(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Change in the Patient-Reported Outcomes Measurement Information System (PROMIS)-25 total score(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Change from baseline in PROMIS-29 total score(At baseline, during the Lead-In Period (at least 6 months), and in the Posttreatment Follow-up Period (up to 5 years))
  • Adverse events - number of participants(During the Lead-in Period (at least 6 months) and during the Posttreatment Follow-up Period (up to 5 years).)
  • Adverse events - number of events(During the Lead-in Period (at least 6 months) and during the Posttreatment Follow-up Period (up to 5 years).)
  • Change in liver ultrasound(At baseline, month 12 and every 6 months thereafter up to 5 years after CSL222 treatment)
  • Number of participants with antibodies against AAV5(From treatment with CSL222 through end of study (up to 5 years after treatment with CSL222))
  • Number of participants who develop FIX inhibitors(From treatment with CSL222 through end of study (up to 5 years after treatment with CSL222))
  • Number of clinically significant clinical laboratory tests (Hematology and Biochemistry) reported as an AE(At baseline and up to 5 years after treatment with CSL222)
  • Number of participants with clinically significant ALT/AST levels(At baseline and up to 5 years after treatment with CSL222)
  • Percentage of participants with clinically significant ALT/AST levels(At baseline and up to 5 years after treatment with CSL222)
  • Number of participants using corticosteroid for change in AST/ALT levels(Up to 5 years after treatment with CSL222)
  • Duration of corticosteroid use for change in AST/ALT levels(Up to 5 years after treatment with CSL222)
  • Mean inflammatory markers values(Up to 5 years after treatment with CSL222)
  • Change from baseline in Inflammatory markers(At baseline and up to 5 years after treatment with CSL222)

研究者

发起方
CSL Behring
申办方类型
Industry
责任方
Sponsor

研究点 (14)

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