NL-OMON52495招募中3 期
Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B - AMT6102 HOPE-B
CSL Behring LCC0 个研究点目标入组 15 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •2. Age >=18 years
- •3. Subjects with congenital hemophilia B with known severe or moderately severe
- •FIX deficiency (<=2% of normal circulating FIX) for which the subject is on
- •continuous routine Factor IX prophylaxis*
- •4. >150 previous exposure days of treatment with FIX protein
- •5. Have been on stable prophylaxis for at least 2 months prior to screening
- •6. Have demonstrated capability to independently, accurately and in a timely
- •manner complete the diary during the lead-in phase as judged by the investigator
- •7. Acceptance to use a condom during sexual intercourse in the period from IMP
- •administration until AAV5 has been cleared from semen, as evidenced by the
- •central laboratory from negative analysis results for at least three
- •consecutively collected semen samples (this criterion is applicable also for
- •subjects who are surgically sterilized)
- •8. Able to provide informed consent following receipt of verbal and written
- •information about the trial,
- •*Continuous routine prophylaxis is defined as the intent of treating with an a
- •priori defined frequency of infusions (e.g., twice weekly, once every two
- •weeks, etc.) as documented in the medical records
排除标准
- •1. History of FIX inhibitors
- •2. Positive FIX inhibitor test at screening and Visit L-Final (based on local
- •laboratory results)
- •3. Screening and Visit L-Final laboratory values (based on central laboratory
- •a. ALT >2 times ULN
- •b. Aspartate aminotransferase (AST) >2 times ULN
- •c. Total bilirubin >2 times ULN (except if this is caused by Gilbert disease)
- •d. Alkaline phosphatase (ALP) >2 times ULN
- •e. Creatinine >2 times ULN limit
- •4. Positive human immunodeficiency virus (HIV) serological test at screening
- •and Visit L-Final, not controlled with anti-viral therapy as shown by CD4+
- •counts <= 200/µL (based on central laboratory results)
- •5. 5. Hepatitis B or C infection with the following criteria present at
- •i. Currently receiving antiviral therapy for this/these infection(s)
- •ii. Positive for any of the following (based on central laboratory results):
- •Hepatitis B surface antigen (HBsAg), except if in the opinion of the
- •investigator this is due to a previous Hepatitis B vaccination rather than
- •active Hepatitis B infection
- •Hepatitis B virus deoxyribonucleic acid (HBV DNA)
- •Hepatitis C virus ribonucleic acid (HCV RNA)
- •6. Known coagulation disorder other than hemophilia B
- •7. Thrombocytopenia, defined as a platelet count below 50 × 109/L, at screening
- •and Visit L-Final (based on central laboratory results)
- •8. Known severe infection 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 protocol procedures or with the degree of tolerance to
- •9. Known significant medical condition that may significantly impact the
- •intended transduction of the vector and/or expression and activity of the
- •protein, including but not limited to:
- •Disseminated intravascular coagulation
- •Accelerated fibrinolysis
- •Advanced liver fibrosis (suggestive of or equal to METAVIR Stage 3 disease; a
- •FibroScan* score of >=9 kPa is considered equivalent)
- •10. Known history of an allergic reaction or anaphylaxis to factor IX products
- •11. Known history of allergy to corticosteroids
- •12. Known uncontrolled allergic conditions or allergy/hypersensitivity to any
- •component of the IMP excipients
- •13. Known medical condition that would require chronic administration of
- •14. Previous gene therapy treatment
- •15. Receipt of an experimental agent within 60 days prior to screening
- •16. Current participation or anticipated participation within one year after
- •IMP administration in this trial in any other interventional clinical trial
- •involving drugs or devices.
研究者
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