EUCTR2017-004305-40-DE进行中（未招募）1 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 - Phase III trial of AMT-061 in subjects with severe or moderately severe hemophilia B.
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 75
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •- Age =18 years
- •- Subjects with congenital hemophilia B with known severe or moderately severe factor IX deficiency (=2% of normal circulating factor IX) for which the subject is on continuous routine factor IX prophylaxis (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).
- •- >150 previous exposure days of treatment with factor IX protein
- •- Have been on stable prophylaxis for at least 2 months prior to screening
- •- Have demonstrated capability to independently, accurately and in a timely manner complete the diary during the lead-in phase as judged by the investigator
- •- 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 3 consecutively collected semen samples (this criterion is applicable also for subjects who are surgically sterilized)
- •- Able to provide informed consent following receipt of verbal and written information about the trial.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 63
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 12
排除标准
- •- History of factor IX inhibitors
- •- Positive factor IX inhibitor test at screening and Visit L-Final (based on
- •local laboratory results)
- •- Screening and Visit L-Final laboratory values (based on central
- •laboratory results):
- •a. ALT >2 times upper normal limit
- •b. Aspartate aminotransferase (AST) >2 times upper normal limit
- •c. Total bilirubin >2 times upper normal limit (except if this is caused by Gilbert disease)
- •d. Alkaline phosphatase (ALP) >2 times upper normal limit
- •e. Creatinine >2 times upper normal limit
- •- Positive human immunodeficiency virus (HIV) serological test at
- •screening and Visit LFinal, not controlled with anti-viral therapy as
- •shown by CD4+ counts = 200/µL (based on central laboratory results)
- •- Hepatitis B or C infection with the following criteria present at Screening:
- •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 extracellular antigen (HBeAg),
- •Hepatitis B virus deoxyribonucleic acid (HBV DNA)
- •Hepatitis C virus ribonucleic acid (HCV RNA)
- •- Known coagulation disorder other than hemophilia B
- •- Thrombocytopenia, defined as a platelet count below 50 × 109/L, at
- •screening and Visit L-Final (based on central laboratory results)
- •- 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 the IMP
- •- 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; e.g. a FibroScan™ score of =9 kPa is considered equivalent)
- •- Known history of an allergic reaction or anaphylaxis to factor IX
- •- Known uncontrolled allergic conditions or allergy/hypersensitivity to
- •any component of the IMP excipients
- •- Known history of allergy to corticosteroids
- •- Known medical condition that would require chronic administration of
- •- Previous gene therapy treatment
- •- Receipt of an experimental agent within 60 days prior to screening
- •- 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
相似试验
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 BBleeder's diseaseChristmas disease1006447710005330NL-OMON52495CSL Behring LCC15
进行中（未招募）
1 期
A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.MedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disordersHemophilia BEUCTR2017-004305-40-BECSL Behring LLC75
招募中
1 期
A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.Haemophilia BMedDRA version: 20.0Level: LLTClassification code: 10018939Term: Haemophilia B (Factor IX) Class: 10010331CTIS2024-510738-42-00CSL Behring LLC71
进行中（未招募）
1 期
A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.MedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disordersHemophilia BEUCTR2017-004305-40-IECSL Behring LLC75
进行中（未招募）
1 期
A phase III trial investigating a gene therapy (AAV-hFIXco-Padua, AMT-061), in adult patients with severe or moderately severe haemophilia B, to firstly evaluate if it's effective, and secondly further describe it's safety profile.Hemophilia BEUCTR2017-004305-40-DKCSL Behring LLC75