EUCTR2019-004480-48-ES进行中（未招募）1 期
A prospective, lead-in study to collect bleeding episodes, Factor VIII (FVIII) infusions, and patient-reported outcomes in patients with hemophilia A
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Bayer AG
- 入组人数
- 140
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
- •- Participant must be =18 years of age at the time of signing the informed consent.
- •- Patients with severe hemophilia A (FVIII:C =1% baseline FVIII activity [FVIII:C] as determined by measurement at the time of screening or from reliable prior documentation in clinical records of the patients).
- •- Previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 150 exposure days (ED) as documented in reliable prior clinical records of the patients.
- •- On regular prophylaxis with FVIII (defined as = 45 weeks/year of treatment with an adequate dose as per label ) and on stable treatment for at least 6 months as documented in reliable prior clinical records of the patients
- •- Well-managed patients with at least 1 documented visit at the hemophilia treatment center in the year prior to enrollment.
- •- Willing to participate in the interventional Phase 3 gene therapy study with BAY2599023.
- •- Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- •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 130
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 10
排除标准
- •Participants are excluded from the study if any of the following criteria apply. Exclusion criteria marked with an asterisk (*) will lead to exclusion of the participant from the subsequent Phase 3 study, if occurring at any time during the lead-in study. If the criteria marked with an asterisk are met during the study, the participant will withdraw from the study.
- •Medical Conditions:
- •- *Current inhibitor to FVIII with a titer = 0.6 BU, confirmed by more than 1 test.
- •- History of inhibitor with a titer >1.0 BU (as documented in reliable prior clinical records of the patients) or with a repeated titer 0.6 to <1.0 BU in more than one subsequent occasion.
- •- *Significant underlying liver disease, as evidenced by any of the following: portal hypertension, splenomegaly, ascites, esophageal varices, hepatic encephalopathy,
- •reduction below normal limits of serum albumin or an advanced liver disease (Child-Pugh Grade B and C), suspicion of liver malignancy or fibrosis by ultrasound / Fibroscan.
- •- Any of the following:
- •-- Hemoglobin <11 g/dL
- •-- Platelets <100,000 cells/µL
- •-- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >1.5× ULN
- •-- Alkaline phosphatase (AP) >2.5 × ULN
- •-- Total bilirubin >1.5 × ULN
- •-- Prothrombin time (PT) or international normalized ratio (INR) >1.0 × ULN
- •-- Creatinine >1.5 mg/dL
- •- Another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B)
- •- *Active hepatitis B or C infection, as reflected by HBsAg or HCV-RNA viral load positivity
- •- Serological evidence of active HIV-1 or HIV-2 as measured by CD4+ cell count <200 cells/mm3 and/or viral load >50 gc/mL.
- •- *Pre-existing immunity against AAVhu37.
- •- *Any current diagnosis of malignancy.
- •- Known or suspected autoimmune diseases requiring immunosuppressive therapy.
- •- Body mass index > 35 kg/m^2.
- •- Contraindication for corticosteroid treatment.
- •- *Any other significant medical condition that would be a risk to the patient or would affect patient’s ability to receive gene transfer following completion of his participation in this lead-in study.
- •Prior/Concomitant Therapy
- •Listed therapies/medications are not allowed at enrollment, during the study and will also not allow transition to the Phase 3 study if introduced during the lead-in study:
- •- Antiviral therapy for hepatitis B or C ,
- •- Pre-medication to tolerate FVIII treatment,
- •- Immunomodulatory drugs (other than corticosteroids),
- •- Efavirenz,
- •- Emicizumab,
- •- Planned major surgery.
- •Prior/Concurrent Clinical Study Experience
- •- Participation in any investigational hemophilia product study within 3 months before screening (participation in any other investigational product study will not be allowed throughout the study).
- •- Has received the same or another gene therapy product.
研究者
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