NL-OMON51102撤回3 期
A prospective, lead-in study to collect bleeding episodes, Factor VIII (FVIII) infusions, and patient-reported outcomes in patients with hemophilia A - Get-8 Lead In
适应症
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- Bayer
- 入组人数
- 13
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 18 至 64（—）
入选标准
- •- Participant must be *18 years of age at the time of signing the informed
- •- 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.
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
- •- 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
- 另有 3 项未显示
研究者
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