EUCTR2018-003086-33-GB进行中（未招募）1 期
Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe hemophilia B (FIX:C <=2%) (BeneGene-2) - BeneGene-2
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 50
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
- •1. Participants must have completed at least 6 months of routine FIX prophylaxis
- •therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study.
- •2. Participants who have documented moderately severe to severe hemophilia B, defined as
- •FIX:C <=2%.
- •3. Participants must agree to suspend prophylaxis therapy for hemophilia B after
- •administration of the IP. FIX replacement therapy is allowed as needed
- •4. Acceptable screening laboratory values as follows:
- •Hemoglobin >=11 g/dL;
- •Platelets >=100,000 cells/mL;
- •Creatinine = 2.0 mg/dL.
- •5. Sex: Male. Contraceptive use by men should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Male participants are eligible to participate if they agree to the following for at least
- •time required for 3 consecutive ejaculate samples to test negative for vector shedding: Refrain from donating sperm. PLUS either: Be abstinent from heterosexual or homosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent.
- •OR Must agree to use contraception/barrier as detailed below:
- •Agree to use male condom when engaging in any activity that allows for
- •passage of ejaculate to another person
- •6. 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 50
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Anti-AAV-Spark100 neutralizing antibodies (nAb) titer=1:1 (i.e., positive for nAb), performed by a central laboratory during screening.
- •2. Prior history of inhibitor to FIX or positive inhibitor testing as measured by the
- •central laboratory >=0.6 Bethesda Units (BU) during screening. Clinical signs or
- •symptoms of decreased response to FIX.
- •3. Known hypersensitivity to FIX replacement product or intravenous immunoglobulin administration.
- •4. History of chronic infection or other chronic disease that investigator deems as an
- •unacceptable risk.
- •5. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation or other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal deation or behavior (including alcoholism) or laboratory abnormality that may ncrease the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
- •6. Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase(ALP) >2x upper limit of normal (ULN)based on central laboratory results.
- •7. Bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is
- •fractionated and direct bilirubin <35%) based on central laboratory results.
- •8. Current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, hepatic encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: Stable chronic liver disease including Gilbert's syndrome, asymptomatic gallstones, is acceptable if the participant otherwise meets entry criteria). Note: Participants who have a central laboratory test value that is outside the range specified by the exclusion criteria may have the test repeated, by the central laboratory, to determine eligibility; however, the result must be available prior to Baseline Visit /Visit 2.
- •9. Currently on antiviral therapy for hepatitis B or C.
- •10. Any participants with a planned surgical procedure requiring FIX surgical prophylactic
- •factor treatment in the next 12 months.
- •11. Participants using therapies that are restricted. See Section 6.5.2 for therapies not
- •allowed during study participation.
- •12. Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within the last 12 weeks, excluding participation in study C0371004.
- •13. Active hepatitis B or C; HBsAg, HBV-DNA positivity, or HCV-RNA positivity.
- •14. Significant liver disease, as defined by pre-existing diagnosis of portal hypertension, splenomegaly or hepatic encephalopathy. Additionally during screening, a serum albumin level below normal limits and/or significant liver fibrosis by any of the following diagnostic modalities: FibroScan score >8 kPa units, Fibro Test/FibroSURE >0.48 or AST-to-Platelet Ratio Index (APRI) >1.In the investigator's opinion, if there is concern regarding the FibroTest results due to a confounding medical history (e.g., proteinuria can impact FibroTest result), the investigator can perform a different assessment of liver fibrosis (e.g., FibroScan or APRI) during the screening period.*Please note: if a participant has a known history of Gilbert's syndrome, a FibroTest cannot be used for fibrosis testing. However, the participant could be tested us
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