A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion - Not Available.

BAXALTA INNOVATIONS GMBH0 个研究点目标入组 10 人开始时间: 2021年9月3日最近更新: 2021年9月13日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: BAXALTA INNOVATIONS GMBH
入组人数: 10

研究概览入排标准研究者记录与标识符相似试验

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. Male, aged 18 to 75 years at the time of screening.
•2. Established severe hemophilia A (FVIII:C <1%, measured following =5 days without FVIII treatment) and/or documented intron 1 inversion or intron 22 inversion mutation in the F8 gene, consistent with severe hemophilia A AND documented evidence of =3 hemorrhages over the previous 12 months requiring treatment with exogenous FVIII or use of FVIII prophylaxis because of history of frequent bleeding episodes.
•3. History of >150 exposure days to exogenously administered FVIII concentrates or cryoprecipitate.
•4. Normal prothrombin time.
•5. Sexually active men must agree to use barrier contraception (combination of a condom and spermicide) or limit sexual intercourse to post-menopausal, surgically sterilized, or contraception-practicing partners for a minimum of 6 months after administration of BAX 888, or until BAX 888 genomes are
•no longer detected in the semen, whichever is sooner.
•6. Willing and able to comply with the requirements of the protocol, including provision of semen samples, maintenance of a diary of bleeding episodes and FVIII protein use.
•7. Signed informed consent.
•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 9
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

排除标准

•1. Bleeding disorder(s) other than hemophilia A.
•2. Personal laboratory evidence of having developed inhibitors to FVIII protein at any time (=0.6 Bethesda Units on any single test).
•3. Documented prior allergic reaction to any FVIII product.
•4. AAV8 neutralizing antibody titer greater than or equal to 1:5.
•5. Positive AAV8-specific T-cell ELISPOTs for any AAV8 peptide pools.
•6. Known hypersensitivity to prednisone, prednisolone, or belatacept.
•7. Having a disease in which treatment with prednisone, prednisolone, or belatacept is not tolerated (including but not limited to osteoporosis with vertebral fractures, severe labile hypertension, and brittle diabetes).
•8. Evidence of markers of potential underlying risk for autoimmune mediated hepatic disease:
•a. Anti-smooth muscle antibody assay results =40 (Inova QUANTA LiteTM Actin IgG enzymelinked immunosorbent assay); values of 31 to 39 will be flagged as possibly abnormal and the Investigator and Medical Monitor will evaluate the subject for eligibility.
•b. Elevated anti-liver-kidney microsomal antibody type 1 (LKM1) titers.
•c. Total IgG >1.5x ULN.
•d. Antinuclear antibody (ANA) titer >1:320; OR ANA titer > 1:80 if demonstrated concurrently with ALT that is > ULN.
•9. Active Hepatitis C: As indicated by detectable HCV ribonucleic acid by polymerase chain reaction.
•10. Hepatitis B: If surface antigen is positive.
•11. Epstein Barr Virus seronegative (if belatacept is instituted as per protocol for immunosuppression).
•12. Seropositive for Human Immunodeficiency Virus (HIV).
•13. Receiving systemic antiviral and/or interferon therapy within 4 weeks prior to enrollment.
•14. Clinically significant infections (e.g., systemic fungal infections) requiring systemic treatment.
•15. Known immune disorder (including myeloma and lymphoma).
•16. Concurrent chemotherapy or biological therapy for treatment of neoplastic disease or other disorders.
•17. An absolute neutrophil count <1000 cells/mm3.
•18. Markers of hepatic inflammation or cirrhosis as evidenced by 1 or more of the following:
•a. Platelet count of <150,000/µL.
•b. Albumin =3.5 g/dL.
•c. Total bilirubin >1.5x ULN and direct bilirubin =0.5 mg/dL.
•d. ALT or AST >1.0x ULN.
•e. Alkaline phosphatase (AP) >2.0x ULN.
•f. History of liver biopsy indicating moderate or severe fibrosis (Metavir staging of F2 or greater).
•g. History of ascites, varices, variceal hemorrhage, or hepatic encephalopathy.
•h. FibroSURE Score of =0.4
•19. Serum creatinine >1.5 mg/dL.
•20. Urine protein >30 mg/dL OR >0.5 g/day.
•21. Body mass index >38.
•22. Orthopedic surgery or other major surgery planned within 6 months after enrollment.
•23. Acute or chronic disease that, in the opinion of the investigator, would adversely affect subject safety or compliance or interpretation of study results.
•24. Received an AAV vector previously or any other gene transfer agent in the previous 12 months prior to Study Day 0.
•25. Received an investigational intervention or participated in another clinical trial within 4 weeks prior to enrollment or within 5 half-lives of the investigational drug administration, whichever is longer.
•26. Significant cardiovascular disease (such as New York Heart Association Class III or IV cardiac disease, congestive heart failure, myocardial infarction within the previous 6 months, unstable arrhythmias, or unstable angina) or significant pulmonary disease (including obstructive pulmonary disease).
•27. Recent history of psychiatric illn

研究者

发起方

BAXALTA INNOVATIONS GMBH

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