EUCTR2018-004024-11-AT进行中（未招募）1 期
An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
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
- •The subject will not be considered eligible for the study without meeting all of the criteria below.
- •1. Male, aged 18 to 75 years at the time of screening.
- •2. Established severe or moderately severe hemophilia B (plasma FIX activity
- •= 2% measured following = 5 half-lives of most recent exposure to exogenous FIX)
- •and either = 3 hemorrhages per year requiring treatment with exogenous FIX or use
- •of prophylactic therapy.
- •3. History of > 150 exposure days to exogenously administered FIX
- •concentrates or
- •cryoprecipitates.
- •4. Sexually active man must agree to use a condom during sexual
- •intercourse or limit sexual intercourse to post-menopausal, surgically
- •sterilized, or contraception-practicing partners in the period from
- •SHP648 administration until AAV8 has been cleared from semen, as
- •evidenced from negative analysis results for at
- •least 2 consecutively collected semen samples assessed at the central
- •laboratory (this criterion is
- •applicable also for subjects who are surgically sterilized).
- •5. 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 19
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •The subject will be excluded from the study if any of the following
- •exclusion criteria are met.
- •1. Bleeding disorder(s) other than hemophilia B.
- •2. Documented laboratory evidence of having developed inhibitors (= 0.6
- •BU on any single test) to FIX proteins at any time.
- •3. Documented prior allergic reaction to any FIX product.
- •4. Anti-AAV8 neutralizing antibody titer > 1:5. Subjects whose
- •laboratory assessments are =1:10 may be
- •re-tested within the same Screening window and, if eligibility criterion is
- •met on retest, may be enrolled after
- •confirmation by the Sponsor's Medical Monitor.
- •5. Known hypersensitivity to prednisolone or prednisone, or to any of the
- •excipients.
- •6. Having a disease in which treatment with prednisolone or prednisone
- •is not tolerated
- •(including, but not limited to osteoporosis with vertebral fractures,
- •vascular necrosis, cataracts and glaucoma,
- •difficult to control hypertension, and diabetes as assessed by the
- •treating physician).
- •7. Active Hepatitis C, as indicated by detectable Hepatitis C virus (HCV)
- •ribonucleic acid (RNA) by reverse transcriptase
- •polymerase chain reaction (rtPCR).
- •8. Hepatitis B, as indicated by positive surface Hepatitis B virus (HBV)
- •antigen test.
- •9. Evidence of markers of potential underlying risk for autoimmune
- •mediated hepatic disease:
- •a. Anti-smooth muscle antibody (ASMA) titer = 1:40. Values of 1:31 to
- •1:39 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.
- •10. Receiving chronic systemic antiviral and/or interferon therapy within
- •4 weeks prior to enrollment.
- •11. Clinically significant infections (e.g., systemic fungal infections)
- •requiring systemic treatment.
- •12. Known immune disorder (including myeloma and lymphoma).
- •13. Concurrent chemotherapy or biological therapy for treatment of
- •neoplastic disease or other disorders.
- •14. An absolute neutrophil count < 1000 cells/mm3.
- •15. History of liver biopsy or imaging indicating moderate or severe
- •fibrosis (Metavir fibrosis stage F2 or
- •16. History of ascites, varices, variceal hemorrhage, or hepatic
- •encephalopathy.
- •17. Any of the following pre-existing diagnoses, which are indicative of
- •significant underlying liver disease, are
- •present in the medical record: portal hypertension, splenomegaly.
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研究者
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