EUCTR2011-004074-28-GB进行中（未招募）不适用
A prospective, open label, multicenter, randomized, safety and preliminary efficacy study of one cycle of Promethera HepaStem® in Urea Cycle Disorders and Crigler-Najjar Syndrome patients - UKHEP001
Promethera Biosciences0 个研究点目标入组 21 人开始时间: 2011年9月28日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 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
- All
入选标准
- •1.Subject shows patency of the portal vein and branches, with normal flow velocity in the main portal vein as confirmed by Doppler ultrasound and accessibility of the portal vein, or respectively, accessibility of the umbilical vein.
- •2.Subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent.
- •3.Female subjects of childbearing potential need to have a negative pregnancy test and must agree to use an acceptable method of contraception throughout the study.
- •Crigler-Najjar Syndrome specific:
- •4.Patient presents with Crigler-Najjar syndrome type 1 and diagnosis must be confirmed by genetic mutation analysis if not available.
- •5.Patient presents with Crigler-Najjar syndrome type 2
- •-poorly controlled under phenobarbital treatment, or
- •-experiencing serious impairment in quality of life.
- •Diagnosis must be confirmed by genetic mutation analysis if not available.
- •Urea Cycle Disorders specific
- •6.Diagnosis of one of the urea cycle disorders (CPSID, OCTD, ASSD, ASLD, Arginase deficiency and NAGSD)
- •- of which the disease is of such severity to warrant liver transplantation or alternatives despite full conservative therapy, or
- •- subject experiencing serious impairment in quality of life despite full conservative therapy.
- •Diagnosis must be confirmed by genetic mutation analysis if not available.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 20
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.The subject is 16 years or older at time of screening.
- •2.The subject presents acute liver failure.
- •3.The subject presents clinical or radiological evidence of liver fibrosis or cirrhosis
- •4.The subject presents or has a history of hepatic or extrahepatic malignancy
- •5.The patient has a non-corrected cardiac malformation.
- •6.The subject has a known medical or family history of coagulopathy.
- •7.The subject participates currently in another clinical trial – except disease registry and observational HepaStem study.
- •8.The subject underwent previous mature liver cell or stem cell transplantation or received an organ liver transplant.
- •9.The subject has a contraindication to immunosuppressive therapy.
- •10.The subject has a known hypersensitivity or allergy to the recommended antibiotics to prevent post-operative infections according to institutional guidelines, and basiliximab, solumedrol or tacrolimus unless alternative drugs can be used without risk for the patient.
- •11.The subject has a known hypersensitivity or allergy to bivalirudin.
- •12.The subject had or has a renal insufficiency treated by dialysis.
- •13.The subject requires valproate therapy.
- •14.The subject has a known hypersensitivity or allergy to contrast agents that cannot be treated adequately.
- •15.The subject has a thrombosis of the portal vein or persisting impairment of anterograde portal blood flow.
- •16.The subject has a porto systemic shunt or fistula assessed by Doppler US.
- •17.For umbilical vein access: The subject has any contraindication for umbilical vein catheterization (eg omphalitis, peritonitis, necrotizing enterocolitis etc)
- •18.Any significant condition which in the Investigator’s opinion may interfere with the subject’s optimal participation in the study
- •19. Patients with disease of such severity that liver transplantation is an absolute indication.
- •20. Patients with mild disease severity, easily controlled under standard of care therapy with no recurrent metabolic crises.
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