EUCTR2014-000650-11-ES进行中（未招募）1 期
A prospective, open label, multi-country, multicenter, efficacy and safety study of several infusions of Promethera HepaStem in Urea Cycle Disorders pediatric patients
Promethera Biosciences0 个研究点目标入组 6 人开始时间: 2014年10月20日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 6
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
- •A patient who meets all of the following inclusion criteria will be eligible
- •for participation in the study:
- •1. The patient is a pediatric patient <12 years prior to infusion.
- •2. The patient presents with one of the following UCDs
- •(carbamoylphosphate synthetase I [CPS I] deficiency [CPS ID],
- •ornithine transcarbamylase [OTC] deficiency [OTCD], argininosuccinic
- •acid synthetase [ASS] deficiency [ASSD], argininosuccinic acid lyase
- •[ASL] deficiency [ASLD], arginase [ARG] deficiency [ARGD],
- •Nacetylglutamate
- •synthase [NAGS] deficiency [NAGSD]) and citrine
- •- of such severity that treatment alternatives are warranted despite full
- •conservative therapy, and/or
- •-experiences serious impairment in quality of life (QoL) despite full
- •conservative therapy.
- •3. The patient shows patency of the portal vein and its branches
- •including mesenteric veins, with normal flow velocity as confirmed by
- •Doppler US and accessibility of the portal vein and /or affluants.
- •4. The patient (if capable of signing) and parents or legal representative
- •have signed a written informed assent/consent form.
- •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
- •A patient who meets any of the following exclusion criteria will not be enrolled in the study:
- •1. The patient has mild disease severity, easily controlled under standard of care therapy, with no recurrent metabolic crises.
- •2. The patient is registered on a liver transplant waiting list or is scheduled for living donor liver transplantation before the end of the study.
- •3. The patient presents acute liver failure.
- •4. The patient presents clinical or radiological evidence of liver cirrhosis.
- •5. The patient presents or has a history of hepatic or extrahepatic malignancy.
- •6. The patient has a known clinically significant cardiac malformation.
- •7. The patient has a personal history of venous thrombosis, or has a clinically significant abnormal value for protein S, protein C, antithrombin III, and /or activated Protein C Resistance (aPCR) at screening. In case of known family history, a complete coagulation work-up should be performed. In all above described cases, results need to be discussed with PB before enrolling the patient in the study.
- •8. The patient participates currently in another clinical trial – except disease registry and observational HepaStem study.
- •9. The patient underwent previous mature liver cell or stem cell transplantation or received an organ liver transplant or received HepaStem infusion.
- •10. The patient has a contraindication to methylprednisolone, tacrolimus.
- •11. The patient has a known hypersensitivity or allergy to bivalirudin.
- •12. The patient has a known hypersensitivity or allergy to the antibiotics
- •preventing post-operative infections that are prescribed according to institutional guidelines, and no alternative prophylaxis can be found.
- •13. The patient had or has a renal insufficiency treated by dialysis.
- •14. The patient requires valproate therapy.
- •15. The patient has a known hypersensitivity or allergy to contrast
- •agents (if applicable) that cannot be treated adequately.
- •16. The patient has a thrombosis of the portal vein or persisting impairment of anterograde portal blood flow.
- •17. The patient has a porto systemic shunt or fistula assessed by Doppler US.
- •18. The site where the catheter is intended to be placed has previously suffered from venous thrombosis or vascular surgical procedures.
- •19. The patient has an ongoing infection or suffered from an infection in the last 2 weeks (including active EBV infection at screening). The patient may be enrolled after resolution of the infection.
- •20. There is any significant condition or disability that, in the Investigator's opinion, may interfere with the patient's optimal participation in the study.
研究者
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