KCT0003619撤回未知
A prospective, open-label study of the safety and efficacy of Hepastem infusion in pediatric patients with urea cycle disorders.
HLB CE0 个研究点目标入组 5 人开始时间: 待定最近更新:
试验速览
- 阶段
- 未知
- 状态
- 撤回
- 发起方
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional Study
入排标准
- 年龄范围
- o Limit 至 12(Year)（—）
- 性别
- All
入选标准
- •1.The patient is a pediatric patient<12 years prior to infusion.
- •2.The patient presents with one of the following UCDs(CPCS1D:carbamoylphosphate synthetase 1 deficiency, OTCD:ornitine transcarbamylase deficiency, ASSD:argininosuccinic acid synthetase deficiency, ASLD:arfininosuccinic acid lase deficiency, ARGD:arginase deficiency)
- •3.The patient has severe disease with inpaired protein tolerance defined as : chronic protein restricted diet and chronic treatment with at least one nitrogen scavenger.
- •4.The patient shows patency of the portal vein and its branches including mesenteric veins, with normal flow velocity as confirmed by Doppler Ultra Sono and accessibility of the portal vein and/ or affluants.
- •5.The patients (if capable of signing) and parents or legal representative have signed a written informed assent/consent form.
排除标准
- •1.The patient presents acute liver failure.
- •2.The patient presents clinical or radiological evidence of liver cirrhosis.
- •3.The patient presents or has a history of hepatic or extrahepatic malignancy.
- •4.The patient has a known clinically significant cardiac malformation.
- •5.The patient has a personal history of venous thrombosis, or has a clinically signifiant abnormal value of 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 LL before enrolling the patient in the study.
- •6.Patients currently receiving other unlicensed clinical trial drugs.
- •7.The patient underwent previous mature liver cell or stem cell transplantation or received an organ liver transplant or eceived Hepastem infusion.
- •8.The patient has a contraindication to methylprednisolone, tacrolimus.
- •9.The patient has a known hypersensitivity or allergy to heparin.
- •10.The patient has a known hypersensitivity or allergy to the antiviotics preventing post-operative infections that are prescribed according to institutional guidelines, and no alternative prophylaxis can be found.
- •11.The patient had or has a renal insufficiency treated by dialysis.
- •12.The patient requires valproate therapy.
- •13.The patient has a knon hyperwensitivity or allergy to contrast agents ( if applicable) that cannot be treated adequately.
- •14.The patient has a thrombosis of the portal vein or persisting impairment of anterograde portal blood flow.
- •15.The patient has a porto systemic shunt or fistula assessed by Doppler US or an Arantius channel or portal hypertension.
- •16.The site where the catheter in intended to be placed has previously suffered from venous thrombosis or vascular surgical procedures.
- •17.The patient has an ongoing infection or sufferd from an infection in the last 2 weeks(including active EBV infection at screening). The patient may be enrolled after resolution of the infection.
- •18.There in any significant condition or disbility that, in the Investigator's opinion, may interfere with the patient's optimal participation in the study.
研究者
相似试验
进行中（未招募）
1 期
An open-label study to characterize the safety and response rate of MabThera (Rituximab) plus chlorambucil in previously untreated patients with CD20-positive B-cell chronic lymphocytic leukemia. - not applicableCD20-positive B-cell chronic lymphocytic leukemiaEUCTR2007-000172-16-GBF. Hoffmann-La Roche Limited100
进行中（未招募）
1 期
Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)Fabry disease (a-galactosidase A deficiency)MedDRA version: 20.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2016-001318-11-DEProtalix Ltd.22
进行中（未招募）
1 期
Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)Fabry disease (a-galactosidase A deficiency)MedDRA version: 19.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2016-001318-11-CZProtalix Ltd.22
进行中（未招募）
1 期
Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)Fabry disease (a-galactosidase A deficiency)MedDRA version: 20.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2016-001318-11-SIProtalix Ltd.22
进行中（未招募）
1 期
Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)EUCTR2016-001318-11-ESProtalix Ltd.22