Efficacy and safety study of infusions of Hepastem in Urea Cycle Disorders pediatric patients.
- Conditions
- Endocrine, nutritional and metabolic disease
- Registration Number
- KCT0003619
- Lead Sponsor
- HLB CE
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- All
- Target Recruitment
- 5
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.
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.
Study & Design
- Study Type
- Interventional Study
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method reagenesis improvement at 6 month post first infusion day(Follow up visit3) : absolute 13C blood urea AUC(area under the curve)-120 min quantified with the 13C tracer method at follow up visits compared with baseline evaluations(measurements at baseline visit1, baseline visit2 and baseline visit3).;Safety : Clinical tatus(physical examination and vital signs);Safety : Morphology of the liver, bile ducts, and portal system;Safety : Laboratory tests;de novo detection of donor-specific circulationg anti-human leukocyte antigen(HLA) antibodies at mean fluorescence intensity(MFI)>1500, and/or other immune-related markers;Safety : Serios Adverse Events(SAEs) and clinically significant Adverse Events(AEs) related to Hepastem (infusion and follow-up), technical intervention, and concomitant treatments.;Safety : Portal vein hemodynamics
- Secondary Outcome Measures
Name Time Method