Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 2:44 PM
Ignite Modification Date:
2025-12-24 @ 2:44 PM
NCT ID:
NCT03884959
Eligibility Criteria:
Inclusion Criteria:
* The patient is a pediatric patient \<12 years
* The patients presents with one of the following UCDs. (CPS1D, OTCD, ASSD, ASLD, ARGD)
* The patient has severe disease with impaired protein tolerance defined as: chronic protein restricted diet AND chronic treatment with at lease one nitrogen scavenger.
* 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 affluents.
* The patient (if capable of signing) and parents or legal representative have signed a written informed consent form.
Exclusion Criteria:
* The patient presents acute liver failure.
* The patient presents clinical or radiological evidence of liver cirrhosis.
* The patient presents or has a history of hepatic or extrahepatic malignancy.
* The patient has a known clinically significant cardiac malformation.
* The patient has a personal history of venous thrombosis, or has a clinically significant abnormal value for protein S, protein C, anti-thrombin 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 sponsor before enrolling the patient in the study.
* Patient currently receiving other unapproved investigational drug or device.
* The patient underwent previous mature liver cell or stem cell transplantation or received an organ liver transplant or received HepaStem infusion.
* The patient has a contraindication to methylprednisolone, tacrolimus.
* The patient has a known hypersensitivity or allergy to heparin.
* 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.
* The patient had or has a renal insufficiency treated by dialysis.
* The patient requires valproate therapy.
* The patient has a known hypersensitivity or allergy to contrast agents (if applicable) that cannot be treated adequately.
* The patient has a thrombosis of the portal vein or persisting impairment of anterograde portal blood flow.
* The patient has a porto systemic shunt or fistula assessed by Doppler US or an Arantius channel or protal hypertension.
* The site where the catheter is intended to be placed has previously suffered from venous thrombosis or vascular surgical procedures.
* 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.
* There is any significant condition or disability that, in the investigator's opinion, may interfere with the patient's participation in the study.
Healthy Volunteers:
False
Sex:
ALL
Maximum Age:
12 Years
Study:
NCT03884959
Study Brief:
Protocol Section:
NCT03884959