HEP002 is a clinical study designed for paediatric patients with urea cycle disorders (UCD). UCD patients will receive several infusions of HepaStem. The efficacy as well as the safety of the medicinal product will be assessed during the year following infusions.
- Conditions
- The urea cycle disorders are inborn errors of metabolism that affect the transfer of nitrogen into urea. There are eight disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, HHH syndrme and citrullinemia type II.Mutations in each of the enzymes have been identified, resulting in failed production of urea, the end product of the urea cycle.MedDRA version: 18.0Level: LLTClassification code 10013373Term: Disorders of urea cycle metabolismSystem Organ Class: 100000004861Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2014-000650-11-ES
- Lead Sponsor
- Promethera Biosciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 6
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
deficiency
- 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
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method