Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

Urea Cycle Disorders Clinical Trial

Official title:

Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02489292
• Other study ID #: HEP002
• Status: Completed
• Phase: Phase 2
• Start date: October 2014
• Est. completion date: March 2017

Study information

• Verified date: October 2020
• Source: Promethera Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: March 2017
• Est. primary completion date: March 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Years

Eligibility

Main Inclusion Criteria:

• Paediatric patients < 12 years prior to infusion
• Patient presents with UCD
• Patient shows patency of the portal vein and branches, with normal flow velocity as confirmed by Doppler US and accessibility of the portal vein and /or affluants.

Main Exclusion Criteria:

• Patient has mild disease severity, easily controlled under standard of care therapy, with no recurrent metabolic crises.
• Patient is registered on a liver transplant waiting list or is scheduled for living donor liver transplantation before the end of the study.
• Patient presents acute liver failure.
• Patient presents clinical or radiological evidence of liver cirrhosis.
• Patient presents or has a history of hepatic or extrahepatic malignancy.
• Patient has a known clinically significant cardiac malformation.
• 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 PB before enrolling the patient in the study.
• Patient had or has a renal insufficiency treated by dialysis.

Related Conditions & MeSH terms

• Disease
• Urea Cycle Disorders
• Urea Cycle Disorders, Inborn

Intervention

Biological:
• HepaStem
HepaStem will be administered in maximum 4 infusion days, spread over an 8-week period with an interval of 2 to 3 weeks between infusion days. The target total dose of cells will be 50x10E6 cells/kg body weight

Locations

Country	Name	City	State
Belgium	Cliniques Universitaires Saint-Luc	Brussels
France	Hôpital Jeanne de Flandre, CHRU Lille	Lille
Poland	Instytut - Pomnik Centrum Zdrowia Dziecka	Warszawa
Spain	Hospital Materno Infatil de Badajoz	Badajoz
Spain	Hospital Universitari Vall d'Hebron de Barcelona	Barcelona
Spain	Hospital Materno Infantil de Málaga	Málaga

Sponsors (1)

Lead Sponsor	Collaborator
Promethera Biosciences

Countries where clinical trial is conducted

Belgium,  France,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy as determined by de novo ureagenesis (C13 tracer method)		at 6m post-first infusion day
Secondary	Efficacy as determined by de novo ureagenesis (C13 tracer method)		at 3, 9 and 12 months post-first infusion day
Secondary	Efficacy as determined by Ammonia (NH3) values		up to 12 months post-first infusion day
Secondary	Efficacy as determined by amino acids in plasma		up to 12 months post-first infusion day
Secondary	Efficacy as determined by report of metabolic decompensations		up to 12 months post-first infusion day
Secondary	Efficacy as determined by report on actual supportive treatment, adjustment of protein restriction and amino acids supplements		up to 12 months post-first infusion day
Secondary	Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators		up to 12 months post-first infusion day
Secondary	To evaluate the safety during the year following HepaStem infusions (composite)	Safety evaluation in terms of (1) clinical status, (2) portal vein hemodynamics, (3) morphology of the liver, bile ducts and portal system, (4) laboratory tests, (5) De novo detection of donor-specific circulating anti-human leukocyte antigen (HLA) antibodies, and/or other immune-related markers, (6) serious adverse events and clinically significant adverse events related to HepaStem, technical intervention, and concomitant treatments.	up to 12 months post-first infusion day