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

Primary Purpose

Urea Cycle Disorders

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

HepaStem

About this trial

This is an interventional treatment trial for Urea Cycle Disorders focused on measuring Urea Cycle Disorder, UCD, cell therapy, stem cell

Eligibility Criteria

undefined - 12 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Cliniques Universitaires Saint-Luc
Hôpital Jeanne de Flandre, CHRU Lille
Instytut - Pomnik Centrum Zdrowia Dziecka
Hospital Materno Infatil de Badajoz
Hospital Universitari Vall d'Hebron de Barcelona
Hospital Materno Infantil de Málaga

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

HepaStem

Arm Description

Target total dose 50x10E6 cells/kg

Outcomes

Primary Outcome Measures

Efficacy as determined by de novo ureagenesis (C13 tracer method)

Secondary Outcome Measures

Efficacy as determined by de novo ureagenesis (C13 tracer method)

Efficacy as determined by Ammonia (NH3) values

Efficacy as determined by amino acids in plasma

Efficacy as determined by report of metabolic decompensations

Efficacy as determined by report on actual supportive treatment, adjustment of protein restriction and amino acids supplements

Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators

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.

Full Information

NCT ID

NCT02489292

First Posted

November 6, 2014

Last Updated

October 13, 2020

Sponsor

Cellaion SA

1. Study Identification

Unique Protocol Identification Number

NCT02489292

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

October 2020

Overall Recruitment Status

Completed

Study Start Date

October 2014 (Actual)

Primary Completion Date

March 2017 (Actual)

Study Completion Date

March 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Cellaion SA

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Urea Cycle Disorders

Keywords

Urea Cycle Disorder, UCD, cell therapy, stem cell

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

HepaStem

Arm Type

Experimental

Arm Description

Target total dose 50x10E6 cells/kg

Intervention Type

Biological

Intervention Name(s)

HepaStem

Intervention Description

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

Primary Outcome Measure Information:

Title

Efficacy as determined by de novo ureagenesis (C13 tracer method)

Time Frame

at 6m post-first infusion day

Secondary Outcome Measure Information:

Title

Efficacy as determined by de novo ureagenesis (C13 tracer method)

Time Frame

at 3, 9 and 12 months post-first infusion day

Title

Efficacy as determined by Ammonia (NH3) values

Time Frame