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

NCT ID: NCT02489292

Last Updated: 2020-10-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-10-31
• Study Completion Date: 2017-03-31

Brief Summary

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

Conditions

Urea Cycle Disorders

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HepaStem

Target total dose 50x10E6 cells/kg

Group Type: EXPERIMENTAL

HepaStem

Intervention Type: BIOLOGICAL

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

Interventions

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

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.

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.

• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cellaion SA

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Hôpital Jeanne de Flandre, CHRU Lille

Lille, , France

Instytut - Pomnik Centrum Zdrowia Dziecka

Warsaw, , Poland

Hospital Materno Infatil de Badajoz

Badajoz, , Spain

Hospital Universitari Vall d'Hebron de Barcelona

Barcelona, , Spain

Hospital Materno Infantil de Málaga

Málaga, , Spain

Countries

Belgium; France; Poland; Spain

Other Identifiers

HEP002

Identifier Type: -

Identifier Source: org_study_id