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

NCT02489292 | Completed Phase 2 DMC

• 1 other identifier: HEP002
• Study Type: interventional
• Target: 5
• Locations: 4 countries
• Sites: 6

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

Keywords

Urea Cycle Disorder; UCD; cell therapy; stem cell

Outcome Measures

Primary Outcomes (1)

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

  at 6m post-first infusion day

Secondary Outcomes (7)

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

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

• Efficacy as determined by Ammonia (NH3) values

  up to 12 months post-first infusion day

• Efficacy as determined by amino acids in plasma

  up to 12 months post-first infusion day

• Efficacy as determined by report of metabolic decompensations

  up to 12 months post-first infusion day

• 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

Study Arms (1)

HepaStem

EXPERIMENTAL

Target total dose 50x10E6 cells/kg

Biological: HepaStem

Interventions

HepaStem 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

HepaStem

Eligibility Criteria

• Age: Up to 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Cellaion SA: lead

Study Sites (6)

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

Location

Hôpital Jeanne de Flandre, CHRU Lille

Lille, 59037, France

Location

Instytut - Pomnik Centrum Zdrowia Dziecka

Warsaw, Poland

Location

Hospital Materno Infatil de Badajoz

Badajoz, 06010, Spain

Location

Hospital Universitari Vall d'Hebron de Barcelona

Barcelona, 08035, Spain

Location

Hospital Materno Infantil de Málaga

Málaga, 29011, Spain

Location

MeSH Terms

Conditions

Urea Cycle Disorders, Inborn

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2014
• First Posted: July 3, 2015
• Study Start: October 1, 2014
• Primary Completion: March 1, 2017
• Study Completion: March 1, 2017
• Last Updated: October 19, 2020

Record last verified: 2020-10

Locations

ES (3); FR (1); BE (1); PL (1)