Clinical Trial: Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Prospective, Open Label, Multicenter, Partially Randomized, Safety Study of One Cycle of Promethera HepaStem in Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (C

Brief Summary:

The purpose of this study is to assess the safety and to appraise the efficacy of one cycle of Hepastem (Heterologous Human Adult Liver-derived Progenitor Cells, HHALPC) infusions in paediatric patients suffering from CN or UCD.

The study duration: 12 months starting from the day of treatment: 6 months active surveillance and 6 months observation post-infusion.


Detailed Summary:
Sponsor: Promethera Biosciences

Current Primary Outcome: Safety of HepaStem in paediatric patients suffering from CN or UCD [ Time Frame: 6 months ]

Evaluation of the clinical status, portal-vein hemodynamics, morphology of the liver, de novo detection of circulating anti-HLA antibodies, and/or other immune related markers as well as Serious Adverse Events (SAEs) and clinically significant Adverse Events (AEs) related to infusion.


Original Primary Outcome:

  • Adverse events and serious adverse events related to infusion and concomitant treatment [ Time Frame: up to 6 month post infusion ]
    Assessed by physical exam, vital signs , Clinical Lab tests and Morphology of liver, bile ducts and portal system by ultrasound
  • Safety of the technical intervention [ Time Frame: During 6 months for four time windows ]
    Reactogenicity of infusion,short and mid term safety of infusion


Current Secondary Outcome:

  • Long-term safety profile of HepaStem in both indications [ Time Frame: From 6 to 12 months post-administration ]
    Assessment of reactogenicity and safety of the treatment during 6 to 12 months post infusion (long-term safety) is evaluated.
  • Preliminary efficacy of HepaStem in both indications (CN and UCD) and for different weight cohorts [ Time Frame: 0-6 months, 6-12 months ]

    UCD: 13C tracer test to measure ureagenesis, ammonium values, amino acids in plasma, neuropsychological assessment and quality of life indicators: (1) report on actual supportive treatment and any adjustment of diet (protein restriction (low protein diet) and amino acids supplements). (2) report on cognitive skills, behaviour, and health related quality of life effect).

    CN: measure of the blood unconjugated bilirubin and serum total bilirubin levels and quality of life indicators: (1) adjustment of duration of phototherapy, (2) report on cognitive skills, behaviour, and (3) health related quality of life effect.



Original Secondary Outcome:

  • long-term safety profile of HepaStem for both indications [ Time Frame: up to 12 months post infusion ]
    Physical examination, Vital signs,Clinical laboratory tests of liver enzymes, renal function, coagulation factors, hematology,Level of anti-HLA and other auto-immune markers, Adverse events and serious adverse events related to infusion and concomitant treatments
  • preliminary efficacy of HepaStem for both indications (CN and UCD) and for different weight cohorts [ Time Frame: 0-6 months, 6-12 months ]


Information By: Promethera Biosciences

Dates:
Date Received: January 9, 2013
Date Started: March 2012
Date Completion:
Last Updated: May 16, 2015
Last Verified: January 2014