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

Phase 1

Completed

• Conditions: Urea Cycle Disorders; Crigler Najjar Syndrome
• Interventions: Biological: HepaStem

• Registration Number: NCT01765283
• Lead Sponsor: Cellaion SA

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 Description

Not available

Study Timeline

• Study Start: 2012-03
• Study First Submitted: 2013-01-09
• Study First Submitted QC: 2013-01-09
• Study First Posted: 2013-01-10
• Primary Completion: 2014-10
• Study Completion: 2015-04
• Status Verified: 2020-10
• Last Update Submitted: 2020-10-13
• Last Update Posted: 2020-10-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Hepastem Intermediate dose	HepaStem	50x106cells/kg
Hepastem High dose	HepaStem	200x106cells/kg
Hepastem Low dose	HepaStem	12.5x106cells/kg

Primary Outcome Measures

Name	Time	Method
Safety of HepaStem in paediatric patients suffering from CN or UCD	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.

Secondary Outcome Measures

Name	Time	Method
Long-term safety profile of HepaStem in both indications	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	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.

Trial Locations

Locations (11)

Saint Luc University Hospital; 🇧🇪 Brussels, Belgium

Universitair Ziekenhuis (UZ) Antwerpen; 🇧🇪 Edegem, Belgium

CHU Bicêtre; 🇫🇷 Le Kremlin Bicêtre Cedex, France

Hôpital Jeanne de Flandre, CHRU Lille; 🇫🇷 Lille Cedex, France

Hôpital des Enfants, CHU de Toulouse; 🇫🇷 Toulouse cedex 9, France

Rambam Medical Center, Meyer Children's Hospital; 🇮🇱 Haifa, Israel

Hadassah Ein-Kerem Medical Center; 🇮🇱 Jerusalem, Israel

Great Ormond Street Hospital London; 🇬🇧 London, United Kingdom

Birmingham Children's Hospital; 🇬🇧 Birmingham, United Kingdom

Ospedale Pediatrico Bambino Gesu di Roma; 🇮🇹 Roma, Italy

Schneider Children's Medical Center of israel; 🇮🇱 Petach Tikva, Israel