A Phase 2 interventional study of HepaStem in Urea Cycle Disorders, sponsored by Cellaion SA. Completed at 6 sites in 4 countries. Open to participants aged Up to 12 Years. Per ClinicalTrials.gov, last updated 2020-10-19.
Sponsored by Cellaion SA · Phase 2, Interventional, and Treatment
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
66 studies on the registry are indexed under Urea Cycle Disorders, Inborn; 14 are open to participants now.
This study's enrollment of 5 is below the median of 17 across 39 interventional studies indexed under Urea Cycle Disorders, Inborn.
Browse Urea Cycle Disorders, Inborn studies →Cellaion SA is the lead sponsor of 7 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria:
Main Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Target total dose 50x10E6 cells/kg
Biological: 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
Efficacy as determined by de novo ureagenesis (C13 tracer method)
Time frame: at 6m post-first infusion day
Efficacy as determined by de novo ureagenesis (C13 tracer method)
Time frame: at 3, 9 and 12 months post-first infusion day
Efficacy as determined by Ammonia (NH3) values
Time frame: up to 12 months post-first infusion day
Efficacy as determined by amino acids in plasma
Time frame: up to 12 months post-first infusion day
Efficacy as determined by report of metabolic decompensations
Time frame: 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
Time frame: up to 12 months post-first infusion day
Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators
Time frame: up to 12 months post-first infusion day
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.
Time frame: up to 12 months post-first infusion day
Ran from Oct 2014 to primary completion in Mar 2017, 29 months.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2020. You cannot join it, but the record below documents what was studied.
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Urea Cycle Disorders, Inborn →
Cellaion SA