A Phase 1/2 interventional study of HepaStem in Urea Cycle Disorders and Crigler Najjar Syndrome, sponsored by Cellaion SA. Completed at 11 sites in 5 countries. Open to participants aged Up to 17 Years. Per ClinicalTrials.gov, last updated 2020-10-19.
Sponsored by Cellaion SA · Phase 1/2, Interventional, and Treatment
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.
66 studies on the registry are indexed under Urea Cycle Disorders, Inborn; 14 are open to participants now.
This study's enrollment of 20 is above 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.
GENERAL:
MAIN INCLUSION CRITERIA
Crigler-Najjar Syndrome specific:
Diagnosis must be confirmed by genetic mutation analysis if not available.
Urea Cycle Disorders specific:
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
12.5x106cells/kg
Biological: HepaStem
50x106cells/kg
Biological: HepaStem
200x106cells/kg
Biological: HepaStem
Also known as: HHALPC,Heterologous Human Adult Liver derived Progenitor Cells
Safety of HepaStem in paediatric patients suffering from CN or UCD
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.
Time frame: 6 months
Long-term safety profile of HepaStem in both indications
Assessment of reactogenicity and safety of the treatment during 6 to 12 months post infusion (long-term safety) is evaluated.
Time frame: From 6 to 12 months post-administration
Preliminary efficacy of HepaStem in both indications (CN and UCD) and for different weight cohorts
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.
Time frame: 0-6 months, 6-12 months
To characterize the engraftment of HepaStem
By liver biopsy, enzymatic activity (quantitative) on the biopsies or, donor sequences by RT PCR or in situ hybridisation (FISH) or immunohistochemistry.
Time frame: at 6 month, and optional at 12 month.
The record gives a start of Mar 2012, a primary completion of Oct 2014 and a completion of Apr 2015. It does not say whether these dates are actual or estimated.
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.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Urea Cycle Disorders, Inborn →
Cellaion SA