An observational study in Urea Cycle Disorders and Crigler Najjar Syndrome, sponsored by Cellaion SA. Completed at 1 site in Belgium. Open to participants aged Up to 23 Years. Per ClinicalTrials.gov, last updated 2019-05-28.
Sponsored by Cellaion SA · Observational
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
The primary objective of the SAF001 study is the long-term safety surveillance of the patients post infusion with HepaStem. Furthermore, the evolution of both the metabolic condition and the quality of life are followed. As much as possible, the surveillance will mimic the standard follow-up of the respective diseases (standard of care). The surveillance will end when the patient is undergoing an organ transplant or takes part in another research study. This surveillance will last up to a maximum of 48 months.
66 studies on the registry are indexed under Urea Cycle Disorders, Inborn; 14 are open to participants now.
This study's enrollment of 9 is below the median of 46 across 23 observational 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.
The SAF001 study will include all patients having received infusions of HepaStem in any former interventional study conducted by Promethera Biosciences.
Main Inclusion Criteria:
Exclusion Criteria:
Characterisation of the long term safety profile of HepaStem therapy.
Assessment of safety will be achieved by evaluating the following parameters * Physical examination * Vital signs * Laboratory tests * Liver tumor markers * Autoimmune markers related to liver pathology * Anti-HLA antibodies specific for donor cell haplotypes * Morphology of liver, bile ducts, and portal system by ultrasound * Morphology of the kidneys by ultrasound * Non-serious or serious Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs) related to HepaStem therapy.
Time frame: 4 years
To characterize the disease evolution after having received HepaStem therapy and to report on general safety.
This assessment is based on the evaluation of: * Report on cognitive skills, behaviour, and health-related quality of life indicators * Non-serious or serious Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs) related to concomitant medications or other causes Indication I: Crigler-Najjar syndrome * Frequency and severity of metabolic decompensation * Metabolic parameters (serum total bilirubin) * Report on supportive treatment and any adjustment of phototherapy and medication (eg phenobarbital treatment) Indication II: Urea cycle disorders * Frequency and severity of metabolic decompensation * Metabolic parameters (NH3 values, amino acids in plasma) * Report on supportive treatment and any adjustment of: * diet (natural protein intake, total protein intake, amino acid supplements) * Medication (eg nitrogen scavengers)
Time frame: 4 years
This study is completed, as verified in May 2019. 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