Urea Cycle DisordersNCT02051049
Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
An observational study of Urea Cycle Disorders and Crigler Najjar Syndrome, run by Cellaion SA at 1 site in Belgium.
Trial summary
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem. Read more about this study
- Who is it for?
- Ages Up to 23 years
- Conditions the study is for
- Urea Cycle Disorders, Crigler Najjar Syndrome
- How many people
- 9 participants
- Where
- 1 siteBelgium
- Study dates
- Mar 2013to Feb 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 23 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The SAF001 study will include all patients having received infusions of HepaStem in any former interventional study conducted by Promethera Biosciences.
Who may be able to join
None listed.
Who cannot join
- Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Long-term Safety Follow-up Study of Patients Having Received Infusions of HepaStem
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
Read the detailed description
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.
- Conditions
- Urea Cycle Disorders, Crigler Najjar Syndrome
- Keywords
- CN, UCD
- Medical subject headings
- Urea Cycle Disorders, Inborn, Crigler-Najjar Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Inborn errors of liver metabolism | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 4 years
Secondary outcomes (1)
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)
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2014 (estimated)
- Study startMar 2013
- Primary completionApr 2018
- Study completionFeb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellaion SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Françoise Smets, MD, Principal investigator, Cliniques universitaires Saint-Luc (Belgium)Dries Dobbelaere, MD/Prof, Principal investigator, CHRU de Lille - Hopital Jeanne de Flandre (France)Isabel Gonçalves, MD/Prof, Principal investigator, Hospital Pediátrico de Coimbra (Portugal)Stephanie Grunewald, MD, Principal investigator, Great Ormond Street Children HospitalGiuliano Torre, MD, Principal investigator, IRCCS OSPEDALE PEDIATRICO DEL BAMBINO GESÃ (Roma)Hanna Mandel, MD, Principal investigator, Rambam Health Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02051049
- Sponsor's protocol ID
- SAF001
- Status last verified
- May 2019
- First submitted
- 29 Jan 2014
- Last updated
- 28 May 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Safety Follow-up Study of Patients Having Received Infusions of HepaStem
- ClinicalTrials.gov ID
- NCT02051049
- Acronym / sponsor ID
- SAF001
- Conditions
- Urea Cycle Disorders, Crigler Najjar Syndrome
- Keywords
- CN, UCD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Cellaion SAIndustry (pharma, biotech, devices)
- Study start
- Mar 2013
- Primary completion
- Apr 2018 (actual)
- Study completion
- Feb 2019 (actual)
- First posted
- 31 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Inborn errors of liver metabolism | – | – |
Who may qualify
- Ages
- Up to 23 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The SAF001 study will include all patients having received infusions of HepaStem in any former interventional study conducted by Promethera Biosciences.
Key inclusion criteria (0 listed)
Key exclusion criteria (1 listed)
- Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Characterisation of the long term safety profile of HepaStem therapy.
Time frame: 4 years
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.
1 secondary outcome
To characterize the disease evolution after having received HepaStem therapy and to report on general safety.
Time frame: 4 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Françoise Smets, MD principal investigator
Cliniques universitaires Saint-Luc (Belgium)
Dries Dobbelaere, MD/Prof principal investigator
CHRU de Lille - Hopital Jeanne de Flandre (France)
Isabel Gonçalves, MD/Prof principal investigator
Hospital Pediátrico de Coimbra (Portugal)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Urea Cycle Disorders and Crigler Najjar Syndrome, run by Cellaion SA at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterisation of the long term safety profile of HepaStem therapy. (time frame: 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2019.