Urea Cycle DisorderPROLONGSTEMNCT03343756
HepaStem Long-Term Safety Registry
An observational study of Urea Cycle Disorder and Crigler-Najjar Syndrome, run by Cellaion SA at 29 sites in 5 countries.
Trial summary
All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences Read more about this study
- Who is it for?
- Ages 5 to 75 years
- Conditions the study is for
- Urea Cycle Disorder, Crigler-Najjar Syndrome, Acute on Chronic Liver Failure
- How many people
- 44 participants
- Where
- 29 sitesin 5 countries
- Study dates
- 4 Apr 2018to 23 May 2024
- 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 5 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients who received HepaStem in the interventional studies conducted by Promethera will be proposed to participate in this PROLONGSTEM study.
Who may be able to join
- All patients having received at least one infusion of HepaStem during a previous interventional clinical study conducted by Promethera.
Who cannot join
- Patients who received an OLT and completed 6-month FU documentation prior to the start of the PROLONGSTEM study.
- Patients who have received mature liver cells or stem cells other than HepaStem prior to the start of the PROLONGSTEM study.
Trial locations and contacts
29 sites in 5 countries.
29 sites in 5 countries
France11 sites
Centre Hospitalier Universitaire Amiens Picardie
Amiens, 80080
Centre Hospitalier Universitaire D'Angers
Angers, 49933
University Hospital Of Bordeaux
Bordeaux, 33608
CHU Lille
Lille, 59037
Hospices Civils De Lyon
Lyon, 69317
Assistance Publique Hopitaux De Marseille
Marseille, 13005
Centre Hospitalier Universitaire De Montpellier
Montpellier, 34295
Assistance Publique Hopitaux De Paris
Paris, 75013
Les Hopitaux Universitaires De Strasbourg
Strasbourg, 67000
CHU Toulouse
Toulouse, 31059
Hopital Paul Brousse
Villejuif, 94804
Belgium8 sites
Hopital Erasme
Anderlecht, 1070
Antwerp University Hospital
Antwerp, 2650
Association Hospitaliere De Bruxelles Et De Schaerbeek Centre Hospitalier Universitaire Brugmann
Brussels, 1020
UCL
Brussels, 1200
Universitair Ziekenhuis Gent
Ghent, 9000
UZ Leuven
Leuven, 3000
CHC MontLegia
Liège, 4000
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert
Spain6 sites
Hospital De La Santa Creu I Sant Pau
Barcelona, 08025
Hospital Universitari Vall D'Hebron
Barcelona, 8035
Hospital General Universitario Gregorio Maranon
Madrid, 28009
Hospital Universitario Ramon Y Cajal
Madrid, 28049
Parc Tauli Hospital Universitari
Sabadell, 08208
Hospital Unviersitario Miguel Servet
Zaragoza, 50009
Bulgaria3 sites
University Multiprofile Hospital For Active Treatment Dr. Georgi Stranski EAD
Pleven, 5803
UMHAT Medica Ruse
Rousse, 7013
Military Medical Academy
Sofia, 1606
About this study
Official title: HepaStem Long-Term Safety Registry - Registry for Patients Who Have Been Administered HepaStem
All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences
- Medical subject headings
- Urea Cycle Disorders, Inborn, Crigler-Najjar Syndrome, Acute-On-Chronic Liver Failure
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of Adverse Events of Specific Interest (AESI)
Adverse Events of Specific Interest (AESI):
- Event with fatal outcome (Death)
- Orthotopic Liver Transplantation (OLT) and Outcome.
- Development of Malignancy or unwanted tissue formation in different organs (tumor malignant or not).
- Disease linked to transmission of adventitious agents or reactivation of latent pathogens.
- Any AE which in the opinion of the Investigator has a plausible causal relationship to HepaStem.
Measured: 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start4 Apr 2018
- Primary completion23 May 2024
- Study completion23 May 2024
Why it stopped: Sponsor decision related to product development prioritisation.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle DisorderStudies 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
- Other
- Time perspective
- Other
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellaion SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03343756
- Sponsor's protocol ID
- PROLONGSTEM
- Acronym
- PROLONGSTEM
- Status last verified
- May 2024
- First submitted
- 10 Nov 2017
- Last updated
- 29 May 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HepaStem Long-Term Safety Registry - Registry for Patients Who Have Been Administered HepaStem
- ClinicalTrials.gov ID
- NCT03343756
- Acronym / sponsor ID
- PROLONGSTEM / PROLONGSTEM
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor decision related to product development prioritisation.
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Cellaion SAIndustry (pharma, biotech, devices)
- Study start
- 4 Apr 2018 (actual)
- Primary completion
- 23 May 2024 (actual)
- Study completion
- 23 May 2024 (actual)
- First posted
- 17 Nov 2017 (actual)
- 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.
All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences
Study design
- Observational model
- Other
- Time perspective
- Other
Who may qualify
- Ages
- 5 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who received HepaStem in the interventional studies conducted by Promethera will be proposed to participate in this PROLONGSTEM study.
Key inclusion criteria (1 listed)
- All patients having received at least one infusion of HepaStem during a previous interventional clinical study conducted by Promethera.
Key exclusion criteria (2 listed)
- Patients who received an OLT and completed 6-month FU documentation prior to the start of the PROLONGSTEM study.
- Patients who have received mature liver cells or stem cells other than HepaStem prior to the start of the PROLONGSTEM study.
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
Occurrence of Adverse Events of Specific Interest (AESI)
Time frame: 60 months
Adverse Events of Specific Interest (AESI): * Event with fatal outcome (Death) * Orthotopic Liver Transplantation (OLT) and Outcome. * Development of Malignancy or unwanted tissue formation in different organs (tumor malignant or not). * Disease linked to transmission of adventitious agents or reactivation of latent pathogens. * Any AE which in the opinion of the Investigator has a plausible causal relationship to HepaStem.
Results and safety
The study was stopped early (reason given: Sponsor decision related to product development prioritisation.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2024.
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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.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Urea Cycle Disorder and Crigler-Najjar Syndrome, run by Cellaion SA at 29 sites in 5 countries.
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 1 inclusion and 2 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: stopped early, as of the sponsor's last update on 29 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in France, Belgium, Spain, Bulgaria, Poland. 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 was stopped early (reason given: Sponsor decision related to product development prioritisation.). Results are not available in the source record.
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?
Occurrence of Adverse Events of Specific Interest (AESI) (time frame: 60 months). 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 29 May 2024.