Urea Cycle DisordersPhase 2NCT02489292
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
A Phase 2 study testing HepaStem in people with Urea Cycle Disorders, run by Cellaion SA at 6 sites in 4 countries.
Trial summary
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders. Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Urea Cycle Disorders
- What is being tested?
- HepaStem
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 6 sitesin 4 countries
- Study dates
- Oct 2014to Mar 2017
- Phase
- Phase 2
- 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 12 years
- All sexes
- No healthy volunteers
- Child
Main Inclusion Criteria:
- Paediatric patients < 12 years prior to infusion
- Patient presents with UCD
- Patient shows patency of the portal vein and branches, with normal flow velocity as confirmed by Doppler US and accessibility of the portal vein and /or affluants.
Main Exclusion Criteria:
- Patient has mild disease severity, easily controlled under standard of care therapy, with no recurrent metabolic crises.
- Patient is registered on a liver transplant waiting list or is scheduled for living donor liver transplantation before the end of the study.
- Patient presents acute liver failure.
- Patient presents clinical or radiological evidence of liver cirrhosis.
- Patient presents or has a history of hepatic or extrahepatic malignancy.
- Patient has a known clinically significant cardiac malformation.
- Patient has a personal history of venous thrombosis, or has a clinically significant abnormal value for protein S, protein C, anti-thrombin III, and /or activated Protein C Resistance (aPCR) at screening. In case of known family history, a complete coagulation work-up should be performed. In all above described cases, results need to be discussed with PB before enrolling the patient in the study.
- Patient had or has a renal insufficiency treated by dialysis.
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
Spain3 sites
Hospital Materno Infatil de Badajoz
Badajoz, 06010
Hospital Universitari Vall d'Hebron de Barcelona
Barcelona, 08035
Hospital Materno Infantil de Málaga
Málaga, 29011
About this study
Official title: Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
- Conditions
- Urea Cycle Disorders
- Being tested
- HepaStem
- Keywords
- Urea Cycle Disorder, UCD, cell therapy, stem cell
- Medical subject headings
- Urea Cycle Disorders, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HepaStemBiological
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
| Study group | Role | What they receive |
|---|---|---|
| HepaStem | Experimental | Target total dose 50x10E6 cells/kg Receives: Biological: HepaStem |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy as determined by de novo ureagenesis (C13 tracer method)
Measured: at 6m post-first infusion day
Secondary outcomes (7)
Efficacy as determined by de novo ureagenesis (C13 tracer method)
Measured: at 3, 9 and 12 months post-first infusion day
Efficacy as determined by Ammonia (NH3) values
Measured: up to 12 months post-first infusion day
Efficacy as determined by amino acids in plasma
Measured: up to 12 months post-first infusion day
Efficacy as determined by report of metabolic decompensations
Measured: 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
Measured: up to 12 months post-first infusion day
Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators
Measured: 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.
Measured: up to 12 months post-first infusion day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2015 (estimated)
- Study startOct 2014
- Primary completionMar 2017
- Study completionMar 2017
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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 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
- NCT02489292
- Sponsor's protocol ID
- HEP002
- Status last verified
- Oct 2020
- First submitted
- 6 Nov 2014
- Last updated
- 19 Oct 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients
- ClinicalTrials.gov ID
- NCT02489292
- Acronym / sponsor ID
- HEP002
- Conditions
- Urea Cycle Disorders
- Keywords
- Urea Cycle Disorder, UCD, cell therapy, stem cell
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Cellaion SAIndustry (pharma, biotech, devices)
- Study start
- Oct 2014 (actual)
- Primary completion
- Mar 2017 (actual)
- Study completion
- Mar 2017 (actual)
- First posted
- 3 Jul 2015 (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 aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HepaStem | Experimental | Biological: HepaStemTarget total dose 50x10E6 cells/kg |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HepaStemHepaStem 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 | Biological | HepaStem |
Primary outcome
Efficacy as determined by de novo ureagenesis (C13 tracer method)
Time frame: at 6m post-first infusion day
7 secondary outcomes
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)
Time frame: up to 12 months post-first infusion day
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 19 Oct 2020.
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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.
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HepaStem in people with Urea Cycle Disorders, run by Cellaion SA at 6 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 19 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Spain, Belgium, France, 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Efficacy as determined by de novo ureagenesis (C13 tracer method) (time frame: at 6m post-first infusion day). 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 19 Oct 2020.