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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   42771   clinical trials with a EudraCT protocol, of which   7044   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    EudraCT Number:2017-003989-27
    Sponsor's Protocol Code Number:PROLONGSTEM
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-10-25
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-003989-27
    A.3Full title of the trial
    PROLONGSTEM: HepaStem Long-Term Safety Registry
    Registry for patients who have been administered HepaStem
    PROLONGSTEM: Registro de seguridad a largo plazo de HepaStem
    Registro para pacientes a los que se administró HepaStem
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    PROLONGSTEM is a long term follow-up study of all the patients who have received at least one infusion of HepaStem
    PROLONGSTEM es un estudio de seguimiento a largo plazo de todos los pacientes que han recibido al menos una inyección de HepaStem
    A.4.1Sponsor's protocol code numberPROLONGSTEM
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/313/2013
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPromethera Biosciences
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPromethera Biosciences
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPromethera Biosciences
    B.5.2Functional name of contact pointClinical Research Department
    B.5.3 Address:
    B.5.3.1Street AddressRue Granbonpré, 11
    B.5.3.2Town/ cityMont-Saint-Guibert
    B.5.3.3Post codeB-1435
    B.5.4Telephone number+32 10 394300
    B.5.5Fax number+32 10 394301
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberOD/101/07-039/07-057-058- 059-060-061-062-063/13
    D.3 Description of the IMP
    D.3.1Product nameHepaStem
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNheterologous human adult liver-derived progenitor cells
    D.3.9.2Current sponsor codeHHALPC
    D.3.9.3Other descriptive nameHHALPC
    D.3.9.4EV Substance CodeSUB122824
    D.3.10 Strength
    D.3.10.1Concentration unit million organisms/ml million organisms/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10 to 50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D. cell therapy medicinal product Yes
    D. therapy medical product No
    D. Engineered Product Yes
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product Yes
    D. classification and reference numberSomatic Cell Therapy Product (EMA/CAT/243155/2011) - Treatment of inborn errors of liver metabolism Tissue engineered product (EMA/CAT/391889/2016) - treatment of fibroinflammatory liver diseases.
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    This study will include 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 including patients suffering from urea cycle disorders (UCD), Crigler-Najjar (CN) and Fibro-inflammatory liver diseases
    Este estudio incluirá a todos los pacientes que hayan recibido al menos una inyección del producto en fase de investigación clínica (PEI), HepaStem HHALPC, durante un estudio clínico de intervención previo realizado por Promethera Biosciences incluyendo pacientes con trastornos del ciclo de la urea (UCD), Crigler-Najjar (CN) y Enfermedades hepáticas fibroinflamatorias
    E.1.1.1Medical condition in easily understood language
    This study is a long term safety follow-up for all the patients that have received at least one dose of HepaStem: patients suffering from UCD, CN and fibro-inflammatory liver diseases
    Este estudio es un seguimiento de seguridad a largo plazo para todos los pacientes que han recibido al menos una dosis de HepaStem: pacientes con UCD, CN y enfermedades hepáticas fibroinflamatorias
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10021601
    E.1.2Term Inborn error of metabolism NOS
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10008954
    E.1.2Term Chronic liver disease and cirrhosis
    E.1.2System Organ Class 100000004871
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10049844
    E.1.2Term Acute liver failure
    E.1.2System Organ Class 100000004871
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10013373
    E.1.2Term Disorders of urea cycle metabolism
    E.1.2System Organ Class 200000003094
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Longitudinal evaluation of the long-term safety post-HepaStem administration.
    Evaluación longitudinal de la seguridad a largo plazo después de la administración de HepaStem.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. All patients having received at least one infusion of HepaStem during a previous interventional clinical study conducted by Promethera Biosciences.
    2. Written Informed Consent.
    For a patient who is a minor (according to the national regulations), and for adult patients legally incapable: the parents or legal representative have provided a signed informed consent before enrolment. If the patient is capable of signing, he/she is required to sign an assent in parallel.
    For adult patient (legally capable): patient has provided a written informed consent before enrolment.
    1. Todos los pacientes que recibieron al menos una inyección de HepaStem durante un estudio clínico de intervención previo realizado por Promethera Biosciences.
    2. Consentimiento informado escrito.
    Para un paciente que es menor de edad (de acuerdo con las reglamentaciones nacionales) y para pacientes adultos legalmente incapaces: los padres o representante legal han proporcionado un consentimiento informado firmado antes de la inscripción. Si el paciente es capaz de firmar, se requiere que firme un consentimiento en paralelo.
    Para pacientes adultos (con capacidad legal): el paciente ha proporcionado un consentimiento informado por escrito antes de la inscripción.
    E.4Principal exclusion criteria
    1. Patients who received an OLT and completed 6-month FU documentation prior to the start of the PROLONGSTEM study.
    2. Patients who have received mature liver cells or stem cells other than HepaStem prior to the start of the PROLONGSTEM study.
    1. Pacientes que recibieron un transplante de hígado y documentación completa de estudio de seguimiento de 6 meses antes del inicio del estudio PROLONGSTEM.
    2. Pacientes que hayan recibido células hepáticas maduras o células madre distintas de HepaStem antes del inicio del estudio PROLONGSTEM.
    E.5 End points
    E.5.1Primary end point(s)
    Occurrence of 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.
    These AESI should be considered as medically important and reported as SAE.
    Ocurrencia de acontecimientos adversos de interés específico (AESI):
    - Evento con resultado fatal (Muerte)
    - Trasplante de hígado y resultado.
    - Desarrollo de tumores malignos o formación de tejido no deseado en diferentes órganos (tumores malignos o no).
    - Enfermedad relacionada con la transmisión de agentes adventicios o la reactivación de patógenos latentes.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patients who previously received the IMP HepaStem in the context of an interventional study with HepaStem administration will be followed-up for long-term safety surveillance up to 5 years in this PROLONGSTEM study.
    Los pacientes que recibieron previamente el PEI HepaStem en el contexto de un estudio de intervención con administración de HepaStem serán objeto de seguimiento para la vigilancia de la seguridad a largo plazo hasta 5 años en este estudio PROLONGSTEM.
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Ultimo paciente Última visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 8
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 12
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2018-10-25. Yes
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women Yes
    F.3.3.4Nursing women Yes
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    Paediatric patients (<18 years old)
    Adult subjects legally incapable
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 22
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Not Applicable:
    This study is a long term follow-up of 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
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-11-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-26
    P. End of Trial
    P.End of Trial StatusOngoing
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