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    Summary
    EudraCT Number:2013-001045-14
    Sponsor's Protocol Code Number:SAF001
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-12-31
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2013-001045-14
    A.3Full title of the trial
    SAF 001: A long-term safety follow-up study of patients having received infusions of HepaStem.
    SAF001: Studio clinico di monitoraggio della sicurezza a lungo termine nei pazienti che hanno ricevuto trattamenti con HepaStem.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study for evaluating long-term safety of patients having received infusions of HepaStem.
    Studio clinico di monitoraggio della sicurezza a lungo termine nei pazienti che hanno ricevuto trattamenti con HepaStem.
    A.3.2Name or abbreviated title of the trial where available
    SAF001
    A.4.1Sponsor's protocol code numberSAF001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPromethera Biosciences
    B.1.3.4CountryBelgium
    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.4.2CountryBelgium
    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 pointVinciane Wouters
    B.5.3 Address:
    B.5.3.1Street AddressRue Granbonpré, 11
    B.5.3.2Town/ cityMont-Saint-Guibert
    B.5.3.3Post code1435
    B.5.3.4CountryBelgium
    B.5.4Telephone number3210394311
    B.5.5Fax number3210394301
    B.5.6E-mailvinciane.wouters@promethera.com
    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.3.9.1CAS number NA
    D.3.9.2Current sponsor codeHHALPC
    D.3.9.3Other descriptive nameNA
    D.3.10 Strength
    D.3.10.1Concentration unit million organisms/ml million organisms/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Yes
    D.3.11.3.5.1CAT classification and reference numberSomatic Cell Therapy Product
    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
    Crigler-Najjar syndrome is associated with a complete or partial hepatic deficit of bilirubin glucuronosyltransferase activity and is apparent during the neonatal period by intense jaundice. The UCD are inborn errors of metabolism that affect the transfer of nitrogen into urea. There
    are 8 disorders: N-Acetylglutamate synthase deficiency, Carbamoyl phosphate synthetase I deficiency, Ornithine transcarbamylase deficiency, citrullinemia I&II, argininosuccinic aciduria, HHH syndrome & argininemia
    E.1.1.1Medical condition in easily understood language
    Liver metabolic diseases: Crigler-Najjar disease and Urea Cycle disorders
    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 16.1
    E.1.2Level LLT
    E.1.2Classification code 10021601
    E.1.2Term Inborn error of metabolism NOS
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the SAF001 long term safety follow-up study in patients having received infusions of HepaStem in a former interventional study (referred as Study A) is to document the safety of HepaStem therapy during a period up to a maximum of 48 months (4 years (Y)) in the SAF001 study (SAF001 Y1+Y2+Y3+Y4).
    This long term safety follow-up study will start when the patient is ending the period of active surveillance post infusion of Hepastem in any former interventional study (Study A) conducted by PB. The safety will be assessed in terms of clinical status (physical examination, vital signs), laboratory, morphology of the liver and kidney, detection of circulating anti-HLA antibodies specific for donor cells or other immune markers related to liver diseases as well as Serious Adverse Events (SAEs) and AEs of Special Interest (AESI) related to HepaStem therapy.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the SAF001 long term safety follow-up study in patients having received infusions of HepaStem in a former interventional study (Study A) are:
    - to document the long term disease evolution after having received Hepastem up to a maximum of 24 months in the SAF study (SAF001 Y1+Y2)
    - to document the long term disease evolution after having received Hepastem up to a maximum of 36 months post infusion (Study A + SAF001 Y1+Y2)
    - to document the long term disease evolution after having received Hepastem up to a maximum of 60 months post infusion (Study A + SAF001 Y1+Y2+Y3+Y4).
    - to document the general safety during a period up to a maximum 60 months post infusion (Study A + SAF001 Y1+Y2+Y3+Y4).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. All patients having received infusions of HepaStem during a former interventional clinical study conducted by PB and who have terminated their participation in that study.
    2. For subjects under 18 years of age (16 year in UK) and for adult subjects legally incapable: the subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent before enrolment or
    For an adult subject legally capable: the subject has provided a written informed consent before enrolment.
    E.4Principal exclusion criteria
    1. Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is to characterise 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.
    E.5.1.1Timepoint(s) of evaluation of this end point
    A 2-year assessment of both safety and disease evolution after having received Hepastem will be done by scheduling visits every 6 months (+/- 1 month) for 2 years (years 1 and 2). An additional 2-year assessment of both safety and disease evolution after having received Hepastem will be done by scheduling a yearly (+/- 1 month) visit and a mid-year (phone) interview during years 3 and 4 (+/- 1 month).
    E.5.2Secondary end point(s)
    The secondary endpoints are to characterize 1. the disease evolution after having received HepaStem therapy and 2. to report on general safety. This assessment will be 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 (including but not limited to tacrolimus immunosuppression therapy)
    Indication I: CN
    - Frequency and severity of metabolic decompensation reported as AESI
    - Metabolic parameters
    o Serum total bilirubin levels and serum unconjugated bilirubin levels
    - Report on supportive treatment and any adjustment of:
    o Phototherapy
    o Medication (eg phenobarbital treatment)

    Indications II: UCD
    - Frequency and severity of metabolic decompensation reported as AESI
    - Metabolic parameters (please refer to section 8, table 4)
    o Ammonium (NH3) values
    o Amino acids in plasma: Alanine, Arginine, Citrulline, Glutamine
    o Urine: Orotic acid for OTC deficiency patients only; argininosuccinate acid for ASL deficiency patients only
    - Report on supportive treatment and any adjustment of:
    o diet (natural protein intake, total protein intake, amino acid supplements)
    o Medication (eg nitrogen scavengers)
    E.5.2.1Timepoint(s) of evaluation of this end point
    A 2-year assessment of both safety and disease evolution after having received Hepastem will be done by scheduling visits every 6 months (+/- 1 month) for 2 years (years 1 and 2). An additional 2-year assessment of both safety and disease evolution after having received Hepastem will be done by scheduling a yearly (+/- 1 month) visit and a mid-year (phone) interview during years 3 and 4 (+/- 1 month).
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    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
    Last patient, last visit.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months48
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months48
    E.8.9.2In all countries concerned by the trial days0
    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: 21
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 13
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    F.1.3Elderly (>=65 years) No
    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 2013-12-31. 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.3.3.6.1Details 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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 21
    F.4.2.2In the whole clinical trial 21
    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)
    This is a long term safety surveillance of the patients following infusion with HepaStem. The surveillance will mimic as much as possible the standard follow-up of the respective diseases (standard of care). The surveillance in the remit of this trial, will end when the patient is undergoing an organ transplant or takes part in another research study.
    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 Decision2014-02-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-02-12
    P. End of Trial
    P.End of Trial StatusCompleted
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