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    EudraCT Number:2017-001731-39
    Sponsor's Protocol Code Number:STRIM002-(205813)
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-07-02
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2017-001731-39
    A.3Full title of the trial
    Methodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy
    Studio metodologico per valutare l’utilità dell’analisi dei siti di inserzione retrovirale in campioni ottenuti da soggetti sottoposti a terapia genica con Strimvelis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Retroviral insertion site methodology study
    Studio metodologico sui siti di inserzione retrovirale
    A.3.2Name or abbreviated title of the trial where available
    Retroviral insertion site methodology study
    Studio metodologico sui siti di inserzione retrovirale
    A.4.1Sponsor's protocol code numberSTRIM002-(205813)
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOrchard Therapeutics (Europe) Ltd
    B.1.3.4CountryUnited Kingdom
    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 supportORCHARD THERAPEUTICS LTD
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationORCHARD THERAPEUTICS LTD
    B.5.2Functional name of contact pointClinical
    B.5.3 Address:
    B.5.3.1Street Address108 Cannon street
    B.5.3.2Town/ cityLondra
    B.5.3.3Post codeEC4N 6EU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+4402033846700
    B.5.5Fax number+4402037270797
    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 Yes
    D. name Strimvelis
    D. of the Marketing Authorisation holderOrchard Therapeutics (Netherlands) BV
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/05/313
    D.3 Description of the IMP
    D.3.1Product nameStrimvelis
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor code.
    D.3.9.4EV Substance CodeSUB176697
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    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 Yes
    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
    Adenosine deaminase (ADA) deficiency severe combined immunodeficiency
    Adenosina deaminasi grave combinata a immunodeficienza
    E.1.1.1Medical condition in easily understood language
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10066367
    E.1.2Term Adenosine deaminase deficiency
    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
    To assess the accuracy and precision of SLiM-PCR methodology for RIS analysis using control insertion site DNA spiked into whole blood samples taken from patients treated with Strimvelis
    Stabilire l’accuratezza e la precisione della metodica SLiM-PCR per l’analisi RIS utilizzando siti di inserzione del DNA di controllo aggiunti a campioni di sangue intero provenienti da pazienti trattati con Strimvelis
    E.2.2Secondary objectives of the trial
    Abundance & Diversity of insertion sites in patients treated with Strimvelis
    Abbondanza e diversità dei siti di inserzione nei pazienti trattati con Strimvelis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, paediatric or adult, patients with ADA-SCID, who have been previously treated with Strimvelis or GSK2696273
    Informed Consent
    2. Capable of giving signed informed consent as described in Appendix 2 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Or signed informed consent provided by the participant’s parent or legal guardian.
    1. Soggetti di sesso maschile o femminile, in età pediatrica o adulta, affetti da ADA-SCID, precedentemente trattati con Strimvelis/GSK2696273.
    Consenso informato
    2. Soggetti in grado di fornire il proprio consenso informato in linea con quanto descritto nell’Appendice 2 del protocollo (soggetti adulti), che prevede di seguire quanto previsto/escluso nel modulo di consenso informato (CI) e nel protocollo in oggetto. In caso di soggetti pediatrici, il consenso deve invece essere firmato dai genitori/tutore legale.
    E.4Principal exclusion criteria
    1. Presence of concomitant condition(s) that in the Investigator’s opinion makes participation in the study unsuitable or may prevent compliance with the protocol requirements.
    2. Unlikely to comply with the requirements of the protocol (i.e. attendance for blood sampling on an approximately annual basis)
    3. Transportation of viable samples to the EU central laboratory from the participant’s home country is not possible
    1. Presenza di una o più condizioni concomitanti che, a giudizio dello sperimentatore, li rendano inadatti alla partecipazione allo studio o che possano rendere il soggetto non compliante ai requisiti del protocollo.
    2. Scarse probabilità di soddisfare i requisiti del protocollo (ad esempio il sottoporsi al prelievo di sangue con cadenza indicativamente annuale.
    3. Impossibilità di trasportare i campioni del soggetto partecipante allo studio dal Suo paese di residenza al laboratorio centralizzato situato in UE.
    E.5 End points
    E.5.1Primary end point(s)
    For subject samples spiked with known amounts of control insertion sites mean abundance and %CV will be calculated:
    •between subjects at every time point
    •within subjects over time points
    •between the same sample within a time point within a subject.
    Per i campioni modificati con quantità note di siti di inserzione di controllo si procederà al calcolo dell’abbondanza media e del % CV (Coefficiente di variazione):
    • inter-soggetto a ciascun timepoint
    • intra-soggetto da un timepoint all’altro
    • all’interno dello stesso campione in riferimento a un timepoint per singolo soggetto.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Samples will be taken approximately annually for 5 years. The study will include newly treated subjects for whom these samples will be taken approximately annually from 1-5 years relative to the treatment date
    I campioni saranno raccolti approssimativamente annualmente per 5 anni. Lo studio includerà inoltre soggetti appena trattati e per questi i campioni saranno raccolti all'incirca ogni anno da 1 (dopo 12 mesi) a 5 anni dalla data di trattamento
    E.5.2Secondary end point(s)
    Clone abundance at each timepoint for each subject will be calculated and displayed.
    •Clones that demonstrate abundance >5% will be listed
    •Descriptive statistics (mean, % CV) on clones present >5% will be
    •Shannon Diversity Index
    •Descriptive statistics (% CV) will be captured
    L’abbondanza dei cloni sarà calcolata e descritta graficamente a ciascun timepoint per ogni soggetto.
    •Saranno elencati i cloni che evidenziano un’abbondanza >5%
    •Saranno prodotte statistiche descrittive (media, % CV) per i cloni con una presenza >5%
    •Indice di diversità di Shannon
    •Elaborazione di statistiche descrittive (% CV)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Samples will be taken approximately annually for 5 years. The study will include newly treated subjects for whom these samples will be taken approximately annually from 1-5 years relative to the treatment date
    I campioni saranno raccolti approssimativamente annualmente per 5 anni. Lo studio includerà inoltre soggetti appena trattati e per questi i campioni saranno raccolti all'incirca ogni anno da 1 (dopo 12 mesi) a 5 anni dalla data di trattamento
    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 No
    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 Yes
    E.6.13.1Other scope of the trial description
    Metodology study
    Studio di metodologia
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E. trial design description
    Studio di Metodologia
    Metodology study
    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.8.2.4Number of treatment arms in the trial1
    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 EEA4
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months0
    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.1.1.1In 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. of subjects for this age range: 5
    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: 2
    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 No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 11
    F.4.2.2In the whole clinical trial 15
    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)
    NA. No treatment will be administred as specified in the protocol.
    NA. Nessun trattamento verrà somministrato come specificato nel protocollo.
    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 Decision2017-10-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-10-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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