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The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
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    The EU Clinical Trials Register currently displays   42758   clinical trials with a EudraCT protocol, of which   7042   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:2010-023111-34
    Sponsor's Protocol Code Number:SG1/001/10
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-03-08
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2010-023111-34
    A.3Full title of the trial
    A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.
    A.4.1Sponsor's protocol code numberSG1/001/10
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOxford BioMedica (UK) 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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 numberEMEA/OD/127/09
    D.3 Description of the IMP
    D.3.1Product nameStarGen
    D.3.2Product code Lentiviral vector containing ABCA4 gene
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntraocular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNStarGen
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number180000 to 1800000
    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) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    D. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.
    MedDRA Classification
    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 safety and tolerability of ascending doses of StarGen in adult patients with Stargardt Macular Degeneration.
    E.2.2Secondary objectives of the trial
    To determine a delay in retinal degeneration following subretinal injection of StarGen, through changes in function relative to he untreated contralateral eye utilising retinal analytical techniques.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Signed and dated written informed consent obtained from the patient in accordance with the local regulations.

    2.Diagnosis of moderate to severe SMD, with at least one pathogenic mutation in the ABCA4 gene on each chromosome, previously confirmed by direct sequencing and co-segregation analysis within the patient’s family.

    3.Women of childbearing potential must have a negative pregnancy test at screening and at baseline, and agreement to using an effective form of contraception such as the contraceptive pill or intra uterine device, or be surgically sterile or postmenopausal, with the last menstrual period being over two years ago. Partners of study patients must agree to use barrier contraception for at least three months after StarGen administration.

    4.Males must agree with their partner to use two forms of contraception, including one barrier method for at least three months following StarGen administration if their partner is of child-bearing potential, or must be surgically sterile.

    5.Affiliated with the French social security healthcare system (French patients only).
    E.4Principal exclusion criteria
    1.Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints: glaucoma or other primary optic neuropathy that has resulted in significant visual field loss, corneal or significant lens opacities, active uveitis, retinopathy and maculopathy (other than that from Stargardt disease) that in the opinion of the investigator is causing significant visual loss, myopia greater than eight diopters spherical equivalent.

    2.Cataract surgery with intraocular lens implantation within 6 months of enrolment.

    3.Aphakia or prior vitrectomy in the study eye.

    4.Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function. For instance malignancies whose treatment could affect central nervous system function, diabetes, juvenile rheumatoid arthritis or sickle cell disease.

    5.Any intraocular surgery or laser in either eye within 6 months of Day 0.

    6.Any contraindication to pupil dilation in either eye.

    7.Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study (e.g., fluorescein, dilation drops), or medications planned for use in the peri-operative period, particularly topical, injected or systemic cortico-steroids.

    8.Any injectable intravitreal treatment to the treated eye or intravitreal device in the treated eye within 6 months prior to screening.

    9.Any periocular injections of corticosteroids to the treated eye within 4 months prior to screening.

    10.Clinically significant laboratory test abnormalities, including full blood count, chemistry panel, liver function tests, electrocardiogram and chest X rays, that in the opinion of the principal investigator, would make the patient unsuitable for participation in the study.

    11.Significant intercurrent illness or infection during the 28 days prior to enrolment.

    12.Pre-menopausal or non-surgically sterile women who are unwilling to use an effective form of contraception such as the contraceptive pill or intrauterine device.

    13.Men or women who do not agree to use contraception as specified in the inclusion criteria.

    14.Alcohol or other substance abuse.

    15.Contraindications to use of anaesthesia (local or general, as appropriate).

    16.Concurrent anti-retroviral therapy that would inactivate the investigational agent.

    17.History of any investigational agent within 28 days prior to StarGen administration.

    18.Participation in a prior ocular gene transfer therapy study.

    19.Enrolment in any other clinical treatment study, for any condition, including those relating to SMD, throughout the duration of the StarGen study.

    20.Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy within the four weeks prior to surgery.

    21.Past medical history of HIV or Hepatitis A, B or C.

    22.Inability to comply with the demands of the study.
    E.5 End points
    E.5.1Primary end point(s)
    The incidence and severity of treatment emergent adverse events.
    Clinically important changes in the following safety assessments:
    Best corrected visual acuity (BCVA)
    Slit-lamp biomicroscopy
    Indirect ophthalmoscopy
    Fundus photography
    Measurement of intraocular pressure
    Visual field - microperimetry
    Ocular Coherence Tomography (OCT)
    Laboratory parameters
    Vital signs
    Concomitant medications
    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 Yes
    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. 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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 No
    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 Yes
    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 subject in the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 28
    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)
    The patients will all be followed up for 48 weeks. After this period they will enter an open label safety study and long-term follow up visits including ophthalmological examinations and recording of adverse events will continue out to 5 years.
    In addition, the investigator will contact the patient by telephone for a subsequent 10 years at a minimum interval of once a year to monitor possible delayed adverse events.
    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 Decision2011-07-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-05-03
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-08-16
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