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    Summary
    EudraCT Number:2017-004783-35
    Sponsor's Protocol Code Number:OPH2005
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2018-01-26
    Trial 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 ConcernedUK - MHRA
    A.2EudraCT number2017-004783-35
    A.3Full title of the trial
    A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subjects with Autosomal Recessive Stargardt Disease
    A.4.1Sponsor's protocol code numberOPH2005
    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 SponsorIVERIC bio
    B.1.3.4CountryUnited States
    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 supportIVERIC bio
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIVERIC bio
    B.5.2Functional name of contact pointVP Global Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressOne Penn Plaza, 35th floor, Suite 3520
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10119
    B.5.3.4CountryUnited States
    B.5.6E-mailkenneth.miller@ivericbio.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameZimura
    D.3.2Product code ARC1905
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravitreal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNavacincaptad pegol
    D.3.9.2Current sponsor codeARC1905
    D.3.9.3Other descriptive nameARC1905 20 MG/ML
    D.3.9.4EV Substance CodeSUB30770
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    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) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    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 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 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
    Autosomal Recessive Stargardt Disease
    E.1.1.1Medical condition in easily understood language
    Autosomal Recessive Stargardt Disease
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10062766
    E.1.2Term Stargardt's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objectives of this study are to evaluate the safety and efficacy of Zimura™ intravitreal injection compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Ophthalmic Inclusion Criteria
    The following inclusion criteria apply to the study eye:
    - At least two pathogenic mutations of ABCA4 gene confirmed by a CLIA-certified laboratory.
    - Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive.
    - Presence of at least one identifiable location of at least 250 micrometers contiguous width of ellipsoid zone loss on SD-OCT within the total 9 ETDRS subfields.
    - The total area of thinned layer(s) on OCT not extending beyond the outer ring of the total 9 ETDRS subfields (e.g. not extending beyond fields 5-9).
    - Fundus autofluorescence may show heterogeneous signals (areas of increased and/or decreased autofluorescence) but no area of subfoveal definitely decreased autofluorescence (DDAF).
    - Clear ocular media and adequate pupillary dilatation in both eyes (OU) to allow for all imaging procedures, including good quality stereoscopic fundus photography, fundus autofluorescence, and spectral domain ocular coherence tomography.
    - Intraocular pressure of 21 mmHg or less in the study eye.

    General Inclusion Criteria
    - Subjects of either gender aged between 18 and 50 years, inclusive.
    - Women of childbearing potential is defined as follows:
    • Woman who has had surgical sterilization (hysterectomy, bilateral oophorectomy, or bilateral salpingectomy)
    • Woman ≥ 60 years of age
    • Woman ≥ 40 and < 60 years of age who fulfills at least one of the following
    * A cessation of menses for at least 12 months and a follicle-stimulating hormone (FSH) test confirming non-childbearing potential (refer to laboratory reference ranges for confirmatory levels)
    * A cessation of menses for at least 24 months without FSH levels confirmed
    For subjects who are women of childbearing potential involved in any sexual intercourse that could lead to her pregnancy, the subject has used a protocol approved highly effective contraceptive method during the trial and agrees to continue the same method until at least 90 days following the last dose of test medication. Protocol approved highly effective contraceptive methods are hormonal contraceptives (i.e., combined oral contraceptive, patch, vaginal ring, injectable, or implant), intrauterine devices or intrauterine systems, abstinence as defined by refraining from heterosexual intercourse during the entire period of the study and until at least 90 days following the last dose of study medication, vasectomy, and tubal ligation.
    If subject is a woman of childbearing potential, she must have a negative serum pregnancy test within 14 days prior to the first injection and have no plans to donate ova during the duration of the trial and at least 90 days following the last dose of test medication.
    If subject is a male, the subject should use condom and not donate sperm during the time of study drug exposure and for 90 days following last exposure of study drug.
    - Provide written informed consent.
    - Ability to return for all trial visits.
    E.4Principal exclusion criteria
    Subjects will not be eligible for the trial if subjects cannot attend all of the trial required visits, or if any of the following criteria are present in the study eye or systematically:

    Ophthalmic Exclusion Criteria
    - Macular atrophy secondary to any condition other than STGD1 in either eye (e.g., drug-induced).
    - Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye.
    - Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening.
    - Any ocular condition in the study eye that would progress during the course of the study that could affect central vision, microperimetry testing or otherwise be a confounding factor.
    - Concomitant treatment with any ocular or systemic medication that is known to be toxic to the lens, retina or optic nerve.
    - Presence of intraocular inflammation (≥ trace cell or flare), macular hole, pathologic myopia (spherical equivalent of -8 diopters or more negative, or axial length of 25mm or more), epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia (pseudophakia with or without an intact capsule is not an exclusion criteria).
    - Presence or history of idiopathic or autoimmune-associated uveitis in either eye.
    - Significant media opacities, including cataract, which might interfere with visual acuity, assessment of toxicity, fundus photography or fundus autofluorescence. Subjects should not be entered if there is likelihood that they will require cataract surgery in the study eye during the study.
    - Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region, regardless of indication.
    - Any ocular or periocular infection or ocular surface inflammation in the past 12 weeks.
    - History of any of the following procedures: Posterior vitrectomy, filtering surgery (e.g. trabeculectomy), glaucoma drainage device, corneal transplant or retinal detachment.

    General Exclusion Criteria
    - Any of the following underlying diseases including:
    • Diabetes mellitus (regardless of HbA1c level)
    • HbA1c value of ≥6.5%*:
    • If the HbA1c value is ≥ 6.5% and ≤ 6.9%, and the patient has no signs or symptoms of diabetes mellitus, has a normal creatinine, has no diabetic retinopathy and no glycosuria, then the patient may have an oral glucose tolerance test (OGTT) at the discretion of the investigator. If the 2-hour glucose value on OGTT is <200 mg/dL (<11.1mmol/L), then the patient may be enrolled.
    • History of other disease, metabolic dysfunction, physical examination finding or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that might affect interpretation of the results of the study or render the subject at high risk for treatment complications.
    • History or evidence of severe cardiac disease (eg, New York Heart Association (NYHA) Functional Class III or IV - see Appendix 17.5), history or clinical evidence of unstable angina, acute coronary syndrome, myocardial infarction or revascularization within last 6 months, ventricular tachyarrhythmia requiring ongoing treatment.
    • Subjects with a clinically significant laboratory value. Laboratory tests may be repeated once before randomization.
    • Stroke within 12 months of trial entry.
    • Any major surgical procedure within one month of trial entry or anticipated during the trial which may interrupt trial participation.
    - Previous therapeutic radiation in the region of the study eye.
    - Any treatment with an investigational agent in the past 60 days for any condition.
    - Women who are pregnant or nursing.
    - Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the Zimura™ formulation.
    - History of systemic treatment with any complement inhibitor agent in the past or the likelihood of treatment with any systemic complement inhibitor agent during the study.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint
    • Mean rate of change in the area of ellipsoid zone defect measured by en face SD-OCT over 18 months.

    Safety Endpoints:
    • Adverse events, vital signs, ophthalmic variables [ophthalmic examination, intraocular pressure (IOP), fluorescein angiogram (FA), fundus autofluorescence (FAF), spectral domain-optical coherence tomography (SD-OCT), microperimetry], electrocardiogram (ECG), and laboratory variables.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to Month 18
    E.5.2Secondary end point(s)
    Secondary and Supportive Endpoints
    • Mean rate of change in the horizontal width of undetectable ellipsoid zone measured by a horizontal scan through the foveal center with SD-OCT over 18 months.
    • Mean rate of change in the area of atrophic lesion (definite decrease in autofluorescence, DDAF) over 18 months measured by fundus autofluorescence (FAF).
    • Mean change in photopic and/or mesopic macular sensitivity measured by microperimetry from Baseline to Month 18.
    • Mean rate of change in the thickness of the outer nuclear layer measured by a horizontal scan through the foveal center at the position of maximum width of ellipsoid zone loss at Baseline over 18 months measured by SD-OCT.
    • Mean change in best corrected visual acuity (Early Treatment Diabetic Retinopathy Study [ETDRS] letters) from Baseline to Month 18.
    • Emergence of at least one new atrophic lesion (DDAF) over 18 months measured by FAF at Month 18.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline to Month 18
    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 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.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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3.1Comparator description
    Zimura and sham administered by an unmasked investigator
    E.8.2.4Number of treatment arms in the trial2
    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 EEA19
    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
    Canada
    France
    Germany
    Hungary
    Israel
    Italy
    Spain
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days2
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days27
    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.1.2.1Number of subjects for this age range: 70
    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 No
    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 35
    F.4.2.2In the whole clinical trial 70
    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)
    None
    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 Decision2018-03-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-29
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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