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    Summary
    EudraCT Number:2012-004952-12
    Sponsor's Protocol Code Number:4429-202
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-03-12
    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 ConcernedGermany - BfArM
    A.2EudraCT number2012-004952-12
    A.3Full title of the trial
    A Phase 2b/3 Multicenter, Randomized, Double-Masked, Dose-Ranging Study Comparing the Efficacy and Safety of Emixustat Hydrochloride (ACU-4429) with Placebo for the Treatment of Geographic Atrophy Associated with Dry Age-Related Macular Degeneration
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    SEATTLE clinical trial
    Safety and Efficacy Assessment Treatment TriaLs of Emixustat Hydrochloride.
    A.4.1Sponsor's protocol code number4429-202
    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 SponsorAcucela Inc.
    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 supportAcucela Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcucela Inc.
    B.5.2Functional name of contact pointAcucela Clinical Trials Helpdesk
    B.5.3 Address:
    B.5.3.1Street Address1301 Second Ave, Suite 1900
    B.5.3.2Town/ citySeattle, WA
    B.5.3.3Post code98101-3805
    B.5.3.4CountryUnited States
    B.5.4Telephone number+12068058310
    B.5.5Fax number+12068058301
    B.5.6E-mailclinicaltrials@acucela.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 nameemixustat hydrochloride
    D.3.2Product code ACU-4429
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNemixustat
    D.3.9.1CAS number 1141934-97-5
    D.3.9.2Current sponsor codeACU-4429 HCl
    D.3.9.3Other descriptive nameEMIXUSTAT HYDROCHLORIDE (ACU-4429 HCL)
    D.3.9.4EV Substance CodeSUB96210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number2.5 to 10
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Geographic atrophy associated with dry age-related macular degeneration
    E.1.1.1Medical condition in easily understood language
    dry macular degeneration
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10063947
    E.1.2Term Geographic atrophy
    E.1.2System Organ Class 100000004853
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine if ACU-4429 reduces the rate of progression of geographic atrophy (GA) compared to placebo in subjects with dry age-related macular degeneration (AMD).
    E.2.2Secondary objectives of the trial
    1. To evaluate the safety and tolerability of ACU-4429 compared to placebo when administered orally for 24 months at fixed doses and in a step-up titration arm.
    2. To assess changes in best-corrected visual acuity (BCVA) with ACU-4429 compared to placebo.
    3. To evaluate the effect of ACU-4429 compared to placebo on the development of choroidal neovascularization (CNV) in the study eye.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males or females, age ≥55 years.
    2. Clinical diagnosis of GA associated with AMD in 1 or both eyes as determined by the Investigator and confirmed by the reading center.
    3. The study eye must meet the following criteria as determined by the central reading center’s assessment of fundus autofluorescence (FAF) imaging at screening:
    a. Total area of GA ≥1.25 - 18 square mm (~0.5 - 7 disc areas) in size.
    b. If GA is multifocal, at least 1 locus of GA must be ≥1.25 mm2 (0.5 disc area) in size, with the overall aggregate area of GA, within the vascular arcades, ≤18 mm2 (~7 disc areas).
    c. The entire lesion must be completely visualized on the macula-centered image (Field 2 –Macula Image). GA must be able to be imaged in its entirety and not contiguous with any areas of peripapillary atrophy.
    4. Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA of ≥35 letters (approximately ≥20/200 Snellen) in the study eye.
    5. Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of GA in the study eye as determined by the Investigator.
    6. Able and willing to provide written informed consent before undergoing any study-related procedures.
    7. Able to reliably administer oral medication by self or with available assistance.
    E.4Principal exclusion criteria
    1. History of, active or high risk of developing CNV:
    a. History of CNV associated with AMD in the study eye
    b. Active CNV in the study eye as confirmed by the central reading center
    c. Or a spherical equivalent >8 diopters of myopia in refractive error in either eye. If the subject has previously undergone refractive surgery, including cataract surgery, then the pre-operative error cannot be >8 diopters of myopia.
    Note: CNV associated with AMD in the non-study eye is allowed regardless of treatment status.
    2. Geographic atrophy associated with a condition other than AMD in either eye, or macular GA with contiguous area of peripapillary atrophy in the study eye.
    3. Presence in either eye of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function, including, but not limited to, uveitis, other macular diseases or uncontrolled glaucoma/ocular hypertension (≥25 mm Hg with or without glaucoma
    medication treatment).
    4. History in study eye of macular edema, ext. beam radiat., macular surgery, or transpup. thermotherapy.
    5. History of any intraocular or ocular surface surgery in either eye within 3 months of screen..
    6. Prev. particip. in an investigat. study of ACU-4429.
    7. Known serious allergy to fluorescein sodium for injection in angiography or hypersensitivity to ACU-4429 or any of the excipients in ACU-4429 tablets (ie, silicif. microcrystall. cellulose, pregelatin. starch, coll. silicon dioxide and stearic acid).
    8. Prohibited medications: Use of a strong inhibitor or inducer of cytochrome P450 enzyme CYP3A4 or a strong inducer of or a strong or moderate inhibitor of CYP2D6 (as listed in Appendix B) beginning 4 weeks prior to screening and throughout the duration of the study period.
    9. [please see protocol]
    10. Change in a systemic prescription medication or a medication newly prescribed within 30 days prior to screening and between screening and baseline.
    11. Participation during the study period in any study using an investigational study drug (within 30 days of screening) or interventional device (within 60 days of screening). Age-related Eye Disease Study (AREDS)-formulated vitamins (which have been under investigation) are allowed during the study period.
    12. History of other disease, metabolic dysfunction, chronic immunosuppression, physical examination finding, or clinical laboratory finding that in the opinion of the Investigator gives reasonable suspicion of a disease or condition which contraindicates the use of an investigational drug or might have affected interpretation of the results of the study or rendered the subject at high risk for treatment complications.
    13. Current or history of cancer (except non-metastatic in situ or well-controlled carcinoma, [eg, basal cell carcinoma]) within 1 year of screening.
    14. Any medical condition (eg, malabsorption, inflammatory bowel disease, or hepatic or renal disease) that could alter the absorption, metabolism, or elimination of drugs.
    15. History of myocardial infarction, stroke, unstable ischemic heart disease, uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure within 6 months of screening.
    16. Abnormal electrocardiogram (ECG) results that are considered by the Investigator to be clinically significant at screening.
    17. Female subjects who are pregnant or lactating.
    18. Female subjects of childbearing potential (ie, not postmenopausal for at least 2 years or not surgically sterile)who are not willing to practice a medically accepted method of birth control with their non-surgically sterile male sexual partner from screening through 30 days following the completion of the study. Medically accepted methods of birth control include hormonal contraceptives, nonhormonal intrauterine contraceptive device with spermicide, condom with spermicide, contraceptive sponge with spermicide, diaphragm with spermicide, cervical cap with spermicide.
    19. Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential (as listed above) from screening through 30 days after completion of the study.
    20. Unstable or poorly controlled medical or ophthalmic conditions that in the opinion of the Investigator might interfere with the efficacy or safety evaluation of the study drug, put the subject at any clinical risk, negatively impact subject compliance, or jeopardize the subject’s ability to complete the study.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint

    The primary efficacy endpoint will be the mean
    rate of change from baseline in the total area of
    the GA lesion(s) in the study eye (in mm2 per
    year) as imaged by FAF.

    Safety Endpoints
    Safety endpoints will include:

    1. Frequency of AEs, discontinuations due to AEs, or dose modifications due to AEs; severity and seriousness of AEs.
    2. Change from baseline in laboratory values, vital signs, physical examination findings, ECGs, and ophthalmic assessments.


    E.5.1.1Timepoint(s) of evaluation of this end point
    FAF will be done on Screening, Baseline (D0), Month 6, 12, 18, 24, Early Termination Visit;

    Frequency of AE's will be evaluated continuously at all study visits;
    Lab values will be evaluated at Screening, Baseline (Day 0), Month 6, 12, 24, 25 , only if safety laboratory test result(s) at the Month 24 or Early Termination visit were considered clin. sig.by Inv.;
    Vital Signs are evaluated at Screening, Baseline (D0), Month 6, 12, 18, 24, and Early Termin. Visit;
    Physical Examination is done at Screening, Month 24 and Early Termination Visit;
    ECG is performed at Screening, Month 6, 12, 18, 24 and Early Termination Visit;
    The timepoints of conduction of ophthalmic assessments depends on the kind of examination, see Schedule of Events provided in Appendix A of the protocol;
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints
    Secondary efficacy endpoints will include:
    1. Mean change from baseline in NL-BCVA score
    2. Proportion of subjects who develop CNV in their study eye at any time during the study.
    E.5.2.1Timepoint(s) of evaluation of this end point
    BCVA and evaluation of CNV will be done at Screening, Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 25 and Early Termination Visit;

    Study exit visit: If follow-up is needed after this visit, it should occur as an unscheduled visit at the Investigator’s discretion or telephone contact.

    If the Early Termination visit occurs ≥ 25 days after discontinuation of study drug, a combined Early Termination/Study Exit visit may be performed. If the Early Termination/Study Exit visits are combined, all assessments scheduled at each of the 2 visits (including ERG, if applicable) should be performed.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    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) Yes
    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) Yes
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 concerned7
    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 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
    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
    This is specified in the protocol (section 9.7) as the last visit of the last subject.
    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 months4
    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 months4
    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.1.2.1Number of subjects for this age range: 48
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 432
    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 state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 480
    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 treatment of subjects after their participation in the clinical study has ended will be in accordance with the expected standard of care for their dry AMD disease.
    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 Decision2013-04-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-02-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-04-15
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