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    Summary
    EudraCT Number:2018-003498-82
    Sponsor's Protocol Code Number:4429-301
    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:2018-12-21
    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 ConcernedGermany - BfArM
    A.2EudraCT number2018-003498-82
    A.3Full title of the trial
    A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of Emixustat for the Treatment of Stargardt Disease
    A.3.2Name or abbreviated title of the trial where available
    The SeaSTAR Study (Safety and efficacy of EmixustAt in STARgardt disease)
    A.4.1Sponsor's protocol code number4429-301
    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
    B.5.2Functional name of contact pointClinical Trial Helpdesk
    B.5.3 Address:
    B.5.3.1Street Address818 Stewart St., Suite 1110
    B.5.3.2Town/ citySeattle, WA
    B.5.3.3Post code98101
    B.5.3.4CountryUnited States
    B.5.4Telephone number+12068058310
    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 hydrochloride
    D.3.9.1CAS number 1141934-97-5
    D.3.9.2Current sponsor codeACU-4429
    D.3.9.3Other descriptive nameEMIXUSTAT HYDROCHLORIDE (ACU-4429)
    D.3.9.4EV Substance CodeSUB96210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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.IMP: 2
    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 hydrochloride
    D.3.9.1CAS number 1141934-97-5
    D.3.9.2Current sponsor codeACU-4429
    D.3.9.3Other descriptive nameEMIXUSTAT HYDROCHLORIDE (ACU-4429)
    D.3.9.4EV Substance CodeSUB96210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    Stargardt Disease
    E.1.1.1Medical condition in easily understood language
    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
    To determine if emixustat hydrochloride (emixustat) reduces the rate of progression of macular atrophy (MA) compared to placebo in subjects with Stargardt disease (STGD)
    E.2.2Secondary objectives of the trial
    1. To evaluate the safety and tolerability of emixustat compared to placebo
    2. To assess changes in retinal sensitivity as determined by photopic microperimetry with emixustat compared to placebo
    3. To assess changes in contrast sensitivity with emixustat compared to placebo
    4. To assess changes in reading speed with emixustat compared to placebo
    5. To assess changes in best-corrected visual acuity (BCVA) letter score with emixustat compared to placebo
    6. To assess changes in total area of decreased autofluorescence (definitely decreased plus questionably decreased) with emixustat compared to placebo
    7. To assess changes in total area of ellipsoid zone (EZ) loss with emixustat compared to placebo
    8. To assess changes in mean outer nuclear layer (ONL) thickness with emixustat compared to placebo
    9. To assess changes in quality-of-life instruments (QOLs) with emixustat compared to placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who meet all of the following criteria at Screening and Baseline (unless otherwise indicated) may be eligible for inclusion in the study:
    1. Males or females, age ≥ 16 years.
    2. Clinical diagnosis of MA secondary to STGD in one or both eyes as determined by the Investigator.
    3. The subject must have 1 or more pathogenic mutation(s) of the ABCA4 (ATP binding cassette subfamily A member 4) gene. If only one ABCA4 pathogenic mutation is identified or if two ABCA4 pathogenic mutations that typically occur on the same allele (ie, “in cis”) are identified, the subject must have a typical STGD phenotype (at least one eye has flecks at the level of the retinal pigmented epithelium [RPE] typically seen in STGD) and be approved by the Sponsor. If 2 or more pathogenic mutations that do not typically occur on the same allele are identified, a typical STGD phenotype and separate Sponsor approval are not required. Segregation analysis is not required. The pathogenicity of all mutations will be determined by the Sponsor working with experts in ophthalmic genetics.
    4. The study eye must meet the following criteria as determined by the central reading center’s assessment of FAF imaging at Screening:
    a. Total area of DDAF
    i. If the lesion is unifocal: ≥ 3.0 – 22.0 mm2 (~1.2 – 8.7 disc areas) in size.
    ii. If the lesion is multifocal: ≥ 1.0 – 22.0 mm2 (~0.4 – 8.7 disc areas) in size.
    b. The entire lesion must be completely visualized on the macula-centered image (Field 2 – Macula Image). The DDAF lesion must be able to be imaged in its entirety, and all lesion borders must be ≥ 300 microns from all image edges.
    5. Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA letter score of ≥ 25 letters (approximately ≥ 20/320 Snellen) in the study eye at Screening.
    6. Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of macular atrophy in the study eye as determined by the Investigator.
    7. Able to reliably administer oral medication by self or with available assistance.
    8. Able and willing to provide written informed consent/assent
    a. For subjects ≥ 18 years of age: able and willing to provide written informed consent before undergoing any study-related procedures.
    b. For subjects ≥ 16 and < 18 years of age: able and willing to provide written informed assent, and has a parent or legal guardian able and willing to provide written informed consent for the minor before the subject undergoes any study-related procedures. Where required by local regulations, both parents must consent to the subject’s participation in the study, if both have legal custody.
    E.4Principal exclusion criteria
    Subjects will be excluded from participation in the study if they meet any of the following criteria at Screening or Baseline (unless otherwise indicated):
    1. Macular atrophy associated with a condition other than STGD in either eye.
    2. DDAF with contiguous area of peripapillary atrophy in the study eye, as determined by the reading center.
    3. Mutation(s) in any of the following genes – elongation of very long chain fatty acids-like 4 (ELOVL4), prominin 1 (PROM1), or peripherin 2 (PRPH2)/retinal degeneration slow (RDS) – determined by the Sponsor working with experts in ophthalmic genetics to likely be disease-causing.
    4. If tested, any mutation:
    a. In a gene encoding a visual cycle protein [e.g., retinal pigment epithelium 65 (RPE65), lecithin:retinol acyltransferase (LRAT), retinol dehydrogenase 12 (RDH12), RDH5, and retinaldehyde binding protein 1 (RLBP1)], confirmed by the Sponsor working with experts in ophthalmic genetics to likely be disease-causing. Testing for these mutations is not required.
    b. Associated with a non-STGD retinal dystrophy/degeneration, confirmed by the Sponsor working with experts in ophthalmic genetics to likely be disease-causing. Testing is not required.
    5. 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, choroidal neovascularization, diabetic retinopathy, uveitis, other macular diseases, or uncontrolled glaucoma/ocular hypertension.
    6. History of any intraocular or ocular surface surgery in either eye ≤ 3 months prior to Screening.
    7. Current or previous participation in an interventional study to treat STGD using gene therapy or stem cell therapy.
    8. Current or previous participation in a study to treat STGD using a vitamin A derivative ≤ 6 months prior to Screening.
    9. Current or previous participation in a study to treat STGD using a complement inhibitor ≤ 6 months prior to Screening.
    10. Known hypersensitivity to emixustat or any of the excipients in emixustat tablets (ie, silicified microcrystalline cellulose, pregelatinized starch, colloidal silicon dioxide, and stearic acid).
    11. Prohibited medications: Please refer to the protocol.
    12. Any of the following laboratory abnormalities at Screening: Please refer to the protocol
    13. Participation in any study using an investigational drug within 30days or 5half-lives (of the investigational drug) of Screening.
    14. Participation in any study of an interventional, investigational device within 60days of Screening.
    15. Anticipated participation during the study period in any study using an investigational drug or an interventional, investigational device.
    16. Presence of other medical or ophthalmic disease, physical examination finding, or clinical laboratory finding that in the opinion of the Investigator contraindicates the use of an investigational drug, places the subject at risk by participating in the study, might interfere with the evaluation of the efficacy or safety of emixustat, negatively impacts subject compliance with the protocol, confounds the ability to interpret data from the study, or jeopardizes the subject’s ability to complete the protocol.
    17. Current or history of cancer (except for adequately treated basal cell or squamous cell carcinoma of the skin) within 1 year of Screening.
    18. History of myocardial infarction, stroke, unstable ischemic heart disease, uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure within 6months of Screening.
    19. Abnormal electrocardiogram (ECG) results that are considered by the Investigator to be clinically significant at Screening.
    20. Female subjects who are pregnant or lactating.
    21. Female subjects of childbearing potential [ie, not postmenopausal (without menses for 12 months without an alternative medical cause) and not surgically sterile via hysterectomy, bilateral salpingectomy, or bilateral oophorectomy] who are not willing to practice a medically accepted method of birth control with their non-surgically sterile male sexual partner from Screening through 30days following the completion of the study. Medically accepted methods of birth control include true abstinence, estrogen+progestogen hormonal contraceptives, progestogen-only hormonal contraceptives, nonhormonal or hormonal intrauterine contraceptive device, bilateral tubal occlusion, male or female condom, contraceptive sponge with spermicide, diaphragm with spermicide, or cervical cap with spermicide. True abstinence is when abstinence from heterosexual intercourse is in line with the preferred and usual lifestyle of the subject and is not just limited to the duration of this study.
    22. 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.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the mean rate of change from baseline in the total area of the MA lesion(s) in the study eye (in mm2 per year), defined as the area of definitely decreased autofluorescence as imaged by reduced-illuminance FAF.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 months
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints will include:
    1. Mean change from baseline in retinal sensitivity as assessed by photopic microperimetry
    2. Mean change from baseline in contrast sensitivity.
    3. Mean change from baseline in reading speed.
    4. Mean change from baseline in ETDRS BCVA letter score.
    5. Mean rate of change from baseline in the total area of decreased autofluorescence (definitely decreased plus questionably decreased), as imaged by reduced-illuminance FAF.
    6. Mean rate of change from baseline in the total area of EZ loss, as imaged on SD-OCT
    7. Mean change from baseline in mean ONL thickness, as imaged on SD-OCT
    8. Mean change from baseline in QOL instruments.
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 months
    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 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) No
    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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 EEA14
    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
    Brazil
    Canada
    Denmark
    France
    Germany
    Italy
    Netherlands
    South Africa
    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 years3
    E.8.9.1In the Member State concerned months8
    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 months8
    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: 7
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Minor 16 to <18
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 89
    F.4.2.2In the whole clinical trial 194
    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 Decision2019-04-08
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-06-23
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