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    Summary
    EudraCT Number:2021-001198-22
    Sponsor's Protocol Code Number:ALXN2040-GA-201
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-03-17
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2021-001198-22
    A.3Full title of the trial
    A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study
    of Danicopan (ALXN2040) in Patients with Geographic Atrophy (GA)
    Secondary to Age-Related Macular Degeneration (AMD)
    A danikopán (ALXN2040) II.fázisú, kétszeresen maszkolt, placebokontrollos, dóziskereső vizsgálata életkorral összefüggőmakuladegeneráció (AMD) miatti geografikus atrófiában (GA) szenvedő betegeknél
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
    A.4.1Sponsor's protocol code numberALXN2040-GA-201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05019521
    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 SponsorAlexion Pharmaceuticals, 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 supportAlexion Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address103-105 Rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.3.4CountryFrance
    B.5.4Telephone number33 1 47100615
    B.5.5Fax number33 1 47100611
    B.5.6E-mailclinicaltrials.eu@alexion.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 nameDanicopan (ALXN2040)
    D.3.2Product code ACH-0144471
    D.3.4Pharmaceutical form Film-coated 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 INNDANICOPAN
    D.3.9.1CAS number 1903768-17-1
    D.3.9.2Current sponsor codeACH-0144471
    D.3.9.3Other descriptive nameDANICOPAN
    D.3.9.4EV Substance CodeSUB189885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboFilm-coated tablet
    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 (AD) secondary to Age-related Macular Degeneration (AMD).
    E.1.1.1Medical condition in easily understood language
    Geographic Atrophy (GA) is an advanced form of age-related macular degeneration. It is a progressive, irreversible condition of the eye that causes severe loss of visual function.
    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 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
    This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD.
    E.2.2Secondary objectives of the trial
    To evaluate the effect of danicopan on disease progression utilizing anatomical measures in the study eye
    To evaluate the effect of danicopan on disease progression utilizing functional measures in the study eye
    To evaluate the effect of danicopan on patient reported outcomes (PROs) in patients with GA secondary to AMD
    To evaluate the PK and PD of danicopan in patients with GA secondary to AMD
    To evaluate the safety and tolerability of danicopan in patients with GA secondary to AMD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Key Inclusion Criteria:
    • Age ≥ 60 years, male or female
    • Presentation of GA secondary to AMD in at least 1 eye.
    • Study eye must have the specified VA (range of 84 to 24 letters; 20/20 to 20/320) using Early Treatment Diabetic Retinopathy Study charts at starting distance of 4 meters.
    • Total GA lesion area of 0.5 to 17.76 mm2 (~0.2 to 7 disc area [DA]) per eye measured by FAF. If GA is multifocal, at least one focal lesion must be ≥ 0.5 mm2 (~0.2 DA).
    • The entire GA lesion must be >1 μm outside of the foveal center

    Note: Additional inclusion/exclusion criteria may apply, per protocol.
    E.4Principal exclusion criteria
    Key Exclusion Criteria:
    • GA in the study eye due to cause other than AMD
    • Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye for intraocular vascular disease
    • Have previously received any stem cell or gene therapy for any ophthalmological condition in either eye
    • Use of any investigational medicinal product (ie, participation in interventional clinical studies for any ophthalmic indications) or use of any regulatory approved treatment for GA in the study eye regardless of route of administration within the last 3 months or 5 half-lives of the last dose of the investigational or commercial product (whichever is longer)
    • Presence of active ocular diseases in the study eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments
    • Known or suspected complement deficiency
    • Hypersensitivity to the investigational drug (danicopan) or any of its excipients, or to fluorescein sodium for injection
    • History or presence of any medical or psychological condition that, in the opinion of the Principal Investigator, would make the patient inappropriate for the study or unable to comply with study procedures or put the patient at undue risk or confound the results of the study.

    Note: Additional inclusion/exclusion criteria may apply, per protocol.
    E.5 End points
    E.5.1Primary end point(s)
    1. Change from Baseline to Week 52 in the square root (sqrt) of total GA lesion area (mm) in the study eye as measured by fundus autofluorescence (FAF)
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 52
    E.5.2Secondary end point(s)
    2. Change from Baseline to Week 104 in the sqrt of the total GA lesion area in the study eye as measured by FAF
    [Time Frame: Baseline, Week 52 and Week 104]

    3. Change from Baseline to Week 52 and Week 104 in the total GA lesion area in the study eye as measured by FAF
    [Time Frame: Baseline, Week 52 and Week 104]

    4. Change from Baseline to Week 52 and Week 104 in monocular Best-corrected Visual Acuity (BCVA) scores in the study eye as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
    [Time Frame: Baseline, Week 52 and Week 104]

    5. Change from Baseline to Week 52 and Week 104 in monocular Low Luminance Visual Acuity (LLVA) scores in the study eye as assessed by the ETDRS chart
    [Time Frame: Baseline, Week 52 and Week 104]

    6. Change from Baseline to Week 52 and Week 104 in monocular low luminance deficit (BCVA-LLVA) in the study eye
    [Time Frame: Baseline, Week 52 and Week 104]

    7. Change form Baseline to Week 52 and Week 104 in monocular reading speeds in the study eye as assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Charts
    [Time Frame: Baseline, Week 52 and Week 104]

    8. Change from Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores
    [Time Frame: Baseline, Week 52 and Week 104]

    9. Plasma concentration of Danicopan over time
    [Time Frame: Up to 4 hours postdose]

    10. Ex vivo serum Alternative Pathway activity
    [Time Frame: Up to 4 hours postdose]

    11. Plasma Bb concentration over time
    [Time Frame: Up to 4 hours postdose]

    12. Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Ocular TEAEs, SAEs, and clinical laboratory abnormalities, and events leading to discontinuation of study drug throughout the study
    [Time Frame: Day 1 through Week 104]
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 52
    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 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 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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA92
    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
    Australia
    Canada
    Czechia
    France
    Germany
    Hungary
    Italy
    Japan
    Korea, Republic of
    Latvia
    Slovakia
    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
    The end of the study is defined as the date the last patient completes
    the last visit (including the OLE, Taper, and Follow-up.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 232
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 160
    F.4.2.2In the whole clinical trial 332
    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 Protocol does not define any post-study treatment plan. Patients completing the study may receive commercial Danicopan once available in their country.
    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 Decision2022-05-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-16
    P. End of Trial
    P.End of Trial StatusOngoing
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