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    Summary
    EudraCT Number:2018-001436-22
    Sponsor's Protocol Code Number:APL2-303
    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:2018-09-27
    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 number2018-001436-22
    A.3Full title of the trial
    A Phase III, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy with Sham Injections in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical research study involving an intravitreal injection in the eye with APL-2 or a sham injection for the treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration
    A.3.2Name or abbreviated title of the trial where available
    Derby
    A.4.1Sponsor's protocol code numberAPL2-303
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03525600
    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 SponsorApellis 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 supportApellis Pharmaceuticals
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationApellis Pharmaceuticals
    B.5.2Functional name of contact pointClin. Development and Med. Affairs
    B.5.3 Address:
    B.5.3.1Street Address6400 Westwind Way, Suite A
    B.5.3.2Town/ cityCrestwood
    B.5.3.3Post codeKY 40014
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16179775701
    B.5.6E-mailfederico@apellis.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 nameAPL-2
    D.3.2Product code APL 2
    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 INNpegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.3Other descriptive nameAPL-2
    D.3.9.4EV Substance CodeSUB192794
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    Geographic Atrophy Secondary to Age-Related Macular Degeneration
    E.1.1.1Medical condition in easily understood language
    Geographic Atrophy Secondary to Age-Related 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 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
    To evaluate the efficacy of APL-2 compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by FAF.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of APL-2 compared to sham injection in patients with GA secondary to AMD with respect to:
    • Low luminance best corrected visual acuity score (LL-BCVA) in the study eye
    • Low luminance deficit (LLD) in the study eye
    • Total area of GA lesion(s) in the study eye
    • Monocular critical print size (study eye), as assessed by MNRead or Radner Reading Charts
    • National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) distance activity subscale score (in select countries)
    • To evaluate the PK of APL-2 as assessed by systemic plasma concentration of APL-2 (in select sites)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 60 years.
    2. NL-BCVA of 24 letters or better using ETDRS charts.
    3. Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center.
    4. The GA lesion must meet the criteria as determined by the central Reading Center's assessment of FAF imaging at screening.
    5. Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the Investigator.
    6. Female subjects must be women of non-child-bearing potential or women of child-bearing potential with a negative pregnancy test at screening and must agree to use protocol defined methods of contraception.
    7. Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception
    8. Willing and able to give informed consent and to comply with the study procedures and assessments.
    E.4Principal exclusion criteria
    1. GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye.
    2. Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm.
    3. Any history or active CNV, associated with AMD or any other cause, including any evidence of retinal pigment epithelium rips or evidence of neovascularization anywhere based on SD-OCT imaging and/or fluorescein angiography as assessed by the Reading Center.
    4. Presence 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 (e.g. clinically significant epiretinal membrane [ERM], full thickness macular hole or uncontrolled glaucoma/ocular hypertension). Benign conditions in the opinion of the investigator such as peripheral retina dystrophy are not exclusionary.
    5. Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
    6. History of laser therapy in the macular region.
    7. Aphakia or absence of the posterior capsule. Note: YAG laser posterior capsulotomy for posterior capsule opacification done at least 60 days prior to screening is not exclusionary.
    8. Any ocular condition other than GA secondary to AMD that may require surgery or medical intervention during the study period or, in the opinion of the Investigator, could compromise visual function during the study period
    9. Any contraindication to IVT injection including current ocular or periocular infection
    10. History of prior intravitreal injection.
    11. Prior participation in another interventional clinical study for intravitreal therapies in either eye (including subjects receiving sham).
    12. Prior participation in another interventional clinical study for geographic atrophy in either eye including investigational oral medication and placebo.
    13. Participation in any systemic experimental treatment or any other systemic investigational new drug within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of study treatment.
    14. Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the 24-month treatment period unlikely, or would make the subject an unsafe study candidate.
    15. Any screening laboratory value (hematology, serum chemistry or urinalysis) that in the opinion of the Investigator is clinically significant and not suitable for study participation.
    16. Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to APL-2 or any of the excipients in APL-2 solution.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Month 12 in total area of GA lesion(s) in the study eye (in mm2) based on Fundus Autofluorescence (FAF).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 12
    E.5.2Secondary end point(s)
    • Change from baseline in monocular reading speed (study eye), as assessed by Minnesota Reading (MNRead) or Radner Reading Charts
    • Change from baseline in Functional Reading Independence Index (FRI) composite score
    • Change from baseline in normal luminance best-corrected visual acuity score (NL-BCVA) as assessed by ETDRS chart.
    • Change from baseline in low luminance best corrected visual acuity score (LL-BCVA) as assessed by ETDRS chart.
    • Change from baseline in low luminance deficit (LLD)
    • Change from baseline at each planned assessment in the total area of GA lesion(s) in the study eye (in mm2) as assessed by FAF.
    • Change from baseline in monocular critical print size (study eye), as assessed by MNRead or Radner Reading Charts, (in selected countries).
    • Change from baseline in the National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI VFQ-25) distance activity subscale score (in select countries)
    • Systemic Plasma concentration of APL-2 (in select sites)
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Month 24 - Change from baseline in monocular reading speed MNRead or Radner Reading Charts
    • Month 24 - Change from baseline in FRI composite score
    • Month 24 - Change from baseline in NL-BCVA
    • Month 12, Month 24 - Change from baseline in LL-BCVA
    • Month 12, Month 24 - Change from baseline in LLD
    • Month 12, Month 24 - Change from baseline in monocular critical print size
    • Month 12, Month 24 - Change from baseline in NEI VFQ-25 distance activity subscale score
    • Over time - Systemic Plasma concentration of APL-2 (in select sites)
    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 No
    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) 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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Sham procedure
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA45
    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
    Brazil
    Canada
    Czech Republic
    France
    Germany
    Israel
    Italy
    Netherlands
    New Zealand
    Poland
    Russian Federation
    Spain
    Ukraine
    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 years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    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 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: 50
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 550
    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 state24
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 270
    F.4.2.2In the whole clinical trial 600
    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)
    Subjects will be offered entry into an open-label extension study at the end of the 24 month treatment period; those who enter the open label extension study will not require the 6 month follow-up period (Months 27 and 30).
    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 Decision2019-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-06-20
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