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    Summary
    EudraCT Number:2020-002931-32
    Sponsor's Protocol Code Number:APL2-GA-305
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-06-02
    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 ConcernedFrance - ANSM
    A.2EudraCT number2020-002931-32
    A.3Full title of the trial
    A Phase 3 open-label, multicenter, extension study to evaluate the long-term safety and efficacy of pegcetacoplan in subjects with geographic atrophy secondary to age-related macular degeneration
    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 Pegcetacoplan for the treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration
    A.4.1Sponsor's protocol code numberAPL2-GA-305
    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 Address100 5th Avenue
    B.5.3.2Town/ cityWaltham
    B.5.3.3Post codeMA 02451
    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 namePegcetacoplan
    D.3.2Product code Pegcetacoplan
    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 long-term safety of IVT injected pegcetacoplan.
    E.2.2Secondary objectives of the trial
    1. To assess changes in the total area of geographic atrophy (GA) lesion in the study eye measured by fundus autofluorescence (FAF).
    2. To assess changes in visual function as measured by:
    a. Normal-luminance best-corrected visual acuity score (NL-BCVA) in the study eye
    b. Low-luminance best-corrected visual acuity score (LL-BCVA) in the study eye
    c. Low-luminance deficit (LLD) in the study eye
    d. Reading speed in the study eye
    3. To assess the macular functional response as assessed by mesopic microperimetry in the
    study eye (selected subjects [those who had the assessment performed in the antecedent
    study] only).
    4. To evaluate changes in subject-reported outcomes as measured by:
    a. National Eye Institute Visual Functioning Questionnaire 25 Item Version (NEI VFQ-25)
    b. Functional Reading Independence (FRI) Index.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Ocular-specific inclusion criteria apply to the study eye only.
    1. Participated in APL2-103 (NCT03777332) or completed the treatment at Month 24 of
    either APL2-303 (Derby, NCT03525613) or APL2-304 (Oaks, NCT03525600). Specifically, for the APL2-303 and APL2-304 studies, the following criterion also applies:
    a. Subjects who did not permanently discontinue treatment but missed the Month 24 visit are also eligible to participate in this extension study; however, to be eligible,
    these subjects must be screened within 60 days from the last day of the expected
    Month 24 visit window for the antecedent study.
    2. Clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection
    of good quality images as determined by the investigator.
    3. Female subjects must be:
    a. Women of nonchildbearing potential, or
    b. Women of childbearing potential (WOCBP) with a negative serum pregnancy test at
    screening and must agree to use protocol defined methods of contraception for the
    duration of the study and 90 days after their last dose of pegcetacoplan, and refrain
    from breastfeeding for the duration of the study.
    4. Males with female partners of childbearing potential must agree to use protocol defined
    methods of contraception and agree to refrain from donating sperm for the duration of the
    study and for 90 days after their last dose of pegcetacoplan.
    5. Willing and able to give informed consent and to comply with the study procedures and assessments.
    E.4Principal exclusion criteria
    Ocular-specific exclusion criteria apply to the study eye only.
    1. Subjects who permanently discontinued the study drug prior to Month 24 in the APL2-304 or APL2-304 studies and only for safety assessments. Temporary pause of the study drug is not exclusionary.
    2. Presence of an active ocular disease that, in the opinion of the investigator, compromises
    or confounds visual function, including, but not limited to, macular hole or other macular
    diseases (eg, clinically significant epiretinal membrane). Benign conditions in the opinion
    of the investigator such as peripheral retinal dystrophy are not exclusionary.
    3. Any contraindication to IVT injection including current ocular or periocular infection.
    4. Medical or psychiatric condition that, in the opinion of the investigator, is clinically
    significant and not suitable for study participation or make consistent follow-up over the 36-month treatment period unlikely.
    5. Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to
    pegcetacoplan or any of the excipients in pegcetacoplan solution.
    6. Pregnancy, breastfeeding, or positive pregnancy test.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence and severity of ocular and systemic AEs
    E.5.1.1Timepoint(s) of evaluation of this end point
    Incidence and severity of ocular and systemic AEs (time frame: up to 36 months)
    E.5.2Secondary end point(s)
    • The total area of GA lesion(s) in the study eye (in mm2) as assessed by fundus autofluorescence (FAF)
    • NL-BCVA score (study eye) as assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) chart
    • LL-BCVA score (study eye) as assessed by ETDRS chart
    • LLD (study eye)
    • Monocular maximum reading speed (study eye) and monocular critical print size, as assessed by Minnesota Reading (MNRead) charts or Radner
    • Binocular maximum reading speed and binocular critical print size, as assessed by MNRead charts or Radner.
    • The number of absolute scotomatous points (study eye) assessed by mesopic microperimetry (selected subjects [those who had the assessment performed in the antecedent study] only)
    • Macular sensitivity (study eye) as assessed by mesopic microperimetry (selected subjects [those who had the assessment performed in the antecedent study] only)
    • Mean FRI Index score
    • NEI VFQ-25 composite score, near activity subscale score, and distance activity subscale score
    E.5.2.1Timepoint(s) of evaluation of this end point
    •The total area of GA lesion(s) in the study eye (in mm2) as assessed by FAF at Month 12, 24 and 36.
    • NL-BCVA score (study eye) and LL-BCVA score (study eye) as assessed by ETDRS chart at Month 12, 24 and 36.
    • LLD (study eye) at Month 12, 24 and 36.
    • Monocular maximum reading speed (study eye) and monocular critical print size at Month 12, 24 and 36.
    • Binocular maximum reading speed and binocular critical print size at Month 12, 24 and 36.
    The number of absolute scotomatous points (study eye) at Month 12, 24, and 36.
    • Macular sensitivity (study eye) at Month 12, 24, and 36.
    • Mean FRI Index score at Month 12, 24, and 36
    • NEI VFQ-25 composite score, near activity subscale score, and distance activity subscale score at Month 12, 24, and 36.
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 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 concerned14
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA52
    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
    Israel
    New Zealand
    United States
    France
    Germany
    Italy
    Netherlands
    Poland
    Spain
    United Kingdom
    Czechia
    Argentina
    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 months0
    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 months0
    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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1160
    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 state75
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 206
    F.4.2.2In the whole clinical trial 1200
    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)
    There are no current plans for pegcetacoplan treatment after study completion. Patients will receive the local standard of care for their disease as required after the trial ends.
    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 Decision2021-08-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-07-27
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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