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A 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

Sponsor
Apellis Pharmaceuticals, Inc. (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT03525613
Collaborator
(none)
600
Enrollment
112
Locations
4
Arms
53
Anticipated Duration (Months)
5.4
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a 30-month, Phase III, multicenter, randomized, double-masked, sham-injection controlled study to assess the efficacy and safety of multiple IVT injections of APL-2 in subjects with GA secondary to AMD.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
600 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal Pegcetacoplan Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Actual Study Start Date :
Aug 30, 2018
Actual Primary Completion Date :
Jun 28, 2021
Anticipated Study Completion Date :
Jan 30, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: APL-2 15mg 0.1 mL Monthly for 24 months

A single dose of 15 mg APL-2/0.1 mL will be administered via intravitreal injection in this study. Subjects will receive an injection every month

Drug: APL-2
Complement (C3) Inhibitor
Experimental: APL-2 15mg 0.1 mL EOM for 24 months

A single dose of 15 mg APL-2/0.1 mL will be administered via intravitreal injection in this study. Subjects will receive an injection every other month

Drug: APL-2
Complement (C3) Inhibitor
Experimental: Sham Procedure Monthly for 24 months

Sham Procedure monthly for 24 months

Other: Sham Procedure
Subjects will receive a Sham procedure every month
Experimental: Sham Procedure Every Other Month for 24 months

Sham Procedure every other month for 24 months

Other: Sham Procedure
Subjects will receive a Sham procedure every other month

Outcome Measures

Primary Outcome Measures

  1. Change from Baseline to Month 12 in total area of GA lesion(s) in the study eye (in mm2 ) based on Fundus Autofluorescence (FAF) [12 months]

Secondary Outcome Measures

  1. Incidence and severity of ocular and systemic treatment-emergent adverse events [30 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:
60 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

The study eye must meet all inclusion criteria. If both eyes meet the inclusion criteria, the eye with the worst visual acuity at the screening visit will be designated as the study eye. If both eyes have the same visual acuity, the right eye will be selected as the study eye.

Ocular- specific inclusion criteria apply to the study eye only, unless otherwise specified.

  • Age ≥ 60 years.

  • Normal Luminance best corrected visual acuity of 24 letters or better using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately 20/320 Snellen equivalent).

  • Clinical diagnosis of GA of the macula secondary to AMD as determined by the Investigator and confirmed by the Reading Center.

  • The GA lesion must meet the following criteria as determined by the central reading center's assessment of Fundus Autofluorescence (FAF) imaging at screening:

  • Total GA area must be ≥ 2.5 and ≤ 17.5 mm2 (1 and 7 disk areas [DA] respectively)

  • If GA is multifocal, at least one focal lesion must be ≥ 1.25 mm2 (0.5 DA), with the overall aggregate area of GA, as specified above in 4a.

  • The entire GA lesion must be completely visualized on the macula centered image and must be able to be imaged in its entirety and not contiguous with any areas of peripapillary atrophy.

  • Presence of any pattern of hyperautofluorescence in the junctional zone of GA. Absence of hyperautofluorescence (i.e. pattern = none) is exclusionary.

  • Adequate clarity of ocular media, adequate pupillary dilation, and fixation to permit the collection of good quality images as determined by the Investigator.

  • Meets the following criteria related to microperimetry:

  • Able to detect fixation target.

  • Total elapsed time to complete the 10-2 68 point exam is ≤ 30 minutes in duration.

  • Reliability test ratio must be ≤ 20%.

  • Subject is willing and able to undertake microperimetry assessment in the opinion of the investigator.

  • Female subjects must be:

  • Women of non-child-bearing potential (WONCBP), or

  • Women of child-bearing potential (WOCBP) with a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study and refrain from breastfeeding for the duration of the study.

  • Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study.

  • Willing and able to give informed consent and to comply with the study procedures and assessments.

Exclusion Criteria:

Ocular specific exclusion criteria apply to the study eye only, unless otherwise specified.

  • GA secondary to a condition other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like plaquenil maculopathy in either eye.

  • Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm.

  • Any history or active choroidal neovascularization (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.

  • 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).

  • Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.

  • History of laser therapy in the macular region.

  • 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.

  • 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.

  • Any contraindication to IVT injection including current ocular or periocular infection.

  • History of prior intravitreal injection.

  • Unable to perform microperimetry reliably in the opinion of the investigator

  • Prior participation in another interventional clinical study for intravitreal therapies in either eye (including subjects receiving sham).

  • Prior participation in another interventional clinical study for geographic atrophy in either eye including investigational oral medication and placebo.

  • 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. Note: clinical trials solely involving observation, over-the-counter vitamins, supplements, or diets are not exclusionary.

  • 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.

  • 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.

  • Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to APL-2 or any of the excipients in APL-2 solution.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Arizona Retina & Vitreous Consultants Phoenix Arizona United States 85021
2 California Retina Consultants Bakersfield California United States 93309
3 Retina Vitreous Associates Medical Group Beverly Hills California United States 90211
4 Retinal Diagnostic Center Campbell California United States 95508
5 Retina Consultants of Orange County Fullerton California United States 92835
6 Atlantis Eyecare Huntington Beach California United States 92647
7 University of California, San Diego, Jacobs Retina La Jolla California United States 92093
8 Byers Eye Institute at Stanford, Stanford School of Medicine Palo Alto California United States 94303
9 Doheny Eye Center UCLA Pasadena California United States 91105
10 Retina Consultants San Diego Poway California United States 92064
11 Retinal Consultants Med Group, Inc. Sacramento California United States 95819
12 Orange County Retina Medical Group Santa Ana California United States 92705
13 California Retina Consultants Santa Barbara California United States 93103
14 Southwest Retina Research Center, LLC Durango Colorado United States 81301
15 Colorado Retina Associates Golden Colorado United States 80401
16 Retina Group of New England Waterford Connecticut United States 06385
17 Blue Ocean Clinical Research / The Macula Center Clearwater Florida United States 33761
18 National Ophthalmic Research Institute (Retina Consultants of Southwest Florida) Fort Myers Florida United States 33912
19 Bascom Palmer Eye Institute Miami Florida United States 33136
20 Bascom Palmer Eye Institute of Naples Naples Florida United States 34103
21 Retina Specialty Institute Pensacola Florida United States 32503
22 Center for Retina and Macular Disease Winter Haven Florida United States 33880
23 Southeast Retina Center, PC Augusta Georgia United States 30909
24 Gailey Eye Clinic Retina Center Bloomington Illinois United States 61704
25 Northwestern Feinberg School of Medicine Chicago Illinois United States 60611
26 Rush University Medical Center Chicago Illinois United States 60612
27 Midwest Eye Institute Indianapolis Indiana United States 46290
28 Retina and Vitreous Associates of Kentucky, PSC dba Retina Associates of Kentucky Lexington Kentucky United States 40509
29 Retina Associates New Orleans Metairie Louisiana United States 70006
30 The Retina Care Center Baltimore Maryland United States 21209
31 The Retina Group of Washington Chevy Chase Maryland United States 20815
32 Cumberland Valley Retina Center Hagerstown Maryland United States 21740
33 Mid Atlantic Retina Specialists Hagerstown Maryland United States 21740
34 Tufts Medical Center Boston Massachusetts United States 02116
35 New England Retina Consultants Springfield Massachusetts United States 01107
36 Michigan Medicine Kellogg Eye Center Ann Arbor Michigan United States 48105
37 Associated Retinal Consultants, P.C Grand Rapids Michigan United States 49564
38 VitreoRetinal Surgery PA Minneapolis Minnesota United States 55435
39 Sierra Eye Associates Reno Nevada United States 89502
40 Retina Center of NJ, LLC Bloomfield New Jersey United States 07003
41 NJ Retina Toms River New Jersey United States 08755
42 Vision Research Center Eye Associates of NM Albuquerque New Mexico United States 87109
43 Ophthalmic Consultants of Long Island Lynbrook New York United States 11563
44 New York Eye and Ear Infirmary of Mount Sinai New York New York United States 10003
45 Vitreous Retina Macula Consultants of NY New York New York United States 10022
46 Duke University, Duke Eye Center Durham North Carolina United States 27710
47 Research - Retina Vitreous Center Edmond Oklahoma United States 73013
48 Oregon Retina, LLP Eugene Oregon United States 97405
49 Mid Atlantic Retina Bethlehem Pennsylvania United States 18017
50 Retina Vitreous Consultants Monroeville Pennsylvania United States 15146
51 Mid Atlantic Retina, Wills Eye Hospital Philadelphia Pennsylvania United States 19107
52 AIO Visionary Eye Care West Mifflin Pennsylvania United States 15122
53 Charles Retina Institute Germantown Tennessee United States 38138
54 Tennessee Retina, PC Nashville Tennessee United States 37203
55 Retina Research Institute of Texas Abilene Texas United States 79606
56 Texas Retina Associates Dallas Texas United States 75231
57 Houston Eye Associates Houston Texas United States 77025
58 Retina Consultants of Houston, PA Houston Texas United States 77030
59 San Antonia Eye Center San Antonio Texas United States 78215
60 Medical Center Ophthalmology Associates San Antonio Texas United States 78240
61 Retina Associates of South Texas San Antonio Texas United States 78240
62 Retinal Consultants of San Antonio San Antonio Texas United States 78240
63 Brown Retina Institute San Antonio Texas United States 78251
64 Strategic Clinical Research Group Willow Park Texas United States 76087
65 University of Utah - John A. Moran Center Salt Lake City Utah United States 84132
66 Emerson Clinical Research Institute Falls Church Virginia United States 22046
67 University of Wisconsin Madison Wisconsin United States 53705
68 Marsden Eye Specialist Parramatta New South Wales Australia 2150
69 Adelaide Eye & Retina Clinic Adelaide South Australia Australia 5000
70 Save Sight Institute Sydney South Block Australia 2000
71 Lions Eye Institute Nedlands Western Australia Australia 6009
72 Strathfield Retina Clinic Strathfield Australia 2135
73 Sydney West Retina Westmead Australia 2145
74 UNIFESP - Federal University São Paulo Brazil 04021-001
75 UHN Toronto Western Hospital Toronto Ontario Canada M5T 2S8
76 AXON Clinical S.R.O Praha Czechia 150 00
77 Gemini Eye Clinic Zlín Czechia 76001
78 CHU de Bordeaux Bordeaux France 33076
79 CHU Dijon Cedex France 21 079
80 Centre Hospitalier Intercommunal de Créteil Créteil France 94010
81 Hopital Lariboisière Paris France 75010
82 CHNO des Quinze-Vingts Paris France 75012
83 Centre Ophtalmologique d'Imagerie et Laser Paris France 75015
84 CHU de Strasbourg Hopital Civil Strasbourg France 67091
85 Clinique du Val d'Ouest Écully France 69130
86 Universitäts-Augenklinik Bonn Bonn Germany 53127
87 University Opthalmology Clinic Freiburg im Breisgau Germany 79106
88 Universitätsmedizin Göttingen Georg-August-Universität Göttingen Germany 37075
89 Universitätsklinikum Schleswig-Holstein Lübeck Germany 23538
90 Augenklinik der LMU München München Germany 80336
91 Augenzentrum am St. Franziskus-Hospital Münster Germany 48145
92 Universitäts-Augenklinik Münster Germany 48149
93 STZ Eyetrial Tübingen Germany 72076
94 Rambam Medical Centre Haifa Israel 3109601
95 Carmel Medical Center Haifa Israel 3436212
96 Hadassah Medical Center Jerusalem Israel 91120
97 Ospedale San Raffaele Milano Italy 20132
98 Luigi Sacco Hospital Milano Italy 20157
99 IRCCS Fondazione G.B. Bietti Roma Italy 128
100 Academisch Medisch Centrum Amsterdam Netherlands 1105 AZ
101 Radboud University Medical Center Oogheelkunde Nijmegen Netherlands 6525
102 Southern Eye Specialists Christchurch New Zealand 8013
103 Oculomedica Eye Centre Bydgoszcz Poland 85-316
104 Oftalmika Eye Hospital Bydgoszcz Poland 85-631
105 Eye Surgery Center Professor Zagorski Rzeszów Poland 35-017
106 Hospital Universitario Puerta de Hierro Majadahonda Madrir Spain 2822
107 Centro Médico Teknon Barcelona Spain 08022
108 Centro de Oftalmologia Barraquer Muntaner Spain 314
109 The Royal Victoria Hospital Belfast United Kingdom BT12 6BA
110 Liverpool University Hospitals NHS Foundation Trust Liverpool United Kingdom L7 8XP
111 Sunderland Eye Infirmary Sunderland United Kingdom SR2 9HP
112 York Teaching Hospital NHS Foundation Trust York United Kingdom YO31 8HE

Sponsors and Collaborators

  • Apellis Pharmaceuticals, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Apellis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT03525613
Other Study ID Numbers:
  • APL2-304
First Posted:
May 15, 2018
Last Update Posted:
Jun 22, 2022
Last Verified:
Jun 1, 2022
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Macular Degeneration
Geographic Atrophy
Atrophy

Study Results

No Results Posted as of Jun 22, 2022