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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)

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ClinicalTrials.gov Identifier: NCT03525613
Recruitment Status : Recruiting
First Posted : May 15, 2018
Last Update Posted : October 2, 2018
Sponsor:
Information provided by (Responsible Party):
Apellis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE April 20, 2018
First Posted Date  ICMJE May 15, 2018
Last Update Posted Date October 2, 2018
Actual Study Start Date  ICMJE August 30, 2018
Estimated Primary Completion Date April 30, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 17, 2018)
Change from Baseline to Month 12 in total area of GA lesion(s) in the study eye (in mm2 ) based on Fundus Autofluorescence (FAF) [ Time Frame: 12 months ]
Original Primary Outcome Measures  ICMJE
 (submitted: May 3, 2018)
Mean change from baseline to Month 12 in total area of GA lesion(s) in the study eye (mm2 ) based on Fundus Autofluorescence (FAF) [ Time Frame: 12 months ]
Change History Complete list of historical versions of study NCT03525613 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 3, 2018)
Incidence and severity of ocular and systemic treatment-emergent adverse events [ Time Frame: 30 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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)
Official Title  ICMJE 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)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Geographic Atrophy
Intervention  ICMJE
  • Drug: APL-2
    Complement (C3) Inhibitor
  • Other: Sham Procedure
    Subjects will receive a Sham procedure every month
  • Other: Sham Procedure
    Subjects will receive a Sham procedure every other month
Study Arms  ICMJE
  • 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
    Intervention: Drug: APL-2
  • 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
    Intervention: Drug: APL-2
  • Experimental: Sham Procedure Monthly for 24 months
    Sham Procedure monthly for 24 months
    Intervention: Other: Sham Procedure
  • Experimental: Sham Procedure Every Other Month for 24 months
    Sham Procedure every other month for 24 months
    Intervention: Other: Sham Procedure
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 3, 2018)
600
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 30, 2022
Estimated Primary Completion Date April 30, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

  1. Age ≥ 60 years.
  2. Normal Luminance best corrected visual acuity of 24 letters or better using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately 20/320 Snellen equivalent).
  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 following criteria as determined by the central reading center's assessment of Fundus Autofluorescence (FAF) imaging at screening:

    1. Total GA area must be ≥ 2.5 and ≤ 17.5 mm2 (1 and 7 disk areas [DA] respectively)
    2. 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.
    3. 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.
    4. Presence of any pattern of hyperautofluorescence in the junctional zone of GA. Absence of hyperautofluorescence (i.e. pattern = none) is exclusionary.
  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. Meets the following criteria related to microperimetry:

    1. Able to detect fixation target.
    2. Total elapsed time to complete the 10-2 68 point exam is ≤ 30 minutes in duration.
    3. Reliability test ratio must be ≤ 20%.
    4. Subject is willing and able to undertake microperimetry assessment in the opinion of the investigator.
  7. Female subjects must be:

    1. Women of non-child-bearing potential (WONCBP), or
    2. 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.
  8. 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.
  9. 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.

  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 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.
  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. Unable to perform microperimetry reliably in the opinion of the investigator
  12. Prior participation in another interventional clinical study for intravitreal therapies in either eye (including subjects receiving sham).
  13. Prior participation in another interventional clinical study for geographic atrophy in either eye including investigational oral medication and placebo.
  14. 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.
  15. 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.
  16. 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.
  17. Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to APL-2 or any of the excipients in APL-2 solution.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 60 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Natalie Gagne 617-977-5708 natalie@apellis.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03525613
Other Study ID Numbers  ICMJE APL2-304
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Apellis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Apellis Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Apellis Pharmaceuticals, Inc.
Verification Date October 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP