A Phase 3 Safety and Efficacy Study of Intravitreal Administration of Zimura (Complement C5 Inhibitor)

• ClinicalTrials.gov Identifier: NCT04435366
• Recruitment Status: Completed
• First Posted: June 17, 2020
• Results First Posted: December 28, 2023
• Last Update Posted: February 23, 2024
• Sponsor: IVERIC bio, Inc.
• Information provided by (Responsible Party): Astellas Pharma Inc ( IVERIC bio, Inc. )

Tracking Information

• First Submitted Date: May 12, 2020
• First Posted Date: June 17, 2020
• Results First Submitted Date: November 1, 2023
• Results First Posted Date: December 28, 2023
• Last Update Posted Date: February 23, 2024
• Actual Study Start Date: June 22, 2020
• Actual Primary Completion Date: July 25, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 7, 2023)

Mean Rate of Change in GA Over 12 Months (Measured at Three Time Points: Baseline, Month 6, and Month 12) [ Time Frame: Baseline and Month 12 ]

The least squares mean rate of growth (slope) estimated based on geographic atrophy (GA) area measured by fundus autofluorescence (FAF) in at least 3 timepoints: Baseline, Month 6, and Month 12 was used to determine mean rate of change (slope) in GA from Baseline to Month 12. The square root of the GA area was used in the analysis.

Original Primary Outcome Measures (submitted: June 15, 2020)

Primary Outcome Measure [ Time Frame: Baseline, Month 6, Month 12 ]

Mean rate of change in GA over 12 months measured at three time points: Baseline, Month 6, and Month 12

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 3 Safety and Efficacy Study of Intravitreal Administration of Zimura (Complement C5 Inhibitor)
• Official Title: A Phase 3 Multicenter, Randomized, Double Masked, Sham- Controlled Clinical Trial to Assess the Safety and Efficacy of Intravitreal Administration of Zimura (Complement C5 Inhibitor) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration
• Brief Summary: The objectives of this study are to evaluate the safety and efficacy of Zimura (avacincaptad pegol) intravitreal administration in patients with geographic atrophy secondary to age-related macular degeneration (AMD)

Detailed Description

Participants will be randomized in a 1:1 ratio to the following monthly treatment groups:

• Avacincaptad pegol 2 mg
• Sham

At Month 12, the participants in the avacincaptad pegol 2mg treatment group will be re-randomized to receive the study drug either on a monthly basis or on an every other month basis

The participants initially randomized to sham treatment will continue with monthly sham administration through Month 23

All participants will have a final follow up visit at Month 24

• Study Type: Interventional
• Study Phase: Phase 3

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Masking Description:

Participant, "Evaluating" Investigator, Reading Center Personnel, and Sponsor Personnel are masked

Primary Purpose: Treatment

Condition

• Geographic Atrophy
• Macular Degeneration

Intervention

• Drug: Avacincaptad Pegol
  Avacincaptad Pegol Intravitreal Injection
  Other Names:

  • Zimura
  • IZERVAY
• Drug: Sham
  Sham Administration (includes placement of the blunt opening of an empty, needleless syringe barrel on the conjunctiva in the inferotemporal quadrant of the eyeball to simulate the pressure of an injection)

Study Arms

• Experimental: Avacincaptad Pegol Treatment Group
  Monthly avacincaptad pegol 2mg intravitreal injections through Month 11 (Year 1) NOTE: At Month 12, participants are re-randomized to receive monthly injections or every other month injections (alternating with sham) from Month 12 to Month 23 (Year 2) - Year 2 is not included in the primary analysis.
  Intervention: Drug: Avacincaptad Pegol
• Sham Comparator: Sham Treatment Group
  Monthly sham administration through Month 11 (Year 1) NOTE: At Month 12, participants continue to receive monthly sham administration from Month 12 to Month 23 (Year 2) - Year 2 is not included in the primary analysis.
  Intervention: Drug: Sham

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: July 26, 2021): 448
• Original Estimated Enrollment (submitted: June 15, 2020): 400
• Actual Study Completion Date: August 22, 2023
• Actual Primary Completion Date: July 25, 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Subjects of either gender aged ≥ 50 years
• Diagnosis of Non-foveal GA secondary to dry AMD

Exclusion Criteria:

• Any prior treatment for AMD (dry or wet) or any prior intravitreal treatment for any indication in either eye, except oral supplements of vitamins and minerals
• Any intraocular surgery or thermal laser within 3 months of trial entry.
• Any prior thermal laser in the macular region, regardless of indication
• Any ocular or periocular infection (including blepharitis), or ocular surface inflammation in the past 12 weeks.
• Previous therapeutic radiation in the region of the study eye
• Any sign of diabetic retinopathy in either eye

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 50 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Argentina, Australia, Austria, Belgium, Brazil, Canada, Colombia, Croatia, Czechia, Estonia, France, Germany, Hungary, Israel, Italy, Latvia, Poland, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT04435366
• Other Study ID Numbers: ISEE2008
• 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: Not Provided
• Current Responsible Party: Astellas Pharma Inc ( IVERIC bio, Inc. )
• Original Responsible Party: Same as current
• Current Study Sponsor: IVERIC bio, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Astellas Pharma Inc
• Verification Date: November 2023