A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy

• ClinicalTrials.gov Identifier: NCT04656561
• Recruitment Status: Not yet recruiting
• First Posted: December 7, 2020
• Last Update Posted: February 26, 2021
• Sponsor: Annexon, Inc.
• Information provided by (Responsible Party): Annexon, Inc.

Study Description

Brief Summary:

This study is being conducted in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) to determine if intravitreal (IVT) injections of ANX007 reduce GA lesion growth rate. The results will be used to guide further development of ANX007 in participants with geographic atrophy. The total duration of participation is expected to be approximately 19 months.

Condition or disease	Intervention/treatment	Phase
Geographic Atrophy	Drug: ANX007; Other: Sham comparator	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 240 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Multicenter, Randomized, Parallel-Group, Double-Masked, 4-Arm, Sham-Controlled Study of the Efficacy, Safety, and Tolerability of ANX007 Administered by Intravitreal Injection in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
• Estimated Study Start Date: March 2021
• Estimated Primary Completion Date: June 2023
• Estimated Study Completion Date: December 2023

Arms and Interventions

Arm	Intervention/treatment
Experimental: ANX007 Group 1; ANX007 administered every month	Drug: ANX007; Form: solution for injection; Route of Administration: IVT
Experimental: ANX007 Group 2; ANX007 administered every other month	Drug: ANX007; Form: solution for injection; Route of Administration: IVT
Sham Comparator: Sham Group 3; Sham injection administered every month	Other: Sham comparator; Form and Route of Administration: pressure to mimic IVT injection
Sham Comparator: Sham Group 4; Sham injection administered every other month	Other: Sham comparator; Form and Route of Administration: pressure to mimic IVT injection

Outcome Measures

Primary Outcome Measures :

1. GA lesion growth rate [ Time Frame: Baseline to Month 12 ]
   Change in GA lesion area as assessed by fundus autofluorescence (FAF)

Secondary Outcome Measures :

1. Safety: Participants with treatment-emergent adverse events (TEAEs) [ Time Frame: Baseline to Month 18 ]
   Number of participants with TEAEs
2. Best corrected visual acuity (BCVA) [ Time Frame: Baseline to Month 18 ]
   Change from baseline in BCVA as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) test chart
3. Low-luminance BCVA (LL-BCVA) [ Time Frame: Baseline to Month 18 ]
   Change from baseline in LL-BCVA
4. Low-luminance visual acuity deficit (LL-VD) [ Time Frame: Baseline to Month 18 ]
   Change from baseline in LL-VD

Eligibility Criteria

• Ages Eligible for Study: 50 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Diagnosis of geographic atrophy of the macula secondary to age-related macular degeneration as determined by the Investigator and confirmed by the Central Reading Center.

• GA lesion must have the following characteristics as determined by the independent Central Reading Center based on assessment of FAF imaging at screening:

  1. Well-demarcated GA with a total area (baseline lesion size) ≥2.5 mm2 and ≤17.5 mm2.
  2. If GA is multifocal, at least 1 focal lesion must measure ≥1.25 mm2 with the overall aggregate area of GA as specified above.
  3. Presence of hyper autofluorescence, any pattern, in the junctional zone of the GA. Absence of hyper autofluorescence (ie, pattern = none) is exclusionary.
  4. 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 peripapillary atrophy.
• Normal luminance best corrected visual acuity (BCVA) of 24 to 83 letters, inclusive, using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (20/25 to 20/320 Snellen equivalent, inclusive).
• A female patient is eligible if she is not pregnant or breastfeeding and is a woman of non-childbearing potential or is using a contraceptive method that is highly effective, with a failure rate of <1% during the study intervention period and for at least 30 days after the last dose of study intervention.

Exclusion Criteria:

• Geographic atrophy due to other causes than AMD such as Stargardt disease, cone-rod dystrophy, pathologic myopia, or toxic maculopathies (eg, plaquenil maculopathy) in either eye.

• Any evidence of choroidal neovascularization (CNV) in the study eye:

  1. Any history of CNV of any cause based on medical history.
  2. Evidence of prior or active CNV or related findings (eg, retinal pigment epithelial rips or tears) based on FAF, SD-OCT imaging, intravenous fluorescein angiography (IVFA) and color fundus photo as assessed by the Central Reading Center.
• Spherical equivalent of -8.00 diopters (D) myopia or higher in the study eye.
• Uncontrolled glaucoma in the study eye (IOP >25 mmHg despite treatment with anti- glaucoma medication) or history of neovascular glaucoma.
• History of glaucoma filtration surgery, minimally-invasive glaucoma surgery involving an implant, or vitrectomy surgery, or other procedure in the study eye that could affect drug distribution and/or clearance.
• Any current or prior ocular disease, other than geographic atrophy, that in the opinion of the Investigator could interfere with the conduct of the study including, but not limited to, insufficient pupil dilation, retinal or optic nerve disease, media opacity, or aphakia in the study eye.
• History of any prior IVT treatment for any indication in the study eye.
• Any prior treatment for AMD in the study eye (eg, surgical, radiation, thermotherapeutic, or laser intervention), except oral supplements or minerals.

Contacts and Locations

Contacts

Contact: Study Coordinator; 650-822-5500; clinicaltrials@annexonbio.com

Sponsors and Collaborators

Annexon, Inc.

Investigators

• Study Director: Clinical Trials; Annexon, Inc.

More Information

• Responsible Party: Annexon, Inc.
• ClinicalTrials.gov Identifier: NCT04656561
• Other Study ID Numbers: ANX007-GA-01
• First Posted: December 7, 2020
• Last Update Posted: February 26, 2021
• Last Verified: February 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Geographic Atrophy; Atrophy; Pathological Conditions, Anatomical; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases