A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Participants with Dry Age-Related Macular Degeneration with Geographic Atrophy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Annexon Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

3 Mar 2025 → ongoing

Decision date (initial)

2025-02-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Annexon, Inc.

External identifiers

EU CT number

2024-515022-88-00

ClinicalTrials.gov

NCT06510816

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Others, Safety

To evaluate efficacy of intravitreal (IVT) injections of ANX007 on visual function and visual acuity

Secondary objectives 5

1. 1. To evaluate efficacy of IVT injections of ANX007 on secondary measures of visual function and visual acuity
2. 2. To compare the change in total loss of the ellipsoid zone (EZ) layer with IVT injections of ANX007 vs Sham control
3. 3. To compare the change in [CCI] IVT injections of ANX007 vs Sham control
4. 4. To assess the safety and tolerability of IVT injections of ANX007
5. 5. To assess patient reported outcomes

Conditions and MedDRA coding

Dry Age-Related Macular Degeneration (AMD) with geographic atrophy (GA)

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

Swedish Medical Products Agency, Spanish Agency For Medicines And Health Products, European Medicines Agency

Plan to share IPD

No

IPD plan description

IPD sharing plan not yet established. The CTIS IPD information will be updated at the later stage after IPD sharing plan is established.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. Fellow eyes treated with an approved anti-complement drug or other locally-administered treatment for GA that was initiated at least 4 weeks prior to randomization may continue treatment with no washout. Fellow eyes treated with an approved therapy for GA for fewer than 4 weeks should not be randomized in the study. GA -treatment-naive fellow eyes should, in the opinion of the Investigator, be unlikely to need treatment for GA in the fellow eye for at least the first year of the study.
2. 2. Participant must be at least 50 years of age at the time of signing the informed consent.
3. 3. Able and willing to participate in a 24-month study with monthly visits.
4. 4. Diagnosis of dry AMD with GA as determined by the Investigator and confirmed by the independent Central Reading Center.
5. 5. The GA lesion must have the following characteristics as determined by the independent Central Reading Center based on assessment of FAF imaging at screening. If both eyes are confirmed to be eligible by the independent Central Reading Center, the determination of the study eye selection is in the opinion of the Investigator. a. Well-demarcated GA with a total area (baseline lesion size) ≥2.5 mm2 and ≤ 17.5 mm2. b. If GA is multifocal, at least one focal lesion must measure ≥1.25 mm2 with the overall aggregate area of GA as specified above in 5a. c. Presence of hyper autofluorescence, any pattern, in the junctional zone of the GA. Absence of hyper autofluorescence (ie., pattern = none) is exclusionary. d. 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.
6. 6. Normal luminance BCVA of 45 to 83 letters using ETDRS methodology(20/25 to 20/100 Snellen equivalent, inclusive). a. [CCI]
7. 7. A female participant is eligible if she is not pregnant or breastfeeding, and one of the following conditions applies: o Is a woman of non-childbearing potential (WONCBP) (defined as having undergone surgical sterilization or being postmenopausal [ie, greater than 50 years old with amenorrhea for at least 12 months without an alternative medical cause]). OR o Is a WOCBP using an acceptable contraceptive method during the study intervention period and for at least 30 days after the last dose of study intervention. • WOCBP must have a negative pregnancy test (PT) within 24 hours before the first dose of study intervention. • The Investigator has reviewed medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.
8. 8. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the Informed Consent Form (ICF) and in this protocol.

Exclusion criteria 18

1. 1. 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.
2. 2. Any evidence of CNV in the study eye: a. Any history of CNV of any cause based on medical history. b. 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.
3. 3. Spherical equivalent of -8.00 diopters (D) myopia or higher in the study eye.
4. 4. Uncontrolled glaucoma in the study eye (Intraocular pressure (IOP) >25 mmHg despite treatment with anti-glaucoma medication) or history of neovascular glaucoma.
5. 5. History of incisional glaucoma surgery (eg, glaucoma filtration surgery, minimally invasive glaucoma surgery implantation of a drainage device); vitrectomy surgery; or other procedure in the study eye that could affect drug distribution and/or clearance.
6. 6. History of cataract surgery less than 3 months prior to dosing in the study eye (cataract surgery should have been uncomplicated to be considered eligible).
7. 7. Any ophthalmic condition that may require surgery during the study period in the study eye
8. 8. Ocular trauma in the study eye within the preceding 6 months.
9. 9. Any active ocular/intraocular infection or inflammation in either eye (e.g., blepharitis, infectious conjunctivitis, keratitis, scleritis, endophthalmitis, uveitis).
10. 10. History of idiopathic, autoimmune-associated, or other uveitis in either eye."
11. 11. Any current or prior ocular condition, other than dry AMD with GA, 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).
12. 12. History of any prior IVT treatment for any indication in the study eye.
13. 13. Any prior treatment for AMD in the study eye ( including investigational treatments, eg, pharmacological, surgical, radiation, thermotherapeutic, light therapy, or laser intervention), or systemic anti-GA or anti-complement treatment (including investigational treatments). NOTE: Oral supplements (multivitamins/minerals, eg, AREDS vitamins) are permitted during the study.
14. 14. Previous participation in any studies of investigational medications not already prohibited by Exclusions 12 and 13, within 3 months or 5 half-lives of the active ingredient (whichever is longer) prior to the start of study treatment.
15. 15. Known hypersensitivity to ANX007 or any of the excipients in the ANX007 solution (as described in the ANX007 Investigator’s Brochure or other ANX molecules (eg ANX005).
16. 16. Known hypersensitivity to fluorescein
17. 17. Active alcohol or substance abuse/dependence or any other reason that makes it unlikely that the participant will comply with study procedures.
18. 18. History of current systemic medical or psychiatric conditions or any other reason, including laboratory findings, that may, in the opinion of the Investigator, contraindicate the use of an investigational medication, affect interpretation of study results, preclude adherence to the study visit schedule, or safe participation in the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Confirmed best corrected visual acuity (BCVA) ≥15-letter loss from baseline through Month 15 as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) method.

Secondary endpoints 1

1. Incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESI)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Annexon Inc.

Sponsor organisation

Annexon Inc.

Address

1400 Sierra Point Parkway

City

Brisbane

Postcode

94005-1808

Country

United States

Scientific contact point

Organisation

Annexon Inc.

Contact name

Jamie Dananberg, MD.

Public contact point

Organisation

Annexon Inc.

Contact name

AJ Acker

Third parties 11

Locations

9 EU/EEA countries · 53 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 94 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications