A study Investigating Subcutaneously Administered Pozelimab in Combination with Cemdisiran or Cemdisiran Alone in Adult Participants with Geographic Atrophy

Phase 3

Recruiting

• Conditions: Geographic Atrophy Secondary to Age-Related Macular Degeneration

• Registration Number: 2023-509547-27-00
• Lead Sponsor: Regeneron Pharmaceuticals Inc.

Brief Summary

This study is researching experimental (study) drugs called pozelimab and cemdisiran. The study is focused on participants who have Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD). Geographic atrophy is a medical term that refers to later-stage cases of AMD which is an eye condition affecting central vision (what one sees straight ahead).

The purpose of this study is to evaluate the progression rate of Geographic Atrophy in eyes of patients treated with cemdisiran alone or in combination with pozelimab compared to those treated with placebo.

The study is looking at several other research questions, including:

* What side effects may happen from taking the study drug(s)

* How much study drug(s) are in the blood at different times

* Whether the body makes antibodies against the study drug(s) (which could make the study drug(s) less effective or could lead to side effects)

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-08-02
• Study First Submitted QC: 2024-08-02
• Study First Posted: 2024-08-07
• Study Start: 2024-10-30
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-26
• Last Update Posted: 2025-08-27
• Primary Completion: 2027-11-29
• Study Completion: 2032-05-21

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 975

Inclusion Criteria

1. Study eye with diagnosis of GA of the macula secondary to AMD as described in the protocol
2. Total GA area in the study eye measuring between ≥2.5 mm^2 and ≤17.5 mm^2 as described in the protocol
3. BCVA of 55 letters or better using ETDRS charts (20/80 Snellen equivalent) in the study eye as described in the protocol
4. Sufficiently clear ocular media, adequate pupillary dilation and fixation to permit quality fundus imaging in the study eye as described in the protocol
5. Willing and able to comply with clinic visits and study-related procedures, including completion of the full series of meningococcal vaccinations and pneumococcal vaccination required per protocol

Key

Exclusion Criteria

1. GA in either eye due to causes other than AMD, such as Stargardt disease, cone rod dystrophy or toxic maculopathies like hydroxychloroquine maculopathy

2. History or current evidence of Macular Neovascularization (MNV) and/or exudation or Peripapillary Choroidal Neovascularization (PPCNV) in either eye as described in the protocol

3. Prior or current Intravitreal (IVT) treatment of any kind for any indication in study eye or fellow eye, except approved or investigational IVT complement inhibitor therapy or anti-VEGF therapy, as long as last dose was ≥6 months prior to randomization

4. Prior intraocular surgery except cataract extraction or minimally invasive glaucoma surgery in study eye as long as date of these procedures was ≥3 months prior to randomization

5. Comorbid progressive ocular condition (eg, diabetic retinopathy, macular edema, uncontrolled glaucoma, full thickness macular hole) in study eye that could affect central vision and confound study

6. Any ophthalmologic condition that reduces the clarity of the media and that, in the opinion of the investigator interferes with ophthalmologic examination of the study eye (e.g., advanced cataract or corneal abnormalities) as described in the protocol

   Systemic Exclusion criteria

7. History or current use of systemic complement inhibitor therapy within 6 months prior to randomization as described in the protocol

8. History of solid organ or bone marrow transplantation

9. Use of chronic (>14 days) systemic corticosteroids (oral or parenteral, ≥20 mg oral prednisone or equivalent) within the previous 30 days prior to the first screening visit as described in the protocol

10. Current or prior use of systemic immunosuppressive therapy other than corticosteroids within 12 months prior to randomization or the likelihood of treatment with any such agent during the study inclusive of the screening period as described in the protocol

11. Not meeting meningococcal or pneumococcal vaccination requirements as described in the protocol

12. Carrier of Neisseria meningitidis based on culture collected during screening

13. Has a hemoglobin A1C ≥ 8.0% during screening as described in the protocol

NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Growth rate (slope) of total GA lesion area (mm^2 /year) from baseline, measured by Fundus Autofluorescence (FAF)	To week 52

Secondary Outcome Measures

Name	Time	Method
Concentrations of total pozelimab in serum	Through week 52 and through week 104
Magnitude of ADA to pozelimab	Through week 52 and through week 104
Occurrence of Treatment-Emergent Adverse Events (TEAEs)	Through week 52, 104, 140 and week 296
Change from baseline in concentration of total Complement component 5 (C5)	Through week 52 and through week 104
Incidence of ADA to cemdisiran	Through week 52 and through week 104
Magnitude of ADA to cemdisiran	Through week 52 and through week 104
Loss of Best Corrected Visual Acuity (BCVA) ≥15 letters [Early Treatment Diabetic Retinopathy Study (ETDRS)] from baseline	At week 52 and week 104
Change from baseline in Low-Luminance Low-Contrast quantitative Visual Acuity (LL-LC-qVA)	At week 52 and week 104
Incidence of Neutralizing Antibody (NAb) to pozelimab	Through week 52 and through week 104
Severity of TEAEs	Through week 52, 104, 140 and week 296
Change from baseline in Low-Contrast quantitative Visual Acuity (LC-qVA)	At week 52 and week 104
Change from baseline in quantitative Contrast Sensitivity Function (qCSF)	At week 52 and week 104
Growth rate (slope) of total GA lesion area (mm^2 /year) from baseline measured by FAF	To week 104
Concentrations of total cemdisiran in plasma	Through week 52 and through week 104
Incidence of Antidrug antibody (ADA) to pozelimab	Through week 52 and through week 104

Trial Locations

Locations (94)

Barnet Dulaney Perkins Eye Center; 🇺🇸 Phoenix, Arizona, United States

Associated Retina Consultants; 🇺🇸 Phoenix, Arizona, United States

Retinal Research Institute; 🇺🇸 Phoenix, Arizona, United States

Retina Associates of Tuscan; 🇺🇸 Tucson, Arizona, United States

Retina Vitreous Associates Medical Group; 🇺🇸 Beverly Hills, California, United States

The Retina Partners; 🇺🇸 Encino, California, United States

Retina Consultants of Orange County; 🇺🇸 Fullerton, California, United States

Salehi Retina Institute dba Retina Associates of Southern California; 🇺🇸 Huntington Beach, California, United States

South Coast Retina Center; 🇺🇸 Long Beach, California, United States

American Institute of Research; 🇺🇸 Los Angeles, California, United States

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