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

To evaluate the effect of systemic Complement Component 5 (C5) inhibition on Geographic atrophy (GA) lesion growth over 52 weeks

Detailed Description

Not available

Study Timeline

• Study First Posted: 2025-02-17
• Last Update Posted: 2025-06-25

Recruitment & Eligibility

• Status: Authorised, recruiting
• Sex: Not specified
• Target Recruitment: 239

Inclusion Criteria

Study eye with diagnosis of GA of the macula secondary to AMD not involving the foveal center point as described in the protocol

Total GA area in the study eye measuring between ≥2.5 mm2 and ≤17.5 mm2 as described in the protocol

BCVA of 35 letters or better using ETDRS charts (20/200 Snellen equivalent) in the study eye as described in the protocol

Sufficiently clear ocular media, adequate pupillary dilation and fixation to permit quality fundus imaging in the study eye as described in the protocol

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

Exclusion Criteria

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

Systemic Exclusion criteria: Current or prior use of systemic immunosuppressive therapy other than corticosteroids or the likelihood of treatment with any such agent during the study inclusive of the screening period

Systemic Exclusion criteria: Not meeting meningococcal or pneumococcal vaccination requirements as described in the protocol

Systemic Exclusion criteria: Carrier of Neisseria meningitidis based on culture collected during screening

Systemic Exclusion criteria: Has a hemoglobin A1C ≥ 8.0% during screening as described in the protocol NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply

History or current evidence of macular neovascularization and/or exudation in either eye as described in the protocol

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 as long as last dose was ≥6 months prior to randomization

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

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

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

Systemic Exclusion criteria: History of solid organ or bone marrow transplantation

Systemic Exclusion criteria: 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Growth rate (slope) of total GA lesion area (mm2 /year) measured by Fundus autofluorescence (FAF)		Growth rate (slope) of total GA lesion area (mm2 /year) measured by Fundus autofluorescence (FAF)

Secondary Outcome Measures

Name	Time	Method
Loss of Best corrected visual acuity (BCVA) ≥15 Early treatment diabetic retinopathy study (ETDRS) letters at any 2 consecutive study visits		Loss of Best corrected visual acuity (BCVA) ≥15 Early treatment diabetic retinopathy study (ETDRS) letters at any 2 consecutive study visits
Change in Low-contrast quantitative visual acuity (LC-qVA)		Change in Low-contrast quantitative visual acuity (LC-qVA)
Change in Low-luminance low-contrast quantitative visual acuity (LL-LC-qVA)		Change in Low-luminance low-contrast quantitative visual acuity (LL-LC-qVA)
Change in Quantitative contrast sensitivity function (qCSF)		Change in Quantitative contrast sensitivity function (qCSF)
Growth rate (slope) of total GA lesion area (mm2 /year) measured by FAF		Growth rate (slope) of total GA lesion area (mm2 /year) measured by FAF
Concentrations of total pozelimab in serum		Concentrations of total pozelimab in serum
Concentrations of total cemdisiran in plasma		Concentrations of total cemdisiran in plasma
Change in concentration of total Complement component 5 (C5)		Change in concentration of total Complement component 5 (C5)
Incidence of Antidrug antibody (ADA) to pozelimab		Incidence of Antidrug antibody (ADA) to pozelimab
Titer of ADA to pozelimab		Titer of ADA to pozelimab
Incidence of ADA to cemdisiran		Incidence of ADA to cemdisiran
Titer of ADA to cemdisiran		Titer of ADA to cemdisiran
Incidence of Neutralizing antibody (NAb) to pozelimab		Incidence of Neutralizing antibody (NAb) to pozelimab
Occurrence of Treatment-emergent adverse events (TEAEs)		Occurrence of Treatment-emergent adverse events (TEAEs)
Severity of TEAEs		Severity of TEAEs

Trial Locations

Locations (71)

Hanusch Krankenhaus Der Wiener Gebietskrankenkasse; 🇦🇹 Vienna, Austria

Medical University Of Graz; 🇦🇹 Graz, Austria

Medical University Of Vienna; 🇦🇹 Vienna, Austria

Klinik Hietzing; 🇦🇹 Vienna, Austria

Centre Hospitalier Universitaire De Nice; 🇫🇷 Nice, France

Hopital Saint Joseph; 🇫🇷 Marseille, France

Centre Hospitalier Universitaire De Poitiers; 🇫🇷 Poitiers, France

Hopital Prive La Louviere; 🇫🇷 Lille, France

Hopital De La Croix-Rousse; 🇫🇷 Lyon Cedex 04, France

Clinique Mathilde; 🇫🇷 Rouen, France

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Hanusch Krankenhaus Der Wiener Gebietskrankenkasse

🇦🇹Vienna, Austria

Oliver Findl

Site contact

+4319102184610

oliver@findl.at