A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy

Phase
Recruitment status
Participants
Lead sponsor
Regeneron Pharmaceuticals
Start date
Number of sites

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)
Acronym
Short title
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Official title
A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Control arm
Registry reports results
Listing at source
Recruitment status
Phase
Study type
Interventional

Completion date
First posted
Registry last updated
Primary completion date
Start date

Participants
Participant count basis
Planned participants
Protocol amendments
Number of sites

Data monitoring committee
Registry sponsor category
Lead sponsor
Regeneron Pharmaceuticals
Source registry
ClinicalTrials.gov

Registry ID
ClinicalTrials.gov ID
Registry record
Open registry record

Allocation
Randomised
Model
Parallel groups
Purpose
Treatment
Masking
Quadruple masking
Masked roles
Participants, Care providers, Investigators, Outcome assessors
Sources for study design

  1. Growth rate (slope) of total GA lesion area (mm^2 /year) from baseline, measured by Fundus Autofluorescence (FAF)

    Timeframe: To week 52

  1. Loss of Best Corrected Visual Acuity (BCVA) ≥15 letters [Early Treatment Diabetic Retinopathy Study (ETDRS)] from baseline

    Timeframe: At week 52 and week 104

  2. Change from baseline in Low-Contrast quantitative Visual Acuity (LC-qVA)

    Timeframe: At week 52 and week 104

  3. Change from baseline in Low-Luminance Low-Contrast quantitative Visual Acuity (LL-LC-qVA)

    Timeframe: At week 52 and week 104

  4. Change from baseline in quantitative Contrast Sensitivity Function (qCSF)

    Timeframe: At week 52 and week 104

  5. Growth rate (slope) of total GA lesion area (mm^2 /year) from baseline measured by FAF

    Timeframe: To week 104

  6. Concentrations of total pozelimab in serum

    Timeframe: Through week 52 and through week 104

  7. Concentrations of total cemdisiran in plasma

    Timeframe: Through week 52 and through week 104

  8. Change from baseline in concentration of total Complement component 5 (C5)

    Timeframe: Through week 52 and through week 104

  9. Incidence of Antidrug antibody (ADA) to pozelimab

    Timeframe: Through week 52 and through week 104

  10. Magnitude of ADA to pozelimab

    Timeframe: Through week 52 and through week 104

  11. Incidence of ADA to cemdisiran

    Timeframe: Through week 52 and through week 104

  12. Magnitude of ADA to cemdisiran

    Timeframe: Through week 52 and through week 104

  13. Incidence of Neutralizing Antibody (NAb) to pozelimab

    Timeframe: Through week 52 and through week 104

  14. Occurrence of Treatment-Emergent Adverse Events (TEAEs)

    Timeframe: Through week 52, 104, 140 and week 296

  15. Severity of TEAEs

    Timeframe: Through week 52, 104, 140 and week 296

Sources for outcomes

Sex
All
Age groups
Adults, Older adults
Minimum age
50 Years
Maximum age
85 Years
Healthy volunteers
No

Key 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

Sources for eligibility

  • Barnet Dulaney Perkins Eye Center-OphthamalogyChandler, United StatesRecruiting
  • Barnet Dulaney Perkins Eye CenterPhoenix, United StatesRecruiting
  • Associated Retina ConsultantsPhoenix, United StatesRecruiting
  • Retinal Research InstitutePhoenix, United StatesRecruiting
  • Retina Macula Institute of ArizonaScottsdale, United StatesRecruiting
  • Retina Associates of TuscanTucson, United StatesRecruiting
  • Retina Partners of Northwest Arkansas, PLLCSpringdale, United StatesRecruiting
  • Retina Vitreous Associates Medical GroupBeverly Hills, United StatesRecruiting
  • Retina Specialists of Beverly Hills- Department of OphthalmologyBeverly Hills, United StatesRecruiting
  • The Retina PartnersEncino, United StatesCompleted
  • Eye Medical Center of FresnoFresno, United StatesRecruiting
  • Retina Consultants of Orange CountyFullerton, United StatesCompleted
  • Salehi Retina Institute dba Retina Associates of Southern CaliforniaHuntington Beach, United StatesRecruiting
  • University of California IrvineIrvine, United StatesRecruiting
  • University of California San Diego, Jacobs Retina CenterLa Jolla, United StatesRecruiting
  • South Coast Retina CenterLong Beach, United StatesRecruiting
  • American Institute of ResearchLos Angeles, United StatesCompleted
  • Kaiser Permanente Los Angeles Medical CenterLos Angeles, United StatesRecruiting
  • Jules Stein Eye Institute in David Geffen School of Medicine at UCLALos Angeles, United StatesRecruiting
  • Retinal Consultants Medical Group Inc ModestoModesto, United StatesRecruiting
  • Northern California Retina Vitreous Associates Medical Group, Inc.Mountain View, United StatesRecruiting
  • California Retina ConsultantsOxnard, United StatesRecruiting
  • Desert Retina ConsultantsPalm Desert, United StatesWithdrawn
  • California Eye Specialists Medical Group Inc.Pasadena, United StatesRecruiting
  • Retina Consultants San DiegoPoway, United StatesRecruiting

And 206 more, listed on the interactive page.

Sources for sites

  • 2023
  • 2023-509547-27-00
Sources for references

Related records

Drugs (2)

Lead sponsors (1)

Conditions (2)

Targets (1)