A Phase 1/2 Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, and Efficacy of One-time Intravitreal Dose of SAR402663 in Participants With Neovascular Age-related Macular Degeneration
Overview
- Phase
- Phase 1
- Intervention
- SAR402663
- Conditions
- Neovascular Age-related Macular Degeneration
- Sponsor
- Sanofi
- Enrollment
- 66
- Locations
- 18
- Primary Endpoint
- Incidence and severity of ocular treatment emergent adverse event (TEAEs)
- Status
- Recruiting
- Last Updated
- 5 months ago
Overview
Brief Summary
This is a Phase 1/Phase 2 multicenter study to evaluate the safety and efficacy of a one-time single-eye intravitreal dose of SAR402663 in participants with neovascular age-related macular degeneration.
Participants will be enrolled in one of 2 parts:
- In Part I (dose escalation), multiple dose levels of SAR402663 will be evaluated in successive cohorts of participants
- In Part II (dose expansion), participants will be randomized to receive one of two dose levels selected based on data from Part I. Participants, investigators and outcomes assessors will be masked to dose.
After receiving one-time dose of SAR402663, participants will undergo regular assessments over 12 months. Following this, participants will enter an extended follow-up (EFU) phase for the assessment of safety and durability of clinical activity of SAR402663 through Year 5.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Between 50 and 90 years of age
- •Participants with diagnosis of macular neovascularization secondary to age-related macular degeneration (nAMD)
- •Study eye with best corrected visual acuity (BCVA) ETDRS Snellen equivalent for dose escalation (Part I) between 20/32 and 20/400 and for expansion (Part II) between 20/25 and 20/200
- •Current or previous use of anti-vascular endothelial growth factor (VEGF) treatment in the study eye
- •Demonstrated a response to anti-VEGF treatment
Exclusion Criteria
- •Any condition in the study eye that may prevent visual acuity improvement or interfere with ocular safety or efficacy assessments
- •History of active ocular infection in the study eye in 6 months prior to screening
- •Active uncontrolled glaucoma in the study eye
- •History of uveitis in either eye
- •Current use of ocular corticosteroids in the study eye
- •Previous gene therapy
- •Any significant poorly controlled illness that would preclude study compliance and follow up
- •The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Arms & Interventions
Part I - SAR402663 open-label (OL)
Participants will receive a single dose of SAR402663 on Day 1. Multiple dose levels of SAR402663 will be evaluated in successive cohorts of participants.
Intervention: SAR402663
Part I - SAR402663 open-label (OL)
Participants will receive a single dose of SAR402663 on Day 1. Multiple dose levels of SAR402663 will be evaluated in successive cohorts of participants.
Intervention: Diluent
Part II - SAR402663 Dose A
Participants will receive a single dose of SAR402663 on Day 1.
Intervention: SAR402663
Part II - SAR402663 Dose A
Participants will receive a single dose of SAR402663 on Day 1.
Intervention: Diluent
Part II - SAR402663 Dose B
Participants will receive a single dose of SAR402663 on Day 1.
Intervention: SAR402663
Part II - SAR402663 Dose B
Participants will receive a single dose of SAR402663 on Day 1.
Intervention: Diluent
Outcomes
Primary Outcomes
Incidence and severity of ocular treatment emergent adverse event (TEAEs)
Time Frame: Day 1 to Week 52
Incidence and severity of ocular treatment emergent serious adverse event (TESAEs)
Time Frame: Day 1 to Week 52
Incidence and severity of non-ocular TEAEs
Time Frame: Day 1 to Week 52
Incidence and severity of non-ocular TESAEs
Time Frame: Day 1 to Week 52
Number of participants with any clinically significant changes in laboratory variables
Time Frame: Day 1 to Week 52
Number of participants with any clinically significant changes in vital signs
Time Frame: Day 1 to Week 52
Secondary Outcomes
- Percentage of participants not requiring supplemental anti-vascular endothelial growth factor (VEGF) therapy(Day 1 to Week 52 and Week 8 to Week 52)
- Annualized injection rates of anti-VEGF therapy(Day 1 to Week 52)
- Change from baseline in BCVA using the ETDRS letter score(Baseline, Week 52)
- Percentage of participants not losing more than or equal to 15 ETDRS letters from baseline(Baseline, Week 52)
- Change from baseline in central subfield thickness (CST), measured by spectral domain optical coherence tomography (SD-OCT)(Baseline, Week 52)