Clinical trial NCT06660667: A Safety and Efficacy Study of One-time SAR402663 in Adults With Neovascular Age-related Macular Degeneration
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Neovascular Age-related Macular Degeneration
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2024-10-28
Enrollment: 66
Sex: ALL
Minimum age (years): 50
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Part I is nonrandomized sequential dose escalation and Part II is randomized parallel assignment
Primary purpose: TREATMENT

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 and investigators 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.


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
* Currently receiving anti-VEGF treatment in the study eye
* Demonstrated a response to aflibercept in the pre-treatment period

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
* Use of ocular corticosteroids in the 3 months prior to screening
* 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.

Primary outcomes:
measure: Incidence and severity of ocular treatment emergent adverse event (TEAEs)
timeFrame: Day 1 to Week 52
measure: Incidence and severity of ocular treatment emergent serious adverse event (TESAEs)
timeFrame: Day 1 to Week 52
measure: Incidence and severity of non-ocular TEAEs
timeFrame: Day 1 to Week 52
measure: Incidence and severity of non-ocular TESAEs
timeFrame: Day 1 to Week 52
measure: Number of participants with any clinically significant changes in laboratory variables
timeFrame: Day 1 to Week 52
measure: Number of participants with any clinically significant changes in vital signs
timeFrame: Day 1 to Week 52
Secondary outcomes:
measure: Percentage of participants not requiring supplemental anti- vascular endothelial growth factor (VEGF) therapy
timeFrame: Day 1 to Week 52 and Week 8 to Week 52
measure: Annualized injection rates of anti-VEGF therapy
timeFrame: Day 1 to Week 52
measure: Change from baseline in BCVA using the ETDRS letter score
timeFrame: Baseline, Week 52
measure: Percentage of participants not losing more than or equal to 15 ETDRS letters from baseline
timeFrame: Baseline, Week 52
measure: Change from baseline in central subfield thickness (CST), measured by spectral domain optical coherence tomography (SD-OCT)
timeFrame: Baseline, Week 52
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Site # 8400011
state: Arizona
city: Beverly Hills
country: United States
facility: Site # 8400023
state: California
city: Gainesville
country: United States
facility: Site # 8400004
state: Florida
city: Lemont
country: United States
facility: Site # 8400005
state: Illinois
city: Reno
country: United States
facility: Site # 8400021
state: Nevada
city: Eugene
country: United States
facility: Site # 8400017
state: Oregon
city: Germantown
country: United States
facility: Site # 8400018
state: Tennessee
city: Austin
country: United States
facility: Site # 8400015
state: Texas
city: Dallas
country: United States
facility: Site # 8400006
state: Texas
Study officials:
References: