Clinical trial NCT04857996: Safety, Tolerability and Evidence of Activity Study of UBX1325 in Patients With Diabetic Macular Edema (BEHOLD)
Official title: A Phase 2a, Prospective, Multicenter, Randomized, Double Masked, Sham-Controlled Study to Assess the Safety, Tolerability and Evidence of Activity of a Single Intravitreal Injection of UBX1325 in Patients With Diabetic Macular Edema
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Diabetic Macular Edema (DME)
Keywords: Retinal disease
Macular edema
Diabetes mellitus
Diabetic macular edema
Lead sponsor: Unity Biotechnology, Inc.
Sponsor class: INDUSTRY
First posted: 2021-04-23
Enrollment: 65
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is intended to assess the exposure, safety, biological activity, and durability of UBX1325, a phosphate pro-drug, and its active parent molecule (UBX0601) following a single intravitreal (IVT) injection of UBX1325 in patients with diabetic macular edema (DME).

Detailed description: This is a Phase 2a Proof-of-Concept (POC) study. The study will enroll approximately 62 patients randomized 1:1 into either the UBX1325 or sham study arms, in order to assess safety and tolerability as well as impact on visual function and retinal anatomy. Patients will be followed for 24 weeks for the primary outcome measure and for an additional 24 weeks (48 weeks total) for the secondary outcome measures.

Eligibility criteria: Inclusion Criteria:

* Patients aged ≥ 18 years.
* Diabetic retinopathy patients (nonproliferative (NPDR) and proliferative (PDR)) with DME (ETDRS-DRSS Score at 65C \[or DR severity level of 8\]or less severe as determined by a Central Reading Center), who had at least 2 anti-VEGF intravitreal (IVT) injections (one or more of the following agents: aflibercept, bevacizumab or ranibizumab) in the preceding 6 months prior to Day 1, with the last anti-VEGF given between 3 and 6 weeks prior to Day 1.
* Center-involved DME with central subfield thickness (CST) ≥300 µm on SD-OCT at Screening
* BCVA in the study eye (most affected) of 73 to 20 ETDRS letters (equivalent to 20/40 to 20/400 on the Snellen chart) at Screening and at Day 1.

Exclusion Criteria:

* Concurrent disease in the study eye or structural damage, other than DME, that could compromise BCVA, prevent BCVA improvement, require medical or surgical intervention during the study period, confound interpretation of the results, or interfere with assessment of toxicity or color fundus photography (CFP) in the study eye.
* Any ocular/intraocular/periocular infection or inflammation in either eye in the past 4 weeks prior to screening
* History of vitreous hemorrhage in the study eye within 2 months prior to Screening
* Any condition, including laboratory findings and findings in the medical history or in the pre-study assessments, that, in the opinion of the Investigator, constitutes a risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation or prevent the patient from fully participating in all aspects of the study
* Significant media opacities, including cataract, which might interfere with VA, assessment of toxicity, or fundus imaging

Primary outcomes:
measure: Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
timeFrame: 48 weeks
measure: Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
timeFrame: Up to Week 12, 24 and 48 weeks
Secondary outcomes:
measure: Changes in Best Corrected Visual Acuity (BCVA) From Baseline
timeFrame: Week 12, 24 and 48
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Retinal Research Institute, LLC
state: Arizona
city: Bakersfield
country: United States
facility: California Retina Consultants
state: California
city: Encino
country: United States
facility: The Retina Partners
state: California
city: Huntington Beach
country: United States
facility: Salehi Retina Institute Inc.
state: California
city: Loma Linda
country: United States
facility: Loma Linda University
state: California
city: Oxnard
country: United States
facility: California Retina Consultants
state: California
city: Longmont
country: United States
facility: Advanced Vision Research Institute
state: Colorado
city: Miami
country: United States
facility: Bascom Palmer Eye Institute
state: Florida
city: Miami
country: United States
facility: MedEye Associates
state: Florida
city: Chicago
country: United States
facility: Northwestern Medical Group
state: Illinois
city: Indianapolis
country: United States
facility: MidWest Eye Institute
state: Indiana
city: Hagerstown
country: United States
facility: Cumberland Valley Retina Consultants
state: Maryland
city: Boston
country: United States
facility: Mass Eye and Ear Institute
state: Massachusetts
city: Reno
country: United States
facility: Sierra Eye Associates
state: Nevada
city: New York
country: United States
facility: New York Eye and Ear Infirmary
state: New York
city: Portland
country: United States
facility: EyeHealth Northwest
state: Oregon
city: Greenville
country: United States
facility: Retina Consultants of Carolina, PA
state: South Carolina
city: McAllen
country: United States
facility: Valley Retina Institute, P.A.
state: Texas
city: Southlake
country: United States
facility: Retina Center of Texas
state: Texas
city: Edmonton
country: Canada
facility: Alberta Retina Research Corporation
state: Alberta
city: North York
country: Canada
facility: Toronto Retina Institute
state: Ontario
city: Montréal
country: Canada
facility: Hopital Maisonneuve-Rosemont
state: Quebec
Study officials:
affiliation: Unity Biotechnology, Inc.
name: Sharon Klier, MD, MPH
role: STUDY_DIRECTOR
References: