Safety, Tolerability and Evidence of Activity Study of UBX1325 in Patients With Diabetic Macular Edema (BEHOLD)

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

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).

Study Overview

• Status: Completed
• Conditions: Diabetic Macular Edema (DME)
• Intervention / Treatment: Drug: UBX1325; Other: Sham

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.

• Study Type: Interventional
• Enrollment (Actual): 65
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T5H OX5
      • Alberta Retina Research Corporation
  • Ontario
    • North York, Ontario, Canada, M3C 0G9
      • Toronto Retina Institute
  • Quebec
    • Montréal, Quebec, Canada, H1T 2M4
      • Hopital Maisonneuve-Rosemont
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85053
      • Retinal Research Institute, LLC
  • California
    • Bakersfield, California, United States, 93309
      • California Retina Consultants
    • Encino, California, United States, 91436
      • The Retina Partners
    • Huntington Beach, California, United States, 92647
      • Salehi Retina Institute Inc.
    • Loma Linda, California, United States, 92354
      • Loma Linda University
    • Oxnard, California, United States, 93036
      • California Retina Consultants
  • Colorado
    • Longmont, Colorado, United States, 80503
      • Advanced Vision Research Institute
  • Florida
    • Miami, Florida, United States, 33136
      • Bascom Palmer Eye Institute
    • Miami, Florida, United States, 33143
      • MedEye Associates
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern Medical Group
  • Indiana
    • Indianapolis, Indiana, United States, 46290
      • Midwest Eye Institute
  • Maryland
    • Hagerstown, Maryland, United States, 21740
      • Cumberland Valley Retina Consultants
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Mass Eye and Ear Institute
  • Nevada
    • Reno, Nevada, United States, 89502
      • Sierra Eye Associates
  • New York
    • New York, New York, United States, 10003
      • New York Eye and Ear Infirmary
  • Oregon
    • Portland, Oregon, United States, 97225
      • EyeHealth Northwest
  • South Carolina
    • Greenville, South Carolina, United States, 29605
      • Retina Consultants of Carolina, PA
  • Texas
    • McAllen, Texas, United States, 78503
      • Valley Retina Institute, P.A.
    • Southlake, Texas, United States, 76092
      • Retina Center of Texas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Sham Comparator: Sham Control	Other: Sham; Sham procedure
Experimental: UBX1325	Drug: UBX1325; Patients will be administered a single 50 μL UBX1325 IVT injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.	The systemic safety and tolerability is evaluated by incidence of Treatment Emergent Adverse Events (TEAEs) by System organ class Preferred Term.	48 weeks
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325	Ocular Safety is evaluated by incidence of Treatment Emergent Adverse Events (TEAEs). Ocular TEAEs consisted of progression of disease or were related to the IVT injection component of the UBX1325 drug administration process	Up to Week 12, 24 and 48 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in Best Corrected Visual Acuity (BCVA) From Baseline	Best Corrected Visual Acuity was assessed using the ETDRS chart starting at 4 meters	Week 12, 24 and 48

Collaborators and Investigators

• Sponsor: Unity Biotechnology, Inc.
• Investigators: Study Director: Sharon Klier, MD, MPH, Unity Biotechnology, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 25, 2021
• Primary Completion (Actual): April 6, 2023
• Study Completion (Actual): April 6, 2023

Study Registration Dates

• First Submitted: April 21, 2021
• First Submitted That Met QC Criteria: April 21, 2021
• First Posted (Actual): April 23, 2021

Study Record Updates

• Last Update Posted (Actual): May 16, 2024
• Last Update Submitted That Met QC Criteria: April 18, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Diabetes mellitus; Diabetic macular edema; Macular edema; Retinal disease
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration; Macular Edema; Edema
• Other Study ID Numbers: UBX1325-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No