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

Phase 2

Completed

• Conditions: Diabetic Macular Edema (DME)
• Interventions: Other: Sham; Drug: UBX1325

• Registration Number: NCT04857996
• Lead Sponsor: Unity Biotechnology, Inc.

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.

Study Timeline

• Study First Submitted: 2021-04-21
• Study First Submitted QC: 2021-04-21
• Study First Posted: 2021-04-23
• Study Start: 2021-06-25
• Primary Completion: 2023-04-06
• Study Completion: 2023-04-06
• Results First Submitted: 2024-02-06
• Status Verified: 2024-04
• Results First Submitted QC: 2024-04-18
• Last Update Submitted: 2024-04-18
• Results First Posted: 2024-05-16
• Last Update Posted: 2024-05-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 65

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sham Control	Sham	-
UBX1325	UBX1325	-

Primary Outcome Measures

Name	Time	Method
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.	48 weeks	The systemic safety and tolerability is evaluated by incidence of Treatment Emergent Adverse Events (TEAEs) by System organ class Preferred Term.
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325	Up to Week 12, 24 and 48 weeks	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

Secondary Outcome Measures

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

Trial Locations

Locations (21)

Loma Linda University; 🇺🇸 Loma Linda, California, United States

The Retina Partners; 🇺🇸 Encino, California, United States

Salehi Retina Institute Inc.; 🇺🇸 Huntington Beach, California, United States

Northwestern Medical Group; 🇺🇸 Chicago, Illinois, United States

Cumberland Valley Retina Consultants; 🇺🇸 Hagerstown, Maryland, United States

Sierra Eye Associates; 🇺🇸 Reno, Nevada, United States

Retina Consultants of Carolina, PA; 🇺🇸 Greenville, South Carolina, United States

Valley Retina Institute, P.A.; 🇺🇸 McAllen, Texas, United States

Hopital Maisonneuve-Rosemont; 🇨🇦 Montréal, Quebec, Canada

Bascom Palmer Eye Institute; 🇺🇸 Miami, Florida, United States

MedEye Associates; 🇺🇸 Miami, Florida, United States

MidWest Eye Institute; 🇺🇸 Indianapolis, Indiana, United States

Mass Eye and Ear Institute; 🇺🇸 Boston, Massachusetts, United States

Advanced Vision Research Institute; 🇺🇸 Longmont, Colorado, United States

California Retina Consultants; 🇺🇸 Oxnard, California, United States

New York Eye and Ear Infirmary; 🇺🇸 New York, New York, United States

Retina Center of Texas; 🇺🇸 Southlake, Texas, United States

Retinal Research Institute, LLC; 🇺🇸 Phoenix, Arizona, United States

Toronto Retina Institute; 🇨🇦 North York, Ontario, Canada

EyeHealth Northwest; 🇺🇸 Portland, Oregon, United States

Alberta Retina Research Corporation; 🇨🇦 Edmonton, Alberta, Canada