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

NCT ID: NCT04857996

Last Updated: 2024-05-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 65 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-06-25
• Study Completion Date: 2023-04-06

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.

Conditions

Diabetic Macular Edema (DME)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

UBX1325

Group Type: EXPERIMENTAL

UBX1325

Intervention Type: DRUG

Patients will be administered a single 50 μL UBX1325 IVT injection

Sham Control

Group Type: SHAM_COMPARATOR

Sham

Intervention Type: OTHER

Sham procedure

Interventions

UBX1325

Patients will be administered a single 50 μL UBX1325 IVT injection

Intervention Type: DRUG

Sham

Sham procedure

Intervention Type: OTHER

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Unity Biotechnology, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sharon Klier, MD, MPH

Role: STUDY_DIRECTOR

Unity Biotechnology, Inc.

Locations

Retinal Research Institute, LLC

Phoenix, Arizona, United States

California Retina Consultants

Bakersfield, California, United States

The Retina Partners

Encino, California, United States

Salehi Retina Institute Inc.

Huntington Beach, California, United States

Loma Linda University

Loma Linda, California, United States

California Retina Consultants

Oxnard, California, United States

Advanced Vision Research Institute

Longmont, Colorado, United States

Bascom Palmer Eye Institute

Miami, Florida, United States

MedEye Associates

Miami, Florida, United States

Northwestern Medical Group

Chicago, Illinois, United States

MidWest Eye Institute

Indianapolis, Indiana, United States

Cumberland Valley Retina Consultants

Hagerstown, Maryland, United States

Mass Eye and Ear Institute

Boston, Massachusetts, United States

Sierra Eye Associates

Reno, Nevada, United States

New York Eye and Ear Infirmary

New York, New York, United States

EyeHealth Northwest

Portland, Oregon, United States

Retina Consultants of Carolina, PA

Greenville, South Carolina, United States

Valley Retina Institute, P.A.

McAllen, Texas, United States

Retina Center of Texas

Southlake, Texas, United States

Alberta Retina Research Corporation

Edmonton, Alberta, Canada

Toronto Retina Institute

North York, Ontario, Canada

Hopital Maisonneuve-Rosemont

Montreal, Quebec, Canada

Countries

United States; Canada

References

Klier S, Dananberg J, Masaki L, Bhisitkul RB, Khanani AM, Maturi R, Salehi-Had H, Mallinckrodt CH, Rathmell JM, Ghosh A, Sapieha P. Safety and Efficacy of Senolytic UBX1325 in Diabetic Macular Edema. NEJM Evid. 2025 May;4(5):EVIDoa2400009. doi: 10.1056/EVIDoa2400009. Epub 2025 Apr 22.

Reference Type: DERIVED

PMID: 40261111 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

UBX1325-02

Identifier Type: -

Identifier Source: org_study_id