Safety and Tolerability Study of UBX1325 in Patients With Diabetic Macular Edema or Neovascular Age-Related Macular Degeneration

A Phase 1, Open-Label, Single Ascending Dose Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of UBX1325 in Patients With Diabetic Macular Edema or Neovascular Age-Related Macular Degeneration

A study to evaluate safety, tolerability, and pharmacokinetics of a single intravitreal injection of UBX1325 in patients diagnosed with diabetic macular edema (DME) or neovascular Age-Related Macular Degeneration.

Study Overview

• Status: Completed
• Conditions: Diabetic Macular Edema; Neovascular Age-related Macular Degeneration
• Intervention / Treatment: Drug: UBX1325

Detailed Description

This is a Phase 1, open-label, first-in-human (FIH), single-ascending dose (SAD) study consisting of approximately 4 cohorts. The total number of patients will be at least 3 per cohort plus 3 additional patients in the maximum tolerated dose (MTD) cohort in order to address the primary objective. Up to an additional 6 nAMD patients will be enrolled in the highest dose cohort. A single dose of UBX1325 will be administered intravitreally and all patients will be followed for approximately 6 months.

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Beverly Hills, California, United States, 90211
      • Retina-Vitreous Associates Medical Group
    • Huntington Beach, California, United States, 92647
      • Salehi Retina Institute
  • Florida
    • Miami, Florida, United States, 33143
      • MedEye Associates
    • Sarasota, Florida, United States, 34239
      • Sarasota Retina Institute
    • Winter Haven, Florida, United States, 33880
      • Center for Retina and Macular Disease
  • Indiana
    • Indianapolis, Indiana, United States, 46290
      • Midwest Eye Institute
  • Texas
    • Houston, Texas, United States, 77025
      • Houston Eye Associates
    • San Antonio, Texas, United States, 78240
      • Medical Center Ophthalmology Associates

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 48 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• nAMD patients who, in the opinion of the Investigator, are inadequately responding to or failed current treatment options.
• Active choroidal neovascularization (CNV) associated with age-related macular degeneration as evidenced on FA and SD-OCT at Day 1, including presence of intraretinal or subretinal fluid.
• BCVA in the study eye (most affected) of 70 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or worse at screening and on Day 1.
• Patients who have the capacity to give informed consent and who are willing and able to comply with all study-related procedures and assessments.

Exclusion Criteria:

• Any ocular/intraocular/periocular infection or inflammation in either eye in the past 4 weeks prior to screening.
• Subretinal hemorrhage with bleeding area ≥4 disc area in the study eye
• Concomitant therapy with anti-VEGF therapies (e.g., Avastin®, Lucentis®, or Eylea®) or previous use of these agents in the study eye within approximately 28 days of study enrollment
• Any retinovascular disease or retinal degeneration other than nAMD in the study eye.
• History of systemic and intraocular steroid use for 6 months prior to Day 1. The use of intravitreal nonbiodegradable steroid implants (ex. Iluvien®, Yutiq®, Retisert®) is prohibited.
• Significant media opacities, including cataract, which might interfere with VA, assessment of toxicity, or fundus imaging.
• Any uncontrolled medical condition that, in the opinion of the investigator, would preclude participation in this study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment with UBX1325; UBX1325, single intravitreal injection, ascending dose	Drug: UBX1325; Investigational drug intravitreal injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Ocular and systemic safety and tolerability of a single intravitreal injection of UBX1325 evaluated by the incidence of dose limiting toxicities (DLTs) and treatment emergent adverse events (TEAEs)	24 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Plasma concentration of UBX1325 following a single intravitreal injection	up to 24 hours post dose

Collaborators and Investigators

• Sponsor: Unity Biotechnology, Inc.
• Investigators: Study Chair: Jamie Dananberg, MD, Unity Biotechnology

Study record dates

Study Major Dates

• Study Start (Actual): October 8, 2020
• Primary Completion (Actual): January 25, 2022
• Study Completion (Actual): January 25, 2022

Study Registration Dates

• First Submitted: August 28, 2020
• First Submitted That Met QC Criteria: August 28, 2020
• First Posted (Actual): September 3, 2020

Study Record Updates

• Last Update Posted (Actual): March 10, 2022
• Last Update Submitted That Met QC Criteria: March 8, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Keywords: Diabetes mellitus; Diabetic macular edema; Macular edema; Retinal disease; Neovascular Age-Related Macular Degeneration
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration; Macular Edema; Edema; Wet Macular Degeneration
• Other Study ID Numbers: UBX1325-01

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