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

Sponsor

Unity Biotechnology, Inc. (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04857996

Collaborator

(none)

Enrollment

Locations

Arms

21.2

Anticipated Duration (Months)

2.8

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is intended to assess the exposure, safety, and biological activity 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).

Condition or Disease	Intervention/Treatment	Phase
Diabetic Macular Edema (DME)	Drug: UBX1325 Other: Sham	Phase 2

Detailed Description

This is a Phase 2 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 Design

Study Type:

Interventional

Actual Enrollment :

65 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Masking Description:

This study will be double masked, so patients and Investigators are masked to the treatment assigned, while certain roles at the site and at the Sponsor are unmasked. Sites will have a qualified injector who is unmasked to perform the study treatment injection or sham procedure, as well as certain post-injection assessments on Day 1 only.

Primary Purpose:

Treatment

Official Title:

A Phase 2, 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

Actual Study Start Date :

Jun 25, 2021

Anticipated Primary Completion Date :

Oct 1, 2022

Anticipated Study Completion Date :

Apr 1, 2023

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: UBX1325

Drug: UBX1325

Patients will be administered a single 50 μL UBX1325 IVT injection

Sham Comparator: Sham Control

Other: Sham

Sham procedure

Outcome Measures

Primary Outcome Measures

Ocular and systemic safety and tolerability of a single IVT injection of UBX1325 evaluated by incidence of treatment emergent adverse events (TEAEs) [Week 24]

Secondary Outcome Measures

Changes in best corrected visual acuity (BCVA) from Baseline [Week 12, 24 and 48]
Proportion of patients who require 2 or more anti-VEGF rescue [Week 12, 24 and 48]
Change in central subfield thickness (CST) from baseline to each study visit as assessed by spectral domain optical coherence tomography (SD-OCT) [Week 12, 24 and 48]
Change in CST area under the curve (AUC) from baseline to each study visit [Week 12, 24 and 48]
Proportion of patients without macular fluid as assessed by SD-OCT [Week 12, 24 and 48]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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
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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Retinal Consultants of Arizona	Phoenix	Arizona	United States	85014
2	California Retina Consultants	Bakersfield	California	United States	93103
3	The Retina Partners	Encino	California	United States	91436
4	Salehi Retina Institute Inc.	Huntington Beach	California	United States	92647
5	Loma Linda University	Loma Linda	California	United States	92350
6	California Retina Consultants	Oxnard	California	United States	93036
7	Advanced Vision Research Institute	Longmont	Colorado	United States	80503
8	MedEye Associates	Miami	Florida	United States	33143
9	Bascom Palmer Eye Institute	Miami	Florida	United States	33146
10	Northwestern University	Evanston	Illinois	United States	60208
11	MidWest Eye Institute	Indianapolis	Indiana	United States	46290
12	Sabates Eye Center	Leawood	Kansas	United States	66211
13	Cumberland Valley Retina Consultants	Hagerstown	Maryland	United States	21740
14	Mass Eye and Ear Institute	Boston	Massachusetts	United States	02114
15	Sierra Eye Institute	Reno	Nevada	United States	89502
16	New York Eye and Ear Infirmary	New York	New York	United States	10003
17	EyeHealth Northwest	Portland	Oregon	United States	97209
18	Retina Consultants of Carolina, PA	Greenville	South Carolina	United States	29605
19	Valley Retina Institute, P.A.	McAllen	Texas	United States	78503
20	Retina Center of Texas	Southlake	Texas	United States	76092
21	University of Alberta	Edmonton	Alberta	Canada	T6G 2R3
22	Toronto Retina Institute	North York	Ontario	Canada	M3C 0G9
23	Universite de Montreal	Montréal	Quebec	Canada	H1T 2M4