Diabetic Macular Edema (DME)Phase 2NCT04857996
Safety, Tolerability and Evidence of Activity Study of UBX1325 in Patients With Diabetic Macular Edema (BEHOLD)
A Phase 2 study testing UBX1325 in people with Diabetic Macular Edema (DME), run by Unity Biotechnology, Inc. at 22 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diabetic Macular Edema (DME)
- What is being tested?
- UBX1325
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 65 participants
- Where
- 22 sitesin 2 countries
- Study dates
- 25 Jun 2021to 6 Apr 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
22 sites in 2 countries.
22 sites in 2 countries
United States19 sites
California Retina Consultants
Bakersfield, California, 93309
Mass Eye and Ear Institute
Boston, Massachusetts, 02114
Northwestern Medical Group
Chicago, Illinois, 60611
The Retina Partners
Encino, California, 91436
Retina Consultants of Carolina, PA
Greenville, South Carolina, 29605
Cumberland Valley Retina Consultants
Hagerstown, Maryland, 21740
Salehi Retina Institute Inc.
Huntington Beach, California, 92647
MidWest Eye Institute
Indianapolis, Indiana, 46290
Loma Linda University
Loma Linda, California, 92354
Advanced Vision Research Institute
Longmont, Colorado, 80503
Valley Retina Institute, P.A.
McAllen, Texas, 78503
Bascom Palmer Eye Institute
Miami, Florida, 33136
MedEye Associates
Miami, Florida, 33143
New York Eye and Ear Infirmary
New York, New York, 10003
California Retina Consultants
Oxnard, California, 93036
Retinal Research Institute, LLC
Phoenix, Arizona, 85053
EyeHealth Northwest
Portland, Oregon, 97225
Sierra Eye Associates
Reno, Nevada, 89502
Retina Center of Texas
Southlake, Texas, 76092
Canada3 sites
Alberta Retina Research Corporation
Edmonton, Alberta, T5H OX5
Hopital Maisonneuve-Rosemont
Montreal, Quebec, H1T 2M4
Toronto Retina Institute
North York, Ontario, M3C 0G9
About this study
Official title: 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).
Read the 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)
- Being tested
- UBX1325, Sham
- Keywords
- Retinal disease, Macular edema, Diabetes mellitus, Diabetic macular edema
- Medical subject headings
- Retinal Diseases, Macular Edema, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UBX1325Drug
Patients will be administered a single 50 μL UBX1325 IVT injection
ShamOther
Sham procedure
| Study group | Role | What they receive |
|---|---|---|
| UBX1325 | Experimental | Not described Receives: Drug: UBX1325 |
| Sham Control | Sham comparator | Not described Receives: Other: Sham |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Up to Week 12, 24 and 48 weeks
Secondary outcomes (1)
Changes in Best Corrected Visual Acuity (BCVA) From Baseline
Best Corrected Visual Acuity was assessed using the ETDRS chart starting at 4 meters
Measured: Week 12, 24 and 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2021
- Study start25 Jun 2021
- Primary completion6 Apr 2023
- Study completion6 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Diabetic Macular Edema (DME)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- Unity Biotechnology, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sharon Klier, MD, MPH, Study director, Unity Biotechnology, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04857996
- Sponsor's protocol ID
- UBX1325-02
- Status last verified
- Apr 2024
- First submitted
- 21 Apr 2021
- Last updated
- 16 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 40261111
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04857996
- Acronym / sponsor ID
- UBX1325-02
- Conditions
- Diabetic Macular Edema (DME)
- Keywords
- Retinal disease, Macular edema, Diabetes mellitus, Diabetic macular edema
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Unity Biotechnology, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 Jun 2021 (actual)
- Primary completion
- 6 Apr 2023 (actual)
- Study completion
- 6 Apr 2023 (actual)
- First posted
- 23 Apr 2021 (actual)
- Results
- Posted 16 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| UBX1325 | Experimental | Drug: UBX1325 |
| Sham Control | Sham comparator | Other: Sham |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UBX1325Patients will be administered a single 50 μL UBX1325 IVT injection | Drug | UBX1325 |
| ShamSham procedure | Other | Sham Control |
Primary outcome
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Time frame: 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
Time frame: 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
1 secondary outcome
Changes in Best Corrected Visual Acuity (BCVA) From Baseline
Time frame: Week 12, 24 and 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40261111Klier 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.
- Individual participant dataSharing planned: No
Related trials for Diabetic Macular Edema (DME)
RecruitingPhase 3
A Phase 3 Trial for Intravitreal Injection of 4D-150 in Adults With Diabetic Macular Edema (DME).
Diabetic Macular Edema (DME)
RecruitingPhase 2
Oral N-Acetylcysteine on Diabetic Macular Edema
Diabetic Macular Edema (DME)
Active, not recruitingPhase 3
COMO: A Phase 3 Randomized, Double-Masked Study Comparing the Efficacy of EYP-1901 Against Aflibercept in DME
Diabetic Macular Edema, DME
RecruitingPhase 3
A Clinical Trial of MK-8748 Compared to Aflibercept in Participants With Diabetic Macular Edema (MK-8748-005)
Diabetic Retinopathy, Macular Edema
RecruitingPhase 3
A Clinical Trial of MK-8748 Compared to Aflibercept in Participants With Diabetic Macular Edema (MK-8748-004)
Diabetic Retinopathy, Macular Edema
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sharon Klier, MD, MPH study director
Unity Biotechnology, Inc.
22 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing UBX1325 in people with Diabetic Macular Edema (DME), run by Unity Biotechnology, Inc. at 22 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325. (time frame: 48 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04857996, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2024.