Trial Outcomes & Findings for Safety, Tolerability and Evidence of Activity Study of UBX1325 in Patients With Diabetic Macular Edema (BEHOLD) (NCT NCT04857996)
NCT ID: NCT04857996
Last Updated: 2024-05-16
Results Overview
The systemic safety and tolerability is evaluated by incidence of Treatment Emergent Adverse Events (TEAEs) by System organ class Preferred Term.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
65 participants
Primary outcome timeframe
48 weeks
Results posted on
2024-05-16
Participant Flow
each participant contributed only one study eye
Unit of analysis: study eye
Participant milestones
| Measure |
Sham Control
Sham: Sham procedure
|
UBX1325
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
|---|---|---|
|
Overall Study
STARTED
|
33 33
|
32 32
|
|
Overall Study
COMPLETED
|
26 26
|
24 24
|
|
Overall Study
NOT COMPLETED
|
7 7
|
8 8
|
Reasons for withdrawal
| Measure |
Sham Control
Sham: Sham procedure
|
UBX1325
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
|---|---|---|
|
Overall Study
Completed 24 weeks before study extension to 48 weeks was available as per Protocol version 4.0
|
4
|
5
|
|
Overall Study
Lost to Follow-up
|
1
|
3
|
|
Overall Study
Site Closure
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Safety, Tolerability and Evidence of Activity Study of UBX1325 in Patients With Diabetic Macular Edema (BEHOLD)
Baseline characteristics by cohort
| Measure |
Sham Control
n=33 Participants
Sham: Sham procedure
|
UBX1325
n=32 Participants
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
Total
n=65 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
61.4 years
STANDARD_DEVIATION 9.09 • n=99 Participants
|
63.6 years
STANDARD_DEVIATION 9.33 • n=107 Participants
|
62.5 years
STANDARD_DEVIATION 9.20 • n=206 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
44 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
53 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Asian
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
White
|
25 Participants
n=99 Participants
|
24 Participants
n=107 Participants
|
49 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Not reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Study Eye
Left Eye
|
11 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
29 Participants
n=206 Participants
|
|
Study Eye
Right eye
|
22 Participants
n=99 Participants
|
14 Participants
n=107 Participants
|
36 Participants
n=206 Participants
|
|
Eye Color
Blue
|
8 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
|
Eye Color
Brown
|
18 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
37 Participants
n=206 Participants
|
|
Eye Color
Green
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Eye Color
Hazel
|
7 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Eye Color
Other
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 48 weeksPopulation: The Safety Analysis Set includes all patients who received a single IVT treatment of UBX1325 or sham. Summary statistics are frequency (percentage of column total).
The systemic safety and tolerability is evaluated by incidence of Treatment Emergent Adverse Events (TEAEs) by System organ class Preferred Term.
Outcome measures
| Measure |
Sham Control
n=33 Participants
Sham: Sham procedure
|
UBX1325
n=32 Participants
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
Overall
n=65 Participants
Overall participants that received either Sham or UBX1325 10μg treatment
|
|---|---|---|---|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Subjects with at Least One Serious TEAE
|
4 Participants
|
5 Participants
|
9 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Blood and lymphatic system disorders - Anemia
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Cardiac disorders - Atrial fibrillation
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Cardiac disorders - Coronary artery occlusion
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Eye disorders-Visual acuity reduced
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Eye disorders - Vitreous haemorrhage
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Infections and infestations - Cellulitis
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Infections and infestations - Pneumonia
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Injury, poisoning and procedural complication - Ankle fracture
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Injury, poisoning and procedural complication- Skin abrasion
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Nervous system disorders - Cerebrovascular accident
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Systemic Safety and Tolerability of a Single IVT Injection of UBX1325.
Vascular disorders - Aortic stenosis
|
1 Participants
|
0 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: Up to Week 12, 24 and 48 weeksOcular 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
Outcome measures
| Measure |
Sham Control
n=33 Participants
Sham: Sham procedure
|
UBX1325
n=32 Participants
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
Overall
n=65 Participants
Overall participants that received either Sham or UBX1325 10μg treatment
|
|---|---|---|---|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Subjects with At Least One Ocular TEAE in Study Eye
|
28 Participants
|
23 Participants
|
51 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Eye disorders
|
28 Participants
|
23 Participants
|
51 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Diabetic retinal oedema
|
18 Participants
|
14 Participants
|
32 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Conjunctival haemorrhage
|
2 Participants
|
8 Participants
|
10 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Vitreous haemorrhage
|
5 Participants
|
2 Participants
|
7 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Macular thickening
|
2 Participants
|
1 Participants
|
3 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Retinal oedema
|
2 Participants
|
1 Participants
|
3 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Cataract subcapsular
|
1 Participants
|
1 Participants
|
2 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Eye irritation
|
0 Participants
|
2 Participants
|
2 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Cataract nuclear
|
1 Participants
|
1 Participants
|
2 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Visual acuity reduced
|
2 Participants
|
0 Participants
|
2 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Anterior chamber pigmentation
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Conjunctival hyperaemia
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Dry eye
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Foreign body sensation in eyes
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Meibomian gland dysfunction
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Ocular hypertension
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Posterior capsule opacification
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Punctate keratitis
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Retinal exudates
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Retinal haemorrhage
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Ocular Safety and Tolerability of a Single IVT Injection of UBX1325
Vitreous floaters
|
1 Participants
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Week 12, 24 and 48Population: MMRM Model for BCVA Change from Baseline (Hypothetical Strategy) Full Analysis Set. The hypothetical strategy included all subjects but excluded observations post-rescue. The Full Analysis Set includes all randomized subjects who received a single IVT treatment of UBX1325 or sham, have a baseline BCVA assessment, and at least one post-baseline BCVA assessment.
Best Corrected Visual Acuity was assessed using the ETDRS chart starting at 4 meters
Outcome measures
| Measure |
Sham Control
n=32 Participants
Sham: Sham procedure
|
UBX1325
n=32 Participants
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
Overall
Overall participants that received either Sham or UBX1325 10μg treatment
|
|---|---|---|---|
|
Changes in Best Corrected Visual Acuity (BCVA) From Baseline
Week 12
|
1.5 ETDRS Letters
Standard Error 1.68
|
4.7 ETDRS Letters
Standard Error 1.59
|
—
|
|
Changes in Best Corrected Visual Acuity (BCVA) From Baseline
Week 24
|
-1.4 ETDRS Letters
Standard Error 1.99
|
6.7 ETDRS Letters
Standard Error 1.88
|
—
|
|
Changes in Best Corrected Visual Acuity (BCVA) From Baseline
Week 48
|
0.6 ETDRS Letters
Standard Error 2.90
|
6.2 ETDRS Letters
Standard Error 2.12
|
—
|
Adverse Events
Sham Control
Serious events: 4 serious events
Other events: 31 other events
Deaths: 0 deaths
UBX1325
Serious events: 5 serious events
Other events: 24 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Sham Control
n=33 participants at risk
Sham: Sham procedure
|
UBX1325
n=32 participants at risk
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Cardiac disorders
Coronary artery occlusion
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Eye disorders
Visual acuity reduced
|
3.0%
1/33 • Number of events 1 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
|
Eye disorders
Vitreous haemorrhage
|
3.0%
1/33 • Number of events 1 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
|
Infections and infestations
Cellulitis
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Infections and infestations
Pneumonia
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
0.00%
0/33 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Nervous system disorders
Cerebrovascular accident
|
3.0%
1/33 • Number of events 1 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
|
Vascular disorders
Aortic stenosis
|
3.0%
1/33 • Number of events 1 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
Other adverse events
| Measure |
Sham Control
n=33 participants at risk
Sham: Sham procedure
|
UBX1325
n=32 participants at risk
UBX1325: Patients will be administered a single 10 μg UBX1325 IVT injection
|
|---|---|---|
|
Eye disorders
Diabetic retinal oedema
|
57.6%
19/33 • Number of events 19 • up to 48 weeks
|
53.1%
17/32 • Number of events 17 • up to 48 weeks
|
|
Eye disorders
Conjunctival haemorrhage
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
25.0%
8/32 • Number of events 8 • up to 48 weeks
|
|
Eye disorders
Cataract subcapsular
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Eye disorders
Vitreous haemorrhage
|
12.1%
4/33 • Number of events 4 • up to 48 weeks
|
9.4%
3/32 • Number of events 3 • up to 48 weeks
|
|
Eye disorders
Eye irritation
|
0.00%
0/33 • up to 48 weeks
|
6.2%
2/32 • Number of events 2 • up to 48 weeks
|
|
Eye disorders
Macular thickening
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Eye disorders
Posterior capsule opacification
|
9.1%
3/33 • Number of events 3 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
|
Eye disorders
Retinal oedema
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Eye disorders
Visual acuity reduced
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
|
Infections and infestations
COVID-19
|
15.2%
5/33 • Number of events 5 • up to 48 weeks
|
12.5%
4/32 • Number of events 4 • up to 48 weeks
|
|
Infections and infestations
Nasopharyngitis
|
9.1%
3/33 • Number of events 3 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Investigations
Electrocardiogram abnormal
|
0.00%
0/33 • up to 48 weeks
|
6.2%
2/32 • Number of events 2 • up to 48 weeks
|
|
Metabolism and nutrition disorders
Type 2 diabetes mellitus
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Nervous system disorders
Headache
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
|
Psychiatric disorders
Depression
|
6.1%
2/33 • Number of events 2 • up to 48 weeks
|
0.00%
0/32 • up to 48 weeks
|
|
Vascular disorders
Hypertension
|
9.1%
3/33 • Number of events 3 • up to 48 weeks
|
3.1%
1/32 • Number of events 1 • up to 48 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place