Age-related Macular Degeneration
Conditions
Brief summary
This is an open label, non-controlled, dose-escalating study assessing the safety, tolerability, and bioactivity of a single intravitreal (i.vt.) injection of RBM-007 in approximately nine subjects with exudative age-related macular degeneration.
Detailed description
Nine subjects in three dose cohorts (3 subjects each cohort) will receive a single i.vt. injection of RBM-007 in the study eye. Subjects will be followed through Day 56.
Interventions
(No additional description)
Sponsors
Ribomic USA Inc
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is an open label, non-controlled, sequential, dose-escalating study
Eligibility
Sex/Gender
ALL
Age
55 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
1. Male or female 55 years of age or older on the date of signing the informed consent form and able and willing to comply with all treatment and follow-up study procedures. 2. At Screening Visit, subjects must meet all the following inclusion criteria: 3. Must have had prior treatment in the study eye with any intravitreal vasoactive endothelial growth factor (VEGF) medication (at least 3 anti-VEGF) treatments within the prior 2-6 months), throughout which clinical examination and SD-OCT imaging has shown recurrent or persistent exudative activity, as shown by the presence of intraretinal or subretinal fluid, and/or subretinal exudation or hemorrhage. 4. Best corrected visual acuity of 65 to 20 Early Treatment of Diabetic Retinopathy Study (ETDRS) letters (20/50 to 20/400) in the study eye. 5. Presence of significant subretinal fluid and/or cystoid macular edema secondary to exudative age-related macular degeneration as assessed by optical coherence tomography in the study eye, with a minimum of 300 µm within the central subfield. 6. Total lesion size of ≤9 disc areas, lesion containing ≤50% hemorrhage and ≤50% subretinal fibrosis and ≤50% retinal pigment epithelial atrophy in the study eye. 7. Reasonably clear media and reasonable fixation ability in the study eye to allow for good quality tomography and fundus photography. At Baseline Visit (Day 0), subjects must meet all the following inclusion criteria: 8. Best Corrected Visual Acuity (BCVA) of 65 to 20 ETDRS letters (20/50 to 20/400) in the study eye. 9. Presence of significant subretinal and/or intraretinal fluid secondary to exudative age-related macular degeneration as assessed by SD-OCT in the study eye, with a minimum of 300 µm within the central subfield. 10. Total lesion size of ≤9 disc areas, containing ≤ 50% hemorrhage and ≤ 50% fibrosis and ≤ 50% retinal pigment epithelial atrophy in the study eye.
Exclusion criteria
Ocular
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Ocular Safety as Assessed Using Biomicroscopy to Investigate Ocular Tolerability | Day 56 | Biomicroscopy is used by an ophthalmologist to assess the health of the front of the eye. The measure is the number of subjects with abnormal findings that were not present at screening. |
| Ocular Safety as Assessed Using Ophthalmoscopy to Investigate Ocular Tolerability | Day 56 | Ophthalmoscopy is used by an ophthalmologist to assess the health of the back of the eye. The measure is the number of subjects with abnormal findings that were not present at screening. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Ocular Tolerability as Assessed by Number and Nature of Adverse Events | Day 56 | Adverse events (ocular or non-ocular) |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| RBM-007 Injectable Solution - 0.2 mg No additional information.
RBM-007 Injectable Solution: (No additional description) | 3 |
| RBM-007 Injectable Solution - 1.0 mg No additional information.
RBM-007 Injectable Solution: (No additional description) | 3 |
| RBM-007 Injectable Solution - 2.0 mg No additional information.
RBM-007 Injectable Solution: (No additional description) | 3 |
| Total | 9 |
Baseline characteristics
| Characteristic | RBM-007 Injectable Solution - 0.2 mg | RBM-007 Injectable Solution - 1.0 mg | RBM-007 Injectable Solution - 2.0 mg | Total |
|---|---|---|---|---|
| Age, Customized Range | 87 years | 80 years | 86 years | 86 years |
| Ocular safety as assessed using biomicroscopy to investigate ocular tolerability | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Ocular safety as assessed using ophthalmoscopy to investigate ocular tolerability | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 3 Participants | 3 Participants | 3 Participants | 9 Participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 3 Participants | 5 Participants |
| Sex: Female, Male Male | 2 Participants | 2 Participants | 0 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 0 / 3 | 0 / 3 |
| other Total, other adverse events | 1 / 3 | 2 / 3 | 2 / 3 |
| serious Total, serious adverse events | 0 / 3 | 0 / 3 | 0 / 3 |
Outcome results
Primary
Ocular Safety as Assessed Using Biomicroscopy to Investigate Ocular Tolerability
Biomicroscopy is used by an ophthalmologist to assess the health of the front of the eye. The measure is the number of subjects with abnormal findings that were not present at screening.
Time frame: Day 56
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| RBM-007 Injectable Solution - 0.2 mg | Ocular Safety as Assessed Using Biomicroscopy to Investigate Ocular Tolerability | 0 Participants |
| RBM-007 Injectable Solution - 1.0 mg | Ocular Safety as Assessed Using Biomicroscopy to Investigate Ocular Tolerability | 0 Participants |
| RBM-007 Injectable Solution - 2.0 mg | Ocular Safety as Assessed Using Biomicroscopy to Investigate Ocular Tolerability | 1 Participants |
Primary
Ocular Safety as Assessed Using Ophthalmoscopy to Investigate Ocular Tolerability
Ophthalmoscopy is used by an ophthalmologist to assess the health of the back of the eye. The measure is the number of subjects with abnormal findings that were not present at screening.
Time frame: Day 56
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| RBM-007 Injectable Solution - 0.2 mg | Ocular Safety as Assessed Using Ophthalmoscopy to Investigate Ocular Tolerability | 0 Participants |
| RBM-007 Injectable Solution - 1.0 mg | Ocular Safety as Assessed Using Ophthalmoscopy to Investigate Ocular Tolerability | 0 Participants |
| RBM-007 Injectable Solution - 2.0 mg | Ocular Safety as Assessed Using Ophthalmoscopy to Investigate Ocular Tolerability | 0 Participants |
Secondary
Ocular Tolerability as Assessed by Number and Nature of Adverse Events
Adverse events (ocular or non-ocular)
Time frame: Day 56
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| RBM-007 Injectable Solution - 0.2 mg | Ocular Tolerability as Assessed by Number and Nature of Adverse Events | 1 Participants |
| RBM-007 Injectable Solution - 1.0 mg | Ocular Tolerability as Assessed by Number and Nature of Adverse Events | 2 Participants |
| RBM-007 Injectable Solution - 2.0 mg | Ocular Tolerability as Assessed by Number and Nature of Adverse Events | 2 Participants |