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A Phase II Study of RBM-007 Alone and RBM-007 With Eylea® in Subjects With Wet Age-related Macular Degeneration

A Multi-Center, Randomized, Double Masked and Active Controlled Phase II Study Assessing the Efficacy and Safety of Intravitreal Injections of RBM-007 Monotherapy and RBM-007 in Combination With Eylea® Compared to Eylea® Monotherapy

Status
Completed
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT04200248
Acronym
TOFU
Enrollment
94
Registered
2019-12-16
Start date
2019-12-02
Completion date
2021-12-22
Last updated
2023-06-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Exudative Age-related Macular Degeneration

Keywords

Fibrosis, Edema, Neovascularization

Brief summary

This is a multicenter, active-controlled, double masked study assessing the safety, efficacy and durability of four monthly intravitreal (IVT) injections of RBM-007 monotherapy, and four monthly RBM-007 injections in combination with Eylea® dosed at every other month, compared to Eylea® monotherapy dosed at every other month in approximately eighty-one subjects with exudative age-related macular degeneration (AMD).

Detailed description

RBM-007 is a novel oligonucleotide-based aptamer having potent anti-FGF2 activity and anti-VEGF-expression activity

Interventions

DRUGRBM-007 Injectable Solution

RBM-007 Injectable Solution

DRUGAflibercept

EYLEA® (aflibercept) Injection, for Intravitreal Use

DRUGSham

Sham intravitreal injection

Sponsors

Ribomic USA Inc
Lead SponsorINDUSTRY

Study design

Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No

Inclusion criteria

1. Provide signed written informed consent. 2. Male or female 55 years of age or older on the date of signing the consent and able and willing to comply with all treatment and study procedures. 3. Diagnosis of exudative age-related macular degeneration in the study eye, for which previous standard treatment with intravitreal anti-vascular endothelial growth factor agents (at least 4 injections over the past 8 months) has demonstrated incomplete resolution of exudation, as assessed by spectral domain optical coherence tomography. 4. Presence of macular edema or subretinal fluid. 5. Absence of central atrophy or retinal epithelial tear in the fovea or any condition preventing visual acuity improvement in the study eye. 6. Visual acuity of 78 to 24 letters (20/32 to 20/320) in the study eye. 7. Visual acuity of 24 letters (20/320) or better in the fellow eye. 8. Reasonably clear media and some fixation in the study eye to allow for good quality tomography and fundus photography

Exclusion criteria

* Ocular: 1. Use of any of the following treatments or anticipated use of any of the following treatments to the study eye: 1. Any intravitreal treatment within 4 weeks prior to Baseline (Visit 1). 2. Intravitreal or periocular corticosteroid, within 90 days prior to Visit 1 (Day 1) and throughout the study. 3. Fluocinolone acetonide intravitreal implant, within 12 months prior to Visit 1 (Day 1) and throughout the study. 4. Visudyne® photodynamic therapy, within 90 days prior to Visit 1 (Day 1) and throughout the study. 2. Uncontrolled or advanced glaucoma, evidenced by an intraocular pressure of \> 21 mmHg or cup/disc ratio \> 0.8 while on medical therapy, or chronic hypotony (\< 6 mmHg) in the study eye. 3. Evidence of any other ocular disease other than wet age-related macular degeneration in the study eye that may confound the outcome of the study 4. History of vitrectomy in the study eye. 5. Need for ocular surgery in the study eye during the course of the study. 6. YAG laser capsulotomy within 30 days prior to Visit 1 (Day 1) in the study eye. 7. Intraocular surgery, including lens removal or laser, within 90 days prior to Visit 1 (Day 1) in the study eye.

Design outcomes

Primary

MeasureTime frameDescription
Visual Acuity - ContinuousWeek 16Mean change in Best Corrected Visual Acuity from Baseline to Week 16

Secondary

MeasureTime frameDescription
Visual Acuity - CategoricalWeek 16Percentage of patients gaining \>= 15 letters as measured by Best Corrected Visual Acuity from Baseline at Week 16
Macular Thickness ChangeWeek 16Change from Baseline in Central Subfield Thickness by spectral domain optical coherence tomography at Week 16
Macular Volume ChangeWeek 16Change from Baseline in macular volume by spectral domain optical coherence tomography at Week 16
Fibrosis ChangeWeek 16Change from Baseline in sub-retinal hyper-reflective material by spectral domain optical coherence tomography at Week 16
Safety - OcularWeek 20Ocular examination (biomicroscopy and ophthalmoscopy) at Week 20 - Number of participants with additional corneal abnormalities

Countries

United States

Participant flow

Participants by arm

ArmCount
Sham + RBM-007
Sham + RBM-007 intravitreal injection RBM-007 Injectable Solution: RBM-007 Injectable Solution Sham: Sham intravitreal injection
32
RBM-007 + Aflibercept
RBM-007 + Aflibercept intravitreal injection RBM-007 Injectable Solution: RBM-007 Injectable Solution Aflibercept: EYLEA® (aflibercept) Injection, for Intravitreal Use
31
Sham + Aflibercept
Sham + Aflibercept intravitreal injection Aflibercept: EYLEA® (aflibercept) Injection, for Intravitreal Use Sham: Sham intravitreal injection
31
Total94

Withdrawals & dropouts

PeriodReasonFG000FG001FG002
Overall StudyDiscontinued prior to treatment332
Overall StudyPersonal reasons001

Baseline characteristics

CharacteristicSham + RBM-007Sham + AfliberceptTotalRBM-007 + Aflibercept
Age, Categorical
<=18 years
0 Participants0 Participants0 Participants0 Participants
Age, Categorical
>=65 years
31 Participants31 Participants90 Participants28 Participants
Age, Categorical
Between 18 and 65 years
1 Participants0 Participants4 Participants3 Participants
Age, Continuous80.41 years
STANDARD_DEVIATION 7.47
78.52 years
STANDARD_DEVIATION 7.07
78.1 years
STANDARD_DEVIATION 7.96
75.32 years
STANDARD_DEVIATION 8.64
Best corrected visual acuity60.24 Letter seen
STANDARD_DEVIATION 13
61.52 Letter seen
STANDARD_DEVIATION 13.83
62.78 Letter seen
STANDARD_DEVIATION 13.13
66.71 Letter seen
STANDARD_DEVIATION 10.81
Central subfield thickness (macula)452.07 Microns
STANDARD_DEVIATION 138.24
437.55 Microns
STANDARD_DEVIATION 129.34
429.58 Microns
STANDARD_DEVIATION 131.2
398.04 Microns
STANDARD_DEVIATION 124.01
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants2 Participants6 Participants2 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
30 Participants29 Participants88 Participants29 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants0 Participants
Fibrosis27 Participants28 Participants82 Participants27 Participants
Macular volume9.03 mm^3
STANDARD_DEVIATION 0.23
9.00 mm^3
STANDARD_DEVIATION 0.23
8.96 mm^3
STANDARD_DEVIATION 0.23
8.84 mm^3
STANDARD_DEVIATION 0.23
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
1 Participants0 Participants1 Participants0 Participants
Race (NIH/OMB)
Black or African American
0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
1 Participants0 Participants1 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
White
30 Participants31 Participants92 Participants31 Participants
Region of Enrollment
United States
32 participants31 participants94 participants31 participants
Sex: Female, Male
Female
21 Participants18 Participants54 Participants15 Participants
Sex: Female, Male
Male
11 Participants13 Participants40 Participants16 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
EG002
affected / at risk
deaths
Total, all-cause mortality
0 / 280 / 290 / 29
other
Total, other adverse events
15 / 2817 / 298 / 29
serious
Total, serious adverse events
2 / 282 / 291 / 29

Outcome results

Primary

Visual Acuity - Continuous

Mean change in Best Corrected Visual Acuity from Baseline to Week 16

Time frame: Week 16

Population: Data presented for population treated (8 subjects discontinued before treatment)

ArmMeasureValue (MEAN)Dispersion
Sham + RBM-007Visual Acuity - Continuous-6.1 Letters seenStandard Error 1.7
RBM-007 + AfliberceptVisual Acuity - Continuous-1.6 Letters seenStandard Error 1.75
Sham + AfliberceptVisual Acuity - Continuous2.4 Letters seenStandard Error 1.69
Secondary

Fibrosis Change

Change from Baseline in sub-retinal hyper-reflective material by spectral domain optical coherence tomography at Week 16

Time frame: Week 16

Population: Data presented for population treated (8 subjects discontinued before treatment)

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Sham + RBM-007Fibrosis ChangeBetter3 Participants
Sham + RBM-007Fibrosis ChangeNot better26 Participants
RBM-007 + AfliberceptFibrosis ChangeBetter8 Participants
RBM-007 + AfliberceptFibrosis ChangeNot better20 Participants
Sham + AfliberceptFibrosis ChangeBetter9 Participants
Sham + AfliberceptFibrosis ChangeNot better20 Participants
Secondary

Macular Thickness Change

Change from Baseline in Central Subfield Thickness by spectral domain optical coherence tomography at Week 16

Time frame: Week 16

Population: Data presented for population treated (8 subjects discontinued before treatment)

ArmMeasureValue (MEAN)Dispersion
Sham + RBM-007Macular Thickness Change36.5 micronsStandard Error 12.6
RBM-007 + AfliberceptMacular Thickness Change-5.8 micronsStandard Error 12.89
Sham + AfliberceptMacular Thickness Change-16.1 micronsStandard Error 12.55
Secondary

Macular Volume Change

Change from Baseline in macular volume by spectral domain optical coherence tomography at Week 16

Time frame: Week 16

Population: Data presented for population treated (8 subjects discontinued before treatment)

ArmMeasureValue (MEAN)Dispersion
Sham + RBM-007Macular Volume Change0.748 mm^3Standard Error 0.12
RBM-007 + AfliberceptMacular Volume Change0.0095 mm^3Standard Error 0.124
Sham + AfliberceptMacular Volume Change-0.010 mm^3Standard Error 0.12
Secondary

Safety - Ocular

Ocular examination (biomicroscopy and ophthalmoscopy) at Week 20 - Number of participants with additional corneal abnormalities

Time frame: Week 20

Population: Data presented for population treated (8 subjects discontinued before treatment)

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Sham + RBM-007Safety - OcularAdditional corneal abnormalities9 Participants
Sham + RBM-007Safety - OcularNo additional corneal abnormalities19 Participants
RBM-007 + AfliberceptSafety - OcularAdditional corneal abnormalities11 Participants
RBM-007 + AfliberceptSafety - OcularNo additional corneal abnormalities18 Participants
Sham + AfliberceptSafety - OcularAdditional corneal abnormalities10 Participants
Sham + AfliberceptSafety - OcularNo additional corneal abnormalities19 Participants
Secondary

Visual Acuity - Categorical

Percentage of patients gaining \>= 15 letters as measured by Best Corrected Visual Acuity from Baseline at Week 16

Time frame: Week 16

Population: Data presented for population treated (8 subjects discontinued before treatment)

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
Sham + RBM-007Visual Acuity - Categorical0 Participants
RBM-007 + AfliberceptVisual Acuity - Categorical0 Participants
Sham + AfliberceptVisual Acuity - Categorical2 Participants

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026