Blepharoptosis
Conditions
Brief summary
Phase 3 study to evaluate the extended safety of RVL-1201 compared to placebo for treatment of blepharoptosis. Eligible subjects will be randomized to one of 2 treatment arms.
Interventions
DRUGRVL-1201
RVL-1201 ophthalmic solution, 0.1%
OTHERVehicle ophthalmic solution
Vehicle placebo ophthalmic solution
Sponsors
RVL Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
9 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male or Female 9 years of age or older 2. Females must not be pregnant or planning to get pregnant and must use acceptable form of contraception 3. Must be able to self-administer study medication 4. Must be able to understand and sign an Informed Consent Form (ICF). For minor subjects, the subject's parent or legal guardian must provide permission by signing an ICF on behalf of the subject and the subject should provide assent.
Exclusion criteria
1. Congenital ptosis 2. Horner syndrome 3. Myasthenia gravis 4. Mechanical ptosis 5. Previous ptosis surgery 6. Resting heart rate outside the normal range 7. Hypertension with resting diastolic blood pressure 8. Pregnancy or lactation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change From Baseline in Intraocular Pressure (IOP) at Day 84 | Screening/Day 1 to Day 84 | Intraocular pressure will be measured in mmHg utilizing a tonometer and using the standard of care. If possible, the same calibrated instrument should be used for a given subject throughout the study. |
| Mean Change From Baseline in Pupil Diameter (PD) at Day 84 | Screening/Day 1 to Day 84 | Pupil diameter will be measured in millimeters (either horizontally or vertically if top of pupil is not visible in photograph) from the external photograph. |
Countries
United States
Participant flow
Recruitment details
Planned sample size approximately 225 subjects, 150 subjects in the RVL-1201 group and 75 subjects in the Vehicle group, to be enrolled at approximately 30 clinical sites in the U.S.
Participants by arm
| Arm | Count |
|---|---|
| RVL-1201 Ophthalmic Solution, 0.1% RVL-1201 (oxymetazoline hydrochloride) ophthalmic solution 0.1% One drop each eye QD for 84 days | 157 |
| Vehicle Ophthalmic Solution Vehicle placebo ophthalmic solution One drop each eye QD for 84 days | 77 |
| Total | 234 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 4 | 0 |
| Overall Study | Lost to Follow-up | 2 | 1 |
| Overall Study | Non-compliance | 2 | 1 |
| Overall Study | Withdrawal by Subject | 6 | 0 |
Baseline characteristics
| Characteristic | RVL-1201 Ophthalmic Solution, 0.1% | Vehicle Ophthalmic Solution | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 2 Participants | 1 Participants | 3 Participants |
| Age, Categorical >=65 years | 90 Participants | 38 Participants | 128 Participants |
| Age, Categorical Between 18 and 65 years | 65 Participants | 38 Participants | 103 Participants |
| Age, Continuous | 63.7 years STANDARD_DEVIATION 14.64 | 63.3 years STANDARD_DEVIATION 14.63 | 63.6 years STANDARD_DEVIATION 14.6 |
| Age, Customized | 66 years | 64 years | 66 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 14 Participants | 5 Participants | 19 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 143 Participants | 72 Participants | 215 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 6 Participants | 3 Participants | 9 Participants |
| Race (NIH/OMB) Black or African American | 12 Participants | 8 Participants | 20 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 2 Participants | 0 Participants | 2 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 137 Participants | 66 Participants | 203 Participants |
| Region of Enrollment United States | 157 Participants | 77 Participants | 234 Participants |
| Sex: Female, Male Female | 126 Participants | 53 Participants | 179 Participants |
| Sex: Female, Male Male | 31 Participants | 24 Participants | 55 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 157 | 0 / 77 |
| other Total, other adverse events | 19 / 157 | 5 / 77 |
| serious Total, serious adverse events | 2 / 157 | 0 / 77 |
Outcome results
Primary
Mean Change From Baseline in Intraocular Pressure (IOP) at Day 84
Intraocular pressure will be measured in mmHg utilizing a tonometer and using the standard of care. If possible, the same calibrated instrument should be used for a given subject throughout the study.
Time frame: Screening/Day 1 to Day 84
Population: The safety population includes all subjects who receive at least one dose of double-masked study treatment and have at least one post-dose visit.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| RVL-1201 Ophthalmic Solution, 0.1% | Mean Change From Baseline in Intraocular Pressure (IOP) at Day 84 | Mean Change from Baseline IOP (OD) | -0.6 mmHg | Standard Deviation 2.4 |
| RVL-1201 Ophthalmic Solution, 0.1% | Mean Change From Baseline in Intraocular Pressure (IOP) at Day 84 | Mean Change from Baseline IOP (OS) | -0.6 mmHg | Standard Deviation 2.42 |
| Vehicle Ophthalmic Solution | Mean Change From Baseline in Intraocular Pressure (IOP) at Day 84 | Mean Change from Baseline IOP (OD) | -0.2 mmHg | Standard Deviation 2.52 |
| Vehicle Ophthalmic Solution | Mean Change From Baseline in Intraocular Pressure (IOP) at Day 84 | Mean Change from Baseline IOP (OS) | -0.2 mmHg | Standard Deviation 0.41 |
Primary
Mean Change From Baseline in Pupil Diameter (PD) at Day 84
Pupil diameter will be measured in millimeters (either horizontally or vertically if top of pupil is not visible in photograph) from the external photograph.
Time frame: Screening/Day 1 to Day 84
Population: The safety population includes all subjects who receive at least one dose of double-masked study treatment and have at least one post-dose visit.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| RVL-1201 Ophthalmic Solution, 0.1% | Mean Change From Baseline in Pupil Diameter (PD) at Day 84 | Mean Change From Baseline PD (OD) | 0.0 Millimeters (mm) | Standard Deviation 0.77 |
| RVL-1201 Ophthalmic Solution, 0.1% | Mean Change From Baseline in Pupil Diameter (PD) at Day 84 | Mean Change from Baseline PD (OS) | 0.0 Millimeters (mm) | Standard Deviation 0.77 |
| Vehicle Ophthalmic Solution | Mean Change From Baseline in Pupil Diameter (PD) at Day 84 | Mean Change From Baseline PD (OD) | -0.1 Millimeters (mm) | Standard Deviation 0.66 |
| Vehicle Ophthalmic Solution | Mean Change From Baseline in Pupil Diameter (PD) at Day 84 | Mean Change from Baseline PD (OS) | -0.1 Millimeters (mm) | Standard Deviation 0.59 |