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Safety and Efficacy Study of RVL-1201 in Acquired Blepharoptosis

R

RVL Pharmaceuticals

Status and phase

Completed
Phase 2
Phase 1

Conditions

Blepharoptosis

Treatments

Drug: RVL-1201 Vehicle Placebo
Drug: RVL-1201

Study type

Interventional

Funder types

Industry

Identifiers

NCT01848041
RVL-1201-001

Details and patient eligibility

About

This is an exploratory, proof of concept study to evaluate the safety and efficacy of RVL-1201 dosed once or twice daily for 14 days compared to a placebo (vehicle) control in patients with ptosis.

Full description

This is an exploratory, proof-of-concept study. The objectives include establishing the efficacy and duration of effect of once daily (QD) or twice daily (BID) administration of RVL-1201 and the safety profile following 14 days of treatment in 72 subjects (24 per arm) with acquired blepharoptosis.

Efficacy will be assessed at each treatment visit by the Humphrey Visual Field 36-point ptosis protocol test, photographic measurement of marginal reflex distance, palpebral fissure distance and contrast sensitivity in the study eye only and Visual Acuity assessment in both eyes.

Safety assessments will include slit lamp examination/corneal fluorescein staining, pupil size measurement, ophthalmoscopy/ fundus examination, tonometry, visual acuity; urine pregnancy test (for women of childbearing potential only), vital signs (Heart Rate/Blood Pressure); and collection of adverse events. Subject rating of study medication comfort and assessment of ongoing tolerability will also be obtained.

Primary efficacy endpoint is the mean increase from baseline in points seen on the HVF 36-point ptosis protocol test at various timepoints according to a hierarchical analysis.

Analysis of exploratory endpoints will provide characterization of the efficacy and duration of effect of RVL-1201 with a variety of efficacy measures, as well as the potential additional effect of BID over QD dosing and safety profile of BID administration of RVL-1201. Exploratory endpoints will be analyzed by each regimen against placebo and between regimens and will include:

  • The change from baseline in HVF, MRD, PFD, VA, and CS.
  • The change from baseline in BP/HR

Enrollment

46 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  1. Adult male or female subjects 18 years of age and older.

  2. Presence of all of the following at Screening:

    1. Loss on HVF 36-point ptosis protocol test of ≥ 8 points in points not seen at or above 10° from fixation in the superior visual field; AND
    2. Marginal reflex distance (MRD), the distance from the central pupillary light reflex to the upper lid margin, of ≤ 2.5 mm in the same eye as Inclusion Criterion #2a; AND
    3. Corrected Snellen visual acuity (VA) of 20/40 or better (refraction must be within 6 months of Visit 1) in the same eye as Inclusion Criteria #2a and #2b.
  3. No contraindications for treatment of both eyes as specified in Exclusion Criteria #1-14.

  4. Female subjects must be 1-year postmenopausal, surgically sterilized, or women of childbearing potential with a negative urine pregnancy test at Visit 1. Women of childbearing potential must use an acceptable form of contraception throughout the study.

  5. Provide informed consent prior to undergoing any study-related procedures.

Exclusion criteria

In either eye:

  1. Congenital ptosis

  2. Pseudoptosis

  3. Horner syndrome

  4. Marcus Gunn jaw-winking syndrome

  5. Myasthenia gravis

  6. Mechanical ptosis, including ptosis due to orbital or lid tumor, cicatricial processes affecting the movements of the upper lid, and enophthalmos

  7. Dermatochalasis as the sole cause of the signs of ptosis

  8. Previous ptosis surgery

  9. Lid position affected by lid or conjunctival scarring

  10. Current use of prescribed dry eye medication or punctal plugs; artificial tears are allowed

  11. Visual field loss from any cause other than ptosis

  12. Inability to fixate on the central fixation target of the HVF

  13. Primary open-angle glaucoma or ocular hypertension, intraocular pressure (IOP) > 24 mm Hg, or current use/use within 1 month prior to Visit 1 of any antiglaucoma medications.

  14. History of closed/narrow angle glaucoma (unless patent peripheral iridotomy has been performed > 3 months prior to Visit 1 and IOP < 20 mm Hg) or normal-tension glaucoma

  15. Use of over-the-counter vasoconstrictor/decongestant eye medication (eg, Visine® L.R.®) or any α-adrenergic agonist (including OTC products) at any time during the study

  16. Contact lens wear during the study period

    General:

  17. Resting heart rate (HR) outside the normal range (60 - 100 beats per minute)

  18. Hypertension diastolic blood pressure (BP) > 105 mm Hg

  19. Use of monoamine oxidase inhibitors (MAOIs; eg, isocarboxazid, phenelzine, tranylcypromine) within 14 days prior to Visit 1 or during the study

  20. Use of beta blockers (eg, propranolol, metoprolol, labetalol) within 14 days prior to Visit 1 or during the study

  21. Use of maprotiline, selective serotonin reuptake inhibitors ([SSRIs] eg, citalopram, escitalopram, paroxetine, fluoxetine, fluvoxamine, sertraline) or tricyclic antidepressants (eg, amitriptyline, doxepin, nortriptyline, amoxapine, clomipramine, desipramine, imipramine, protriptyline, trimipramine) at any time during the study

  22. A history of myocardial infarction, angina, arrhythmia, or irregular pulse

  23. Advanced arteriosclerotic disease

  24. History of thyroid disease

  25. Insulin-dependent diabetes or diabetes requiring oral hypoglycemic drugs; diet-controlled diabetes is allowed

  26. Pregnancy or lactation

  27. Diagnosed benign prostatic hypertrophy requiring medicinal therapy.

  28. History of contact or systemic allergic reaction to oxymetazoline or other sympathomimetic drugs (eg, phenylephrine, pseudoephedrine, ephedrine, phenylpropanolamine, fepradinol, or methoxamine)

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

46 participants in 3 patient groups, including a placebo group

RVL-1201 once daily
Experimental group
Description:
RVL-1201 0.1% ophthalmic solution dosed one full drop per eye in the morning; one full drop of vehicle (placebo) per eye approximately 8 hours after the morning dose
Treatment:
Drug: RVL-1201
RVL-1201 twice daily
Experimental group
Description:
RVL-1201 0.1% ophthalmic solution dosed one full drop per eye BID; approximately 8 hours between the morning dose and the afternoon dose
Treatment:
Drug: RVL-1201
RVL-1201 vehicle (placebo)
Placebo Comparator group
Description:
RVL 1201 ophthalmic solution vehicle (placebo) dosed one full drop per eye BID; approximately 8 hours between the morning dose and the afternoon dose
Treatment:
Drug: RVL-1201 Vehicle Placebo

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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