Clinical Efficacy of H.P. Acthar Gel 80 U/ml to Improve the Signs and Symptoms in Subjects With Dry Eye Disease

Study Overview
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Brief Summary
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Study Title: A Phase 4 Study to Assess the Clinical Efficacy of H.P. Acthar Gel 80 U/ml to Improve the Signs and Symptoms in Subjects with Dry Eye Disease

Detailed Description
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Objectives: The primary objective of this study is to investigate the safety and efficacy of H.P. Acthar Gel 80 U/ml in subjects with a documented clinical diagnosis of dry eye disease. Study Population: The study population will consist of subjects diagnosed with dry eye disease. Number of Subjects: Approximately 12 subjects Investigational Product: H.P. Acthar Gel 80 U/ml sufficient for the duration of the study will be supplied by Mallinkrodt to the enrolled subjects Route and Duration of Administration: Product will be injected subcutaneously weekly by subjects for approximately 12 weeks. Study Design: This is a Phase 4, single center single arm study designed to evaluate the safety and efficacy of H.P. Achthar Gel in subjects with dry eye disease. Approximately 25 subjects will be screened and 12 subjects enrolled at one center in the United States. Subjects will be given 80 international units of study medication subcutaneously depending on the severity of the disease as determined by the primary investigator. The study will include 3 study visits over 12 weeks. At Visit 1 Screening (14 +/- 1 days prior to Day 1), subjects meeting inclusion/exclusion criteria will begin investigational drug use. Subjects will return for evaluations at Visit 2 (Day 42+/-3 days) and Visit 3 (Day 84 +/-5 days). Subjects will be released from the study at the end of Visit 3 (Day 84+/- 5 days.)

Official Title
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Study Title: A Phase 4 Study to Assess the Clinical Efficacy of H.P. Acthar Gel 80 U/ml to Improve the Signs and Symptoms in Subjects With Dry Eye Disease

Conditions
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Dry Eye Disease

Intervention / Treatment
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* Drug: Corticotropin 80Unit/Ml Repository Injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: At Visit 1 (Screening) individuals of either gender or race will be eligible for study participation if they Provide written informed consent and HIPAA authorization prior to any study related procedures Are 18 years of age or older Are willing and able to follow instructions and can be present for required study visits. Have documented clinical diagnosis of dry eye disease in one or both eyes. Have a score of at least 40mm on the ocular discomfort scale Have at least 5 spk on one or both corneas Have a grade of 1 or greater in the nasal or temporal areas of one or both eyes. Have normal lid anatomy. Are women of child bearing potential who are not pregnant or lactating and who are either abstinent and willing to remain so for the course of the trial or have an IUD in place for at least 3 months prior and through Visit 4, barrier method with spermicide for at least 3 months prior and through Visit 4, stable hormonal contraceptive for at least 3 months prior and through Visit 4 or in a monogamous relationship with a surgically sterilized (vasectomized) partner at least 6 months prior to Visit 1 and through the course of the trial. Are postmenopausal (no menstrual cycle for at least one year prior to Visit 1) or have undergone bilateral tubal ligation, hysterectomy, hysterectomy with uni or bilateral oophorectomy, or bilateral oophorectomy. Exclusion Criteria: In order for subjects to be eligible for the study Have a known hypersensitivity or contraindication to the investigational product or their components. Have used any of the following medications within 14 days prior to screening a. Topical or nasal vasoconstrictors Subjects can be on the following medications if they have been on a stable dose for 12 weeks topical cyclosporine, topical lifitegrast and/or topical loteprednol etabonate. Tetracycline compounds, omega 3s, anticholinergics, anticonvulsants, antidepressants, retinoids, systemic immunosuppressive agents including oral corticosteroids, non-steroidals, antihistamines or mast cell stabilizers, punctal plugs, contact lens wear and glaucoma medications. Subjects must be unwilling to abstain from eyelash growth medications for the duration of the trial. Subjects must not have had penetrating intraocular surgery, refractive surgery or corneal transplantation, eyelid surgery within 12 weeks prior to Visit 1. Subjects with a history of herpetic keratitis. Have serious or severe disease or uncontrolled medical condition that in the judgement of the investigator could confound study assessments or limit compliance.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Acthar gel 80 U/ml<br>Patients who continue to experience clinically significant symptoms of dry eye disease even after utilizing traditional methods of treatment for dry eye including but not limited to artificial tears, warm compresses, topical anti-inflammatories like cyclosporine and/or lifitegrast. Patients will receive repository corticotropin intramuscular injections 80 u/ml 2-3 times weekly for up to 3 months as judged by the investigator. | Drug: Corticotropin 80Unit/Ml Repository Injection<br>* H.P. Acthar Gel (repository corticotropin injection) is an adrenocorticotropic hormone (ACTH) analogue used for: Treatment during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus.<br>* Other names: H.P. Acthar gel;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dry Eye Comfort Questionnaire, SANDE | Subjects will be asked to subjectively rate their eye dryness at Visit 1,2 and 3. Subjects will be instructed to rate eye dryness using the scale below. The total length of the line from no dryness to maximal dryness is 100 mm. The length of the line between the no dryness starting point and the first point at which the subject mark crosses the line will be measured in mm. This assessment is a general assessment of the change of both eyes of dryness measure from baseline to final study visit. The measure of discomfort was for the instantaneous measurement at the time of the specific visit. | 12 weeks total. Measure is change on SANDE scale (0 is no dryness and 100mm is maximal dryness) from baseline to final value. Higher values reflect greater amounts of subjective dryness. | 
| 1. Conjunctival Staining With Lissamine Green | Change in Conjunctival staining with lissamine green. | 12 weeks total. Measure is change from baseline to final, | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Intraocular Pressure | goldmann tonometry used by qualified technician with fluorescein staining | 12 weeks measuring change from baseline to final |