Repository Corticotropin Injection in Keratoconjunctivitis Sicca

Efficacy of Repository Corticotropin Injection in Patients With Severe and Recalcitrant Keratoconjunctivitis Sicca

The purpose of this pilot study is to assess repository corticotropin injection (RCI) in the form of H.P. Acthar Gel in patients with severe keratoconjunctivitis sicca (KCS, or dry eye disease). This pilot study is a non-randomized, open-label, interventional study to assess the efficacy and timeline of RCI for the treatment of severe KCS recalcitrant to conventional therapy. The purpose is to acquire preliminary data to support and guide the design of a future, double-masked, randomized, controlled clinical trial.

Study Overview

• Status: Withdrawn
• Conditions: Keratoconjunctivitis Sicca
• Intervention / Treatment: Drug: repository corticotropin injection

Detailed Description

This study is a non-randomized, open-label, interventional pilot study with sixteen weeks (112 days) of active treatment, a possible twelve-week treatment extension, and twenty-four weeks of washout follow-up. Intervention is with HP Acthar Gel dosed at 80 U BIW SC indicated for use in anterior segment disease and keratitis. Data will be collected from five subjects. All subjects will be treated with 80 U BIW SC and clinically examined at two, four, six, nine, twelve, and sixteen weeks with a potential extension of up to twenty-eight weeks. Extension will be contingent upon specific outcome criteria defined in this protocol. Four clinical visits will occur at two, four, eight, and twelve weeks after initiating the drug taper to assess for regression of the signs and symptoms of KCS.

• Study Type: Interventional
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama at Birmingham

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Age ≥ 18 years
• Severe keratoconjunctivitis sicca (KCS) as defined by meeting at least three of the following in the same or both eye(s): (1) OSDI ≥ 33, (2) sum corneal staining with fluorescein of ≥ 5 in at least one eye, (3) MMP-9 concentration ≥ 40 ng/ml in at least one eye as measured by InflammaDry®, and (4) bulbar hyperemia of ≥ 2.0 in at least one eye as measured by the Oculus Keratograph® 5M
• Recalcitrant or intolerant to treatment with cyclosporine 0.05% ophthalmic emulsion (Restasis®) or lifitegrast 5% ophthalmic solution (Xiidra®)

Exclusion Criteria:

• Past or present history of: scleroderma, osteoporosis, systemic bacterial or fungal infection (including but not limited to tuberculosis), psychosis, ocular herpes simplex, peptic ulcer, congestive heart failure, diabetes mellitus, myasthenia gravis, hypothyroidism, liver cirrhosis, primary adrenocortical insufficiency or adrenocortical hyperfunction, central serous chorioretinopathy.
• Any corticosteroid use within 60 days of study enrollment
• Topical cyclosporine 0.05% ophthalmic emulsion (Restasis) or lifitegrast 5% ophthalmic solution (Xiidra) use within 60 days of study enrollment
• Recent surgery within 90 days
• Current uncontrolled, sustained hypertension
• Post-prandial blood glucose value of ≥ 140 mg/dl as measured at the baseline visit
• Sensitivity to proteins of porcine origin
• Anticipated administration of live or live attenuated vaccines during the course of the study
• Known sensitivity to steroid or complications from prior steroid use that subject is unwilling to tolerate
• Pregnancy (as assessed by urine hCG) or nursing
• Participation in a clinical trial involving a drug or device within the past 30 days
• Investigator discretion based upon medical history and/or opinion of ability to maintain protocol compliance

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment; Treated with repository corticotropin injection	Drug: repository corticotropin injection; 80 units twice per week as a subcutaneous injection; Other Names: HP Acthar Gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Conjunctival hyperemia	Change from baseline in bulbar conjunctival hyperemia as measured by the Oculus Keratograph 5M	16 weeks
Corneal staining	Change from baseline in corneal staining with fluorescein	16 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient symptoms	Change from baseline in patient symptoms as measured by Ocular Surface Disease Index (OSDI)	16 weeks
Tear cytokine concentration	Change from baseline in tear cytokine concentration as measured by Raybiotech Human Dry Eye Disease Array Q1	16 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Needle Fear	Change from baseline in Needle Fear Survey	16 weeks

Collaborators and Investigators

• Sponsor: University of Alabama at Birmingham
• Investigators: Principal Investigator: Jason J Nichols, OD, MPH, PhD, University of Alabama at Birmingham

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2018
• Primary Completion (Actual): November 1, 2018
• Study Completion (Actual): November 1, 2018

Study Registration Dates

• First Submitted: January 2, 2018
• First Submitted That Met QC Criteria: January 5, 2018
• First Posted (Actual): January 12, 2018

Study Record Updates

• Last Update Posted (Actual): December 14, 2018
• Last Update Submitted That Met QC Criteria: December 12, 2018
• Last Verified: December 1, 2018

More Information

Terms related to this study

• Keywords: dry eye; corticotropin
• Additional Relevant MeSH Terms: Eye Diseases; Lacrimal Apparatus Diseases; Conjunctivitis; Conjunctival Diseases; Keratitis; Corneal Diseases; Dry Eye Syndromes; Keratoconjunctivitis Sicca; Keratoconjunctivitis; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Adrenocorticotropic Hormone
• Other Study ID Numbers: F00000001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No