Efficacy and Safety of H.P. ACTHAR GEL in Adults With Retinal Vasculitis

Open-label, Single-center, Prospective Study on the Efficacy and Safety of Repository Corticotropin Injection (H.P. ACTHAR GEL) in the Treatment of Adults With Non-infectious Retinal Vasculitis

The purpose of this study is to evaluate the short- and long-term efficacy and safety of Acthar for the treatment of adults with non-infectious retinal vasculitis.

Study Overview

• Status: Unknown
• Conditions: Uveitis, Posterior; Vasculitis Retinal
• Intervention / Treatment: Drug: H.P. ACTHAR GEL

• Study Type: Interventional
• Enrollment (Anticipated): 40
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Waltham, Massachusetts, United States, 02451
      • Recruiting
      • Ocular Immunology & Uveitis Foundation
      • Contact: C. Stephen Foster, MD; Phone Number: 781-891-6377; Email: sfoster@mersi.com
      • Contact: Stephen D. Anesi, MD; Phone Number: 781-891-6377; Email: sanesi@mersi.com
      • Principal Investigator: C. Stephen Foster, MD
      • Sub-Investigator: Stephen D. Anesi, MD
      • Sub-Investigator: Peter Chang, MD

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:

• Any adult patient with active retinal vasculitis (involving arteries or veins) as a manifestation of non-infectious ocular inflammatory disease; fundus must be viewable with wide-field FA in the study eye
• If both eyes are involved, both are eligible for inclusion in the study
• Patients already on non-steroidal immunosuppressive therapy may continue on this during the study, but the dose must not be increased or decreased within 6 weeks of initiating the trial
• Willing and able to sign the informed consent form

Exclusion Criteria:

• Patients under age 18
• Patients who are pregnant (must be ruled out in women of child-bearing age)
• Active infectious ocular or systemic disease
• Patients with active infectious ocular or extraocular disease
• Patients with history of malignancy, except for dermatologic entities of basal or squamous cell carcinoma which have been completely excised or removed previously
• Patients with systemic illness involving abnormalities of the hypothalamic-pituitary-adrenal axis; patients with primary adrenocortical insufficiency or adrenocortical hyperfunction
• Patients with known hypersensitivity to Acthar
• Patients on other non-steroidal systemic immunomodulatory medications with dose adjusted sooner than 6 weeks prior to study drug administration
• Patients currently on or recently treated with (within 6 weeks) systemic corticosteroid
• Patients with periocular or intraocular injections of medications administered to help control inflammation sooner than 6 weeks prior to study drug administration
• Patients with severe disease that warrants critical attention, deemed unsafe for the study by the investigator, or contraindicated, including but not limited to, patients with scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, or sensitivity to proteins of porcine origin. Administration of live or live attenuated vaccines is contraindicated in patients during the entire duration of the study and for one month prior

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: H.P. ACTHAR GEL	Drug: H.P. ACTHAR GEL; H.P. Acthar Gel is an adrenocorticotropic hormone (ACTH) analogue indicated for ophthalmic diseases.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of responders to Acthar at the end of treatment.	A responder will be defined by the binominal presence or absence of active retinal vasculitis as evaluated at Week 24. Active retinal vasculitis is defined as angiographic leakage from retinal arterioles, venules, or capillaries as determined by investigator, seen on wide-field angiography. Complete resolution or absence of active retinal vasculitis at Week 24 is considered success or "remission".	24 Weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Presence or absence of active retinal vasculitis at all other time points.	1 Day to 24 Weeks
Safety assessments, including drug tolerability and adverse events; other ocular and/or systemic complications.	1 Day to 24 Weeks
Percentage of patients requiring rescue therapy.	1 Day to 24 Weeks

Collaborators and Investigators

• Sponsor: Ocular Immunology and Uveitis Foundation

Study record dates

Study Major Dates

• Study Start (ACTUAL): March 22, 2017
• Primary Completion (ANTICIPATED): February 1, 2020
• Study Completion (ANTICIPATED): August 1, 2020

Study Registration Dates

• First Submitted: December 30, 2016
• First Submitted That Met QC Criteria: February 23, 2017
• First Posted (ACTUAL): March 1, 2017

Study Record Updates

• Last Update Posted (ACTUAL): September 12, 2019
• Last Update Submitted That Met QC Criteria: September 10, 2019
• Last Verified: September 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Eye Diseases; Retinal Diseases; Panuveitis; Uveal Diseases; Vasculitis; Uveitis; Uveitis, Posterior; Retinal Vasculitis
• Other Study ID Numbers: 120160924

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No