Safety and Effectiveness of Acthar Gel for Inflammation of the Eye's Uvea (Middle Layer)

A Multicenter, Open Label Pilot Study to Explore the Efficacy and Safety of Acthar Gel in Subjects With Severe Noninfectious Intermediate Uveitis, Posterior Uveitis, or Panuveitis (NIPPU)

The main reason for this study is to see if Acthar Gel can reduce inflammation in the uvea.

Also, safety information when using it for this purpose will be collected.

Study Overview

• Status: Terminated
• Conditions: Panuveitis; Uveitis, Posterior; Uveitis, Intermediate
• Intervention / Treatment: Drug: Acthar Gel

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Florida
    • Clearwater, Florida, United States, 33761
      • Blue Ocean Clinical Research
  • Indiana
    • Indianapolis, Indiana, United States, 46290
      • Midwest Eye Institute
  • Massachusetts
    • Waltham, Massachusetts, United States, 02451
      • Massachusetts Eye Research and Surgery Institution (MERSI)
  • New Jersey
    • Palisades Park, New Jersey, United States, 07650
      • Metropolitan Eye Research and Surgery Institute
  • North Dakota
    • Fargo, North Dakota, United States, 58103
      • Bergstrom Eye Research, LLC
  • Texas
    • McAllen, Texas, United States, 78503
      • Valley Retina Institute, PA
    • San Antonio, Texas, United States, 78240
      • Foresight Studies, LLC
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Virginia Eye Consultants

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:

• Is male or nonpregnant, nonlactating female
• Has been diagnosed with current severe NIPPU

• Has active disease at the Baseline Visit as defined by the presence of at least 1 of the following parameters in at least one eye despite at least 2 weeks of maintenance therapy with oral prednisone (or oral corticosteroid equivalent):

  1. Has active, inflammatory, chorioretinal and/or inflammatory retinal vascular lesion
  2. Has ≥ 2+ anterior chamber cells [Standardization of Uveitis Nomenclature (SUN) criteria]
  3. Has ≥ 1.5+ vitreous haze
• Is willing to taper current doses of corticosteroid and immunomodulatory therapy to the minimum effective dose during the study.
• If under treatment with any immunosuppressants, immunomodulators, or biologic agents for a comorbid condition, has been on a stable dose for 2 weeks before screening

Exclusion criteria:

• Has proliferative or severe nonproliferative diabetic retinopathy, clinically significant macular edema due to diabetic retinopathy, neovascular/wet age-related macular degeneration, abnormality of vitreoretinal interface with the potential for macular structural damage independent of the inflammatory process or severe vitreous haze that precludes visualization of the fundus at the Baseline Visit
• Has Type 1 or Type 2 diabetes mellitus, tuberculosis, history of hepatitis, peptic ulcer, active infection, or any contraindication for treatment with Acthar Gel

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
Other: Acthar Gel; Participants received Acthar 1 mL (80 units [U]) subcutaneously (SC) 2 times per week for 36 weeks followed by a taper to Acthar 1 mL (80 U) SC once a week for 2 weeks, then 0.5 mL (40 U) SC once a week for 2 weeks.	Drug: Acthar Gel; Acthar gel for subcutaneous injection 80 units per mL (40 units per 0.5 mL); Other Names: Corticotropin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Score on the Nussenblatt Grading Scale	The Nussenblatt grading scale uses photographic measurement of cloudy, declining vision (vitreous haze). The scale goes from 0 to 4, and lower scores are better.	Week 36
Score on the Laser Flare Scale	Protein that escapes from blood vessels inside the eye make a sudden burst of scattered light (flare) when a laser shines on them. This is called a laser flare. It is used to measure the amount and size of proteins in the clear fluid in the front of the eyeball (aqueous humour). The larger the flare, the more inflammation is inside the eye (intraocular inflammation) in patients with uveitis. Laser flare is scored on a scale from 0 (no visible flare when compared with the normal eye) to 3 (severe - very dense flare). Lower scores are better.	Week 36
Score on the Aqueous Cell Scale	The number of cells (like white blood cells) floating in the aqueous humour (aqueous cells) are measured on a scale from 0 (none) to 4 (more than 30 cells), and lower scores are better.	Week 36

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Diurnal Intraocular Pressure (IOP)	Eye pressure is measured in millimeters of mercury (mmHg). Normal eye pressure ranges from 12-22 mmHg, and eye pressure greater than 22 mmHg is considered higher than normal. During a 24-hour period, IOP normally varies by 2 to 6 mmHg because of different amounts of secretion and drainage at different times of the day. The word diurnal means "around the day," and IOP means "pressure inside the eye", so diurnal IOP is the measure of pressure inside the eye during a 24-hour period. Diurnal IOP is measured in millimeters of mercury (mmHg) using Goldmann Applanation Tonometry (GAT). Lower scores are better.	Week 36

Collaborators and Investigators

• Sponsor: Mallinckrodt
• Investigators: Study Director: Clinical Team Lead, Mallinckrodt

Study record dates

Study Major Dates

• Study Start (Actual): October 5, 2018
• Primary Completion (Actual): July 14, 2020
• Study Completion (Actual): July 14, 2020

Study Registration Dates

• First Submitted: August 21, 2018
• First Submitted That Met QC Criteria: August 30, 2018
• First Posted (Actual): September 4, 2018

Study Record Updates

• Last Update Posted (Actual): August 3, 2021
• Last Update Submitted That Met QC Criteria: July 12, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Uveal Diseases; Choroid Diseases; Choroiditis; Uveitis; Uveitis, Posterior; Panuveitis; Uveitis, Intermediate; Pars Planitis; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Adrenocorticotropic Hormone
• Other Study ID Numbers: MNK61074105

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No
• IPD Plan Description: Discussion of statistical endpoints and analysis are included in manuscripts. Summary aggregate (basic) results (including adverse events information) and the study protocol are made available on clinicaltrials.gov (NCT03656692) when required by regulation. Individual de-identified patient data will not be disclosed. Requests for additional information should be directed to the company at medinfo@mnk.com.

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.: Yes