Open Label Study of Acthar SQ Gel Injection in Patients With Active Anterior Uveitis

• Sponsor: Washington University School of Medicine (Other)
• Overall Status: Terminated
• CT.gov ID: NCT02769702
• Collaborator: Mallinckrodt (Industry)
• Enrollment: 2
• Location: 1
• Arm: 1
• Duration (Months): 7.2
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

Uveitis is an acute or chronic inflammatory condition of unknown etiology. Although uveitis often responds adequately to topical corticosteroids, there are many patients for which this treatment is either inadequate or not tolerated. A patient with inadequate response to treatment would manifest uveitis activity by slit lamp examination determination of anterior chamber cellularity. Lack of tolerance of therapy commonly manifests as ocular hypertension (greater than 21 mmHg measured by tonometry)complicating chronic topical corticosteroid administration, leading to glaucoma and permanent visual loss. Moreover, systemic corticosteroids may be required at a dose unsafe for chronic administration. In these situations, an immunosuppressive medication is often added as a "steroid-sparing" agent. If and when there is clinical response to the added immunosuppressive, the oral and/or topical corticosteroid dose can be reduced or eliminated to avoid toxicity.

There are several reasons for believing that Acthar might be beneficial in the treatment of uveitis patients. In addition to increasing adrenal production or cortisol, Acthar has another important mechanisms of action mediated by its binding of melanocortin receptors. Melanocortin down-regulates activity of B and T lymphocytes, monocytes and macrophages. In animal studies, melanocortin peptides down-regulate T helper cells, up-regulate T Regulatory cells, and decrease B lymphocyte production of B Lymphocyte Stimulator. In macrophages, there is down-regulation of IL-1, IL-2, INF gamma, TNF alpha, nitric oxide and adhesion molecules. In other cells, in addition to IL-10 upregulation (monocytes), there is down-regulation of VACM and ECAM (endothelial cells), prostaglandins (fibroblasts) and MCP-1 and RANTES (renal tubules).CNS mediation of systemic inflammation may also be down-regulated by melanocortin receptor binding by Acthar.

Condition or Disease	Intervention/Treatment	Phase
Uveitis	Drug: Acthar	Phase 4

Detailed Description

Patients eligible for the study will be identified by the investigators and given a copy of the consent form to read. Patients interested in enrolling will be screened and if eligible will be enrolled in the study. The screening visit and the baseline visit may occur on the same day. Patients enrolling will be instructed in self-administering Acthar 80 IU twice a week. Patients will keep an injection log which will be inspected at each visit (4, 8, and 12 weeks). Ophthalmology data will be collected at each visit and recorded on the case report form by the ophthalmology technician working with the sub-investigator at the time of each visit. Patients will come to the Center for Clinical Studies each visit for blood draw, blood pressure recording, and recording of adverse events.

Study Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Prospective Open Label Study of Acthar SQ Gel Injection in Patients With Active Anterior Uveitis Who Are Not Well Controlled With, or Intolerant of, Topical or Systemic Corticosteroids
• Study Start Date: Jun 1, 2016
• Actual Primary Completion Date: Jan 6, 2017
• Actual Study Completion Date: Jan 6, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Intervention Acthar 80 IU SC twice w eek	Drug: Acthar; Acthar 80 IU SC twice a week; Other Names: Acthar Gel

Outcome Measures

Primary Outcome Measures

1. Change From Baseline in Eye With Uveitis of Anterior Chamber Cellularity Graded From 0-4 on a Likert Scale Determined by Slit Lamp Examination [Baseline and 12 weeks]

   standard assessment of uveitis activity. Scores were assessed using a Likert scale using 0 to 4, higher score reflects more cellularity.

Secondary Outcome Measures

1. Change in Baseline in Eye With Uveitis of Anterior Chamber Protein Graded 0-4 on a Likert Scale Determined by Slit Lamp Evaluation [Baseline and 12 weeks]

   standard assessment of uveitis activity. Scores were assessed using a Likert scale using 0 to 4, higher score reflects more protein.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Active anterior uveitis requiring oral and/or topical corticosteroid therapy

Exclusion Criteria:

• Uncontrolled diabetes

• Uncontrolled glaucoma

• HIV infection or other infection for which corticosteroid therapy contraindicated

• Contraindication to ACTHAR

• Scleroderma

• Osteoporosis

• Ocular herpes simplex

• Systemic fungal infection

• Recent surgery

• Uncontrolled hypertension

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Washington University in St. Louis	Saint Louis	Missouri	United States	63110

Sponsors and Collaborators

• Washington University School of Medicine
• Mallinckrodt

Investigators

• Principal Investigator: Richard D Brasington, MD, Washington U Rheumatology

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Washington University School of Medicine
• ClinicalTrials.gov Identifier: NCT02769702
• Other Study ID Numbers: Acthar Uveitis Study
• First Posted: May 12, 2016
• Last Update Posted: Oct 1, 2018
• Last Verified: Sep 1, 2018

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Washington University School of Medicine: Uveitis
• Additional relevant MeSH terms: Uveitis; Uveitis, Anterior; Iridocyclitis; Adrenocorticotropic Hormone

Study Results

Participant Flow

Arm/Group Title	Acthar 80 IU
Arm/Group Description	Acthar 80 IU SC twice week
Period Title: Overall Study
STARTED	2
COMPLETED	2
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Acthar 80 IU
Arm/Group Description	Acthar 80 IU SC twice week
Overall Participants	2
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	2 100%
>=65 years	0 0%
Sex: Female, Male (Count of Participants)
Female	2 100%
Male	0 0%

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in Eye With Uveitis of Anterior Chamber Cellularity Graded From 0-4 on a Likert Scale Determined by Slit Lamp Examination
Description	standard assessment of uveitis activity. Scores were assessed using a Likert scale using 0 to 4, higher score reflects more cellularity.
Time Frame	Baseline and 12 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Acthar 80 IU
Arm/Group Description	Acthar 80 IU SC twice week
Measure Participants	2
Baseline	0.5 (0.70)
12 Weeks	1.25 (1.06)

2. Secondary Outcome

Title	Change in Baseline in Eye With Uveitis of Anterior Chamber Protein Graded 0-4 on a Likert Scale Determined by Slit Lamp Evaluation
Description	standard assessment of uveitis activity. Scores were assessed using a Likert scale using 0 to 4, higher score reflects more protein.
Time Frame	Baseline and 12 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Acthar 80 IU
Arm/Group Description	Acthar 80 IU SC twice week
Measure Participants	2
Baseline	NA (NA)
12 Weeks	1.0 (1.41)

Adverse Events

Arm/Group Title	Acthar 80 IU
Arm/Group Description	Acthar 80 IU SC twice week
All Cause Mortality
	Acthar 80 IU
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Acthar 80 IU
	Affected / at Risk (%)	# Events
Total	0/2 (0%)
Other (Not Including Serious) Adverse Events
	Acthar 80 IU
	Affected / at Risk (%)	# Events
Total	0/2 (0%)

Limitations/Caveats

This study was prematurely terminated, only 2 participants enrolled. Although results are reported they should be interpreted with caution due to small sample size.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.