ACTH as A Re-emerging theRapy for Uveitis (The ACTHAR Study)

NCT02931175 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: ACTHAR-01
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 7

Brief Summary

The study aims to evaluate the potential role of ACTH gel in the management of non-infectious uveitis.

Conditions

Uveitis

Keywords

ACTH gel; Non-Infectious Uveitis; ACTHAR; Posterior Uveitis; Intermediate Uveitis; Pan Uveitis; Uveitis

Outcome Measures

Primary Outcomes (2)

• Incidence of ocular adverse events (AEs)

  The incidence and severity of ocular adverse events, as identified by eye examination.

  Month 6

• Incidence of systemic adverse events (AEs)

  The incidence and severity of systemic adverse events.

  Month 6

Secondary Outcomes (16)

• Mean change from Baseline (BL) in vitreous haze (VH)

  Month 6

• Proportion of patients achieving a ≥ 1 and ≥ 2 step improvement or resolution of VH

  Month 3, 6, 12

• Mean change from BL in graded anterior chamber cells

  Month 3, 6, 12

• Mean change from BL in Best Corrected Visual Acuity (BCVA)

  Month 3, 6, 12

• Mean change in degree of inflammation

  Month 3, 6, 12

Study Arms (2)

Group 1

EXPERIMENTAL

Mandatory twice a week (Mondays and Thursdays) treatment with SC ACTH gel 80 U/day starting at BL until month 6. Starting at month 6, the treatment will be administered on as needed basis, based on the retreatment criteria.

Drug: ACTH gel

Group 2

EXPERIMENTAL

Mandatory thrice a week (Mondays, Wednesdays, and Fridays) treatment with SC ACTH gel 80 U/day starting at BL until month 6. Starting at month 6, the treatment will be administered on as needed basis, based on the retreatment criteria.

Drug: ACTH gel

Interventions

ACTH gel DRUG

Starting at month 6, retreatment will be offered to study subjects who have demonstrated any level of response during the first 6 months and who meet any of these Retreatment Criteria listed below. Patients receiving retreatment will receive the dose that was assigned to them at randomization.

Also known as: Corticotropin

Group 1; Group 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• In order to be eligible for the study, patients will be required to meet the criteria of 1 of the 3 following disease cohorts:
• Active disease and are receiving no treatment. Active disease is defined as having at least 1+ Vitreous Haze using the Standardized Uveitis Nomenclature (SUN) Working Group scale and/or at least 1+ Vitreous Cell Count using Foster \& Vitale scale.
• Active disease and are receiving prednisone ≥10 mg/day (or equivalent dose of another corticosteroid) or at least 1 other systemic immunosuppressant (all systemic immunosuppressants other than corticosteroids will be discontinued 30 days prior to the first administration of the study drug on Day 0). Patients receiving combination of prednisone ≥10 mg/day and at least one other systemic immunosuppressant are also eligible in this category.
• Have inactive disease, defined as having \<= 0.5+ Vitreous Haze OR \<= 0.5+ Vitreous Cell Count (SUN scale), and are receiving prednisone ≥10 mg/day (or equivalent dose of another corticosteroid) or at least 1 other systemic immunosuppressant (all systemic immunosuppressants other than corticosteroids will be discontinued 30 days prior to the first administration of the study drug on Day 0). Patients receiving combination of prednisone ≥10 mg/day and at least one other systemic immunosuppressant are also eligible in this category.

You may not qualify if:

• Subjects who have any of the following at the screening visit are not eligible for enrolment in this study:
• Any significant ocular disease that could compromise vision in the study eye. These include, but are not limited to:
• Diabetic retinopathy: proliferative diabetic retinopathy (PDR) or non-proliferative diabetic retinopathy (NPDR) that compromise the vision.
• Age-related macular degeneration;
• Myopic degeneration with active subfoveal choroidal neovascularization.
• Advanced glaucoma status post trabeculectomy or tube/valve placement
• Any of the following treatments within 90 days prior to Day 0 or anticipated use of any of the following treatments to the study eye:
• Intravitreal injections (including but not limited to steroids or anti-vascular endothelial growth factors);
• Posterior subtenon's steroids.
• Intraocular surgery within 90 days prior to Day 0 in the study eye;
• Capsulotomy within 30 days prior to Day 0 in the study eye;
• Any known ocular surgery (including cataract extraction or capsulotomy) of the study eye anticipated within the first 180 days following Day 0;
• Intraocular pressure(IOP) ≥25 mmHg in the study eye (glaucoma patients maintained on no more than 2 topical medications with IOP \<25 mmHg are allowed to participate);
• Pupillary dilation inadequate for quality stereoscopic fundus photography in the study eye;
• Media opacity that would limit clinical visualization;

Sponsors & Collaborators

• Quan Dong Nguyen: lead
• Mallinckrodt: collaborator

Study Sites (7)

Retina Vitreous Associates, Medical Group

Los Angeles, California, 90017, United States

Location

Stanford University

Palo Alto, California, 94303, United States

Location

Metropolitan Eye Research and surgery Institute

Waltham, Massachusetts, 02451, United States

Location

Retina Centers Professional Corporation - RCPC

Cleveland, Ohio, 44139, United States

Location

Retina Consultants of Houston

Bellaire, Texas, 77401, United States

Location

Texas Retina Assiciates

Dallas, Texas, 75231, United States

Location

TExas Retina Associates

Dallas, Texas, 75231, United States

Location

Related Publications (4)

• Sadiq MA, Agarwal A, Hassan M, Afridi R, Sarwar S, Soliman MK, Do DV, Nguyen QD. Therapies in Development for Non-Infectious Uveitis. Curr Mol Med. 2015;15(6):565-77. doi: 10.2174/1566524015666150731103847.

  PMID: 26238367 BACKGROUND

• Montero-Melendez T. ACTH: The forgotten therapy. Semin Immunol. 2015 May;27(3):216-26. doi: 10.1016/j.smim.2015.02.003. Epub 2015 Feb 26.

  PMID: 25726511 BACKGROUND

• Durrani OM, Meads CA, Murray PI. Uveitis: a potentially blinding disease. Ophthalmologica. 2004 Jul-Aug;218(4):223-36. doi: 10.1159/000078612.

  PMID: 15258410 BACKGROUND

• Lee K, Bajwa A, Freitas-Neto CA, Metzinger JL, Wentworth BA, Foster CS. A comprehensive review and update on the non-biologic treatment of adult noninfectious uveitis: part I. Expert Opin Pharmacother. 2014 Oct;15(15):2141-54. doi: 10.1517/14656566.2014.948417.

  PMID: 25226529 BACKGROUND

MeSH Terms

Conditions

Uveitis; Uveitis, Posterior; Uveitis, Intermediate

Interventions

Adrenocorticotropic Hormone

Condition Hierarchy (Ancestors)

Uveal Diseases; Eye Diseases; Panuveitis

Intervention Hierarchy (Ancestors)

Melanocortins; Pro-Opiomelanocortin; Hypothalamic Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pituitary Hormones, Anterior; Pituitary Hormones; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Nerve Tissue Proteins; Proteins

Study Officials

• Quan D Nguyen, MD MSc

  Ocular Imaging Research and Reading Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD, MSc (USA)

Study Record Dates

• First Submitted: October 6, 2016
• First Posted: October 12, 2016
• Study Start: November 10, 2017
• Primary Completion: April 3, 2020
• Study Completion: October 28, 2021
• Last Updated: December 17, 2025

Record last verified: 2018-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)