Clinical Trial: Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

Brief Summary: Treatment with ACTHAR Gel will result in a reduction of ocular inflammation in patients with active ocular sarcoidosis that requires systemic immunosuppressant therapy (hypothesis)

Detailed Summary:

The initial treatment of ocular sarcoidosis usually relies on a combination of topical glucocorticoids and oral glucocorticoids, both of which are associated with significant ocular and systemic toxicities. Steroid-sparing therapies are limited by variable and unpredictable efficacy, prolonged time until clinical response, medication intolerance, and difficulties obtaining payor approval. As a result, it is not uncommon that treating physicians must choose between excessive glucocorticoid toxicity versus poor control of ocular inflammation. Ongoing ocular inflammation, in turn, leads to eventual visual loss and occasionally blindness.

There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in sarcoidosis uveitis. Adrenocorticotropic hormone, through activation of melanocortin receptors on leukocytes, can dampen immune responses through non-glucocorticoid dependent mechanisms. The proposed study will aim to define whether there is effectiveness for ACTHAR gel in these patients, delineate an effect dosing regimen, and provide information about the safety of this approach for moderate to severe ocular sarcoidosis.

ACTHAR is a 39-amino acid peptide natural form of adrenocorticotropin hormone (ACTH) that was initially approved in 1952 by the FDA. It has since been approved for 19 indications including respiratory sarcoidosis, multiple sclerosis, and infantile spasms.


Sponsor: The Cleveland Clinic

Current Primary Outcome: Proportion of patients with clinically significant improvement in visual acuity [ Time Frame: Measured at 24 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Proportion of patients with clinically significant improvement in the resolution-intraocular inflammation [ Time Frame: Measured at 24 weeks ]
  • Proportion of patients experiencing a tapering of ocular/oral steroids by at least 50% [ Time Frame: Measured at 24 weeks ]
  • Proportion of patients with a clinically significant reduction-cystoid macular edema [ Time Frame: Measured at 24 weeks ]


Original Secondary Outcome: Same as current

Information By: The Cleveland Clinic

Dates:
Date Received: March 11, 2016
Date Started: March 2016
Date Completion: December 2017
Last Updated: October 25, 2016
Last Verified: October 2016