Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

NCT ID: NCT02725177

Last Updated: 2023-11-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-07-31
• Study Completion Date: 2022-03-31

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 Description

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.

Conditions

Ocular Sarcoidosis; Panuveitis; Anterior Uveitis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

H.P. Achtar Gel 80 U

H.P. Acthar Gel (repository corticotropin) Injection, 80 U daily for 10 days, then 80 U twice weekly for up to a total of 24 weeks on therapy.

Group Type: EXPERIMENTAL

Repository Corticotropin Injection

Intervention Type: DRUG

Treatment with ACTHAR Gel for 24 weeks

• Initial treatment with 80 units daily for ten days (induction phase)
• Maintenance treatment with 80 units twice weekly (maintenance phase)

Repository Corticotropin Injection -Treatment Extension

Intervention Type: DRUG

24 open label extension permitted in subjects who respond to treatment

Interventions

Repository Corticotropin Injection

Treatment with ACTHAR Gel for 24 weeks

• Initial treatment with 80 units daily for ten days (induction phase)
• Maintenance treatment with 80 units twice weekly (maintenance phase)

Intervention Type: DRUG

Repository Corticotropin Injection -Treatment Extension

24 open label extension permitted in subjects who respond to treatment

Intervention Type: DRUG

Other Intervention Names

H.P. Acthar Gel; ACTH Gel; ACTH; H.P. Acthar Gel; ACTH Gel; ACTH

Eligibility Criteria

Inclusion Criteria

• Patient with sarcoidosis as defined by ATS/ERS/WASOG guidelines (American Thoracic Society/European Respiratory Society /World Association of Sarcoidosis and Other Granulomatous Diseases)
• Any posterior, intermediate or panuveitis of sufficient severity to warrant therapy, in the opinion of the treating physician --OR-- Anterior uveitis requiring 4 or more daily applications of topical corticosteroids to maintain control of inflammation, or uncontrolled with topical therapy
• Persistent disease activity (active uveitis) at the time of screening

Exclusion Criteria

• Other cause for ocular inflammation
• Uncontrolled diabetes, hypertension, or other contra-indication to increased dosage of glucocorticoids
• Recent (less than 4 weeks) intra-ocular or intra-orbital steroid injection
• Escalation of immunosuppressive medications between screening and initiation of the study medication
• Severe extra-ocular sarcoidosis likely to require additional therapy (in the opinion of the investigator)
• Administration of an investigational medication for sarcoidosis within 3 months, or 5 half-lives, whichever is longer
• Have a history of any opportunistic infection within 6 months prior to screening
• Have any history of malignancy, except fully resected cutaneous squamous cell cancer or cutaneous basal cell cancer, or cervical carcinoma in-situ with a minimum of 5 years period without recurrence
• Severe other organ disease felt to be likely to lead to death within the next six months
• Unable to follow the study protocol, including the requisite travel and follow-up ocular testing
• Women of childbearing potential must be using adequate birth control measures (abstinence, hormonal contraceptives, intrauterine device, barrier method with spermicide, or surgical sterilization) and must agree to continue such precautions, and not become pregnant or plan a pregnancy for 6 months after receiving their last treatment with study agent. Women of childbearing potential must test negative on a serum pregnancy test at screening.
• Breastfeeding women are excluded from participation

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mallinckrodt

INDUSTRY

Sponsor Role: collaborator

The Cleveland Clinic

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Daniel A Culver, DO

Role: PRINCIPAL_INVESTIGATOR

The Cleveland Clinic

Locations

Cleveland Clinical Foundation

Cleveland, Ohio, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

Ocular Sarcoidosis 15-1072

Identifier Type: -

Identifier Source: org_study_id