search
Back to results

Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

Primary Purpose

Ocular Sarcoidosis, Panuveitis, Anterior Uveitis

Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Repository Corticotropin Injection
Repository Corticotropin Injection -Treatment Extension
Sponsored by
The Cleveland Clinic
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Ocular Sarcoidosis focused on measuring Ocular Sarcoidosis

Eligibility Criteria

18 Years - 99 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Cleveland Clinical Foundation

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

H.P. Achtar Gel 80 U

Arm Description

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.

Outcomes

Primary Outcome Measures

Proportion of Patients With Clinically Significant Improvement in Visual Acuity
The primary outcome was proportion of patients meeting at least one of the following variables 1) improved visual acuity, 2) resolution of intraocular inflammation, 3) ability to taper ocular or oral steroids by at least 50% or 4) reduction of cystoid macular edema, with no worsening of any single one and no need for additional sarcoidosis therapies at 24 weeks .

Secondary Outcome Measures

Proportion of Patients With Clinically Significant Improvement in the Resolution-intraocular Inflammation
Proportion of Patients Experiencing a Tapering of Ocular/Oral Steroids by at Least 50%
Proportion of Patients With a Clinically Significant Reduction-cystoid Macular Edema

Full Information

First Posted
March 11, 2016
Last Updated
January 20, 2023
Sponsor
The Cleveland Clinic
Collaborators
Mallinckrodt
search

1. Study Identification

Unique Protocol Identification Number
NCT02725177
Brief Title
Ocular Sarcoidosis Open Label Trial of ACTHAR Gel
Official Title
Ocular Sarcoidosis Open Label Trial of ACTHAR Gel
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Completed
Study Start Date
July 2016 (Actual)
Primary Completion Date
December 2021 (Actual)
Study Completion Date
March 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
The Cleveland Clinic
Collaborators
Mallinckrodt

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ocular Sarcoidosis, Panuveitis, Anterior Uveitis
Keywords
Ocular Sarcoidosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
H.P. Achtar Gel 80 U
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Repository Corticotropin Injection
Other Intervention Name(s)
H.P. Acthar Gel, ACTH Gel, ACTH
Intervention Description
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
Intervention Name(s)
Repository Corticotropin Injection -Treatment Extension
Other Intervention Name(s)
H.P. Acthar Gel, ACTH Gel, ACTH
Intervention Description
24 open label extension permitted in subjects who respond to treatment
Primary Outcome Measure Information:
Title
Proportion of Patients With Clinically Significant Improvement in Visual Acuity
Description
The primary outcome was proportion of patients meeting at least one of the following variables 1) improved visual acuity, 2) resolution of intraocular inflammation, 3) ability to taper ocular or oral steroids by at least 50% or 4) reduction of cystoid macular edema, with no worsening of any single one and no need for additional sarcoidosis therapies at 24 weeks .
Time Frame
Measured at 24 weeks
Secondary Outcome Measure Information:
Title
Proportion of Patients With Clinically Significant Improvement in the Resolution-intraocular Inflammation
Time Frame
Measured at 24 weeks
Title
Proportion of Patients Experiencing a Tapering of Ocular/Oral Steroids by at Least 50%
Time Frame
Measured at 24 weeks
Title
Proportion of Patients With a Clinically Significant Reduction-cystoid Macular Edema
Time Frame
Measured at 24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Daniel A Culver, DO
Organizational Affiliation
The Cleveland Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cleveland Clinical Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44145
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

We'll reach out to this number within 24 hrs