Ocular SarcoidosisNCT02725177
Ocular Sarcoidosis Open Label Trial of ACTHAR Gel
A study testing Repository Corticotropin Injection and Repository Corticotropin Injection -Treatment Extension in people with Ocular Sarcoidosis and Panuveitis, run by The Cleveland Clinic at 1 site in United States.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Ocular Sarcoidosis, Panuveitis, Anterior Uveitis
- What is being tested?
- Repository Corticotropin Injection and Repository Corticotropin Injection -Treatment Extension
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2016to Mar 2022
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cleveland Clinical Foundation
Cleveland, Ohio, 44145
About this study
Treatment with ACTHAR Gel will result in a reduction of ocular inflammation in patients with active ocular sarcoidosis that requires systemic immunosuppressant therapy (hypothesis)
Read the 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
- Being tested
- Repository Corticotropin Injection, Repository Corticotropin Injection -Treatment Extension
- Keywords
- Ocular Sarcoidosis
- Medical subject headings
- Panuveitis, Uveitis, Anterior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Repository Corticotropin InjectionDrug
Also called H.P. Acthar Gel, ACTH Gel, ACTH
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 ExtensionDrug
Also called H.P. Acthar Gel, ACTH Gel, ACTH
24 open label extension permitted in subjects who respond to treatment
| Study group | Role | What they receive |
|---|---|---|
| H.P. Achtar Gel 80 U | Experimental | 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. Receives: Drug: Repository Corticotropin Injection; Drug: Repository Corticotropin Injection -Treatment Extension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Patients With Clinically Significant Improvement in Visual Acuity
The primary outcome was percentage 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 .
Measured: Measured at 24 weeks
Secondary outcomes (3)
Percentage of Patients With Clinically Significant Improvement in the Resolution-intraocular Inflammation
Measured: Measured at 24 weeks
Proportion of Patients Experiencing a Tapering of Ocular/Oral Steroids by at Least 50%
Measured: Measured at 24 weeks
Proportion of Patients With a Clinically Significant Reduction-cystoid Macular Edema
Measured: Measured at 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2016 (estimated)
- Study startJul 2016
- Primary completionDec 2021
- Study completionMar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Ocular SarcoidosisStudies of the same condition, open and under way.
- More trials run by The Cleveland ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Cleveland Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor
- Investigators
- Daniel A Culver, DO, Principal investigator, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02725177
- Sponsor's protocol ID
- Ocular Sarcoidosis 15-1072
- Status last verified
- Nov 2023
- First submitted
- 11 Mar 2016
- Last updated
- 14 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ocular Sarcoidosis Open Label Trial of ACTHAR Gel
- ClinicalTrials.gov ID
- NCT02725177
- Acronym / sponsor ID
- Ocular Sarcoidosis 15-1072
- Conditions
- Ocular Sarcoidosis, Panuveitis, Anterior Uveitis
- Keywords
- Ocular Sarcoidosis
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- The Cleveland ClinicUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- Jul 2016 (actual)
- Primary completion
- Dec 2021 (actual)
- Study completion
- Mar 2022 (actual)
- First posted
- 31 Mar 2016 (estimated)
- Results
- Posted 15 Feb 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| H.P. Achtar Gel 80 U | Experimental | Drug: Repository Corticotropin Injection, Drug: Repository Corticotropin Injection -Treatment ExtensionH.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. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (12 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Repository Corticotropin InjectionAlso: H.P. Acthar Gel, ACTH Gel, ACTHTreatment 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) | Drug | H.P. Achtar Gel 80 U |
| Repository Corticotropin Injection -Treatment ExtensionAlso: H.P. Acthar Gel, ACTH Gel, ACTH24 open label extension permitted in subjects who respond to treatment | Drug | H.P. Achtar Gel 80 U |
Primary outcome
Percentage of Patients With Clinically Significant Improvement in Visual Acuity
Time frame: Measured at 24 weeks
The primary outcome was percentage 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 .
3 secondary outcomes
Percentage 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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Ocular Sarcoidosis
Not yet recruitingObservational study
Early Prediction of Outcomes Following Optic Neuritis: Development and Acceptability of a Prognostic Tool
Optic Neuritis, Multiple Sclerosis
RecruitingObservational study
EACVI Study on Multimodality Cardiovascular Imaging of Inflammatory Cardiovascular Diseases
Myocarditis, ANCA Associated Vasculitis
RecruitingObservational study
Rheumatology Patient Registry and Biorepository
Rheumatic Diseases, Adult Onset Still Disease
RecruitingObservational study
Quantification & Classification of Inflammatory Cells in Uveitis Using OCT
Anterior Uveitis (AU), Birdshot Chorioretinitis
RecruitingObservational study
Swiss Pediatric Inflammatory Brain Disease Registry (Swiss-Ped-IBrainD)
Optic Neuritis, Transverse Myelitis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel A Culver, DO principal investigator
The Cleveland Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Repository Corticotropin Injection and Repository Corticotropin Injection -Treatment Extension in people with Ocular Sarcoidosis and Panuveitis, run by The Cleveland Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Feb 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Patients With Clinically Significant Improvement in Visual Acuity (time frame: Measured at 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Nov 2023.