Trial Outcomes & Findings for Ocular Sarcoidosis Open Label Trial of ACTHAR Gel (NCT NCT02725177)

Results Overview

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 .

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

9 participants

Primary outcome timeframe

Measured at 24 weeks

Results posted on

2023-11-14

Participant Flow

Participant milestones

Participant milestones
Measure	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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Overall Study STARTED	9
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Overall Study Physician Decision	4
Overall Study Adverse Event	1

Baseline Characteristics

Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	H.P. Achtar Gel 80 U n=9 Participants 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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Age, Continuous	51 years n=93 Participants
Sex: Female, Male Female	6 Participants n=93 Participants
Sex: Female, Male Male	3 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	2 Participants n=93 Participants
Race (NIH/OMB) White	7 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants

PRIMARY outcome

Timeframe: 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 .

Outcome measures

Outcome measures
Measure	H.P. Achtar Gel 80 U n=9 Participants 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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Percentage of Patients With Clinically Significant Improvement in Visual Acuity	3 Participants

SECONDARY outcome

Timeframe: Measured at 24 weeks

Outcome measures

Outcome measures
Measure	H.P. Achtar Gel 80 U n=4 Participants 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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Percentage of Patients With Clinically Significant Improvement in the Resolution-intraocular Inflammation	2 Participants

SECONDARY outcome

Timeframe: Measured at 24 weeks

Outcome measures

Outcome measures
Measure	H.P. Achtar Gel 80 U n=4 Participants 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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Proportion of Patients Experiencing a Tapering of Ocular/Oral Steroids by at Least 50%	2 Participants

SECONDARY outcome

Timeframe: Measured at 24 weeks

Outcome measures

Outcome measures
Measure	H.P. Achtar Gel 80 U n=4 Participants 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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Proportion of Patients With a Clinically Significant Reduction-cystoid Macular Edema	1 Participants

Adverse Events

H.P. Achtar Gel 80 U

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	H.P. Achtar Gel 80 U n=9 participants at risk 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. 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) Repository Corticotropin Injection -Treatment Extension: 24 open label extension permitted in subjects who respond to treatment
Vascular disorders Fluid Retention	77.8% 7/9 • 24 Weeks
General disorders Insomnia	44.4% 4/9 • 24 Weeks
Vascular disorders Hypertension	44.4% 4/9 • 24 Weeks
Psychiatric disorders Depression	33.3% 3/9 • 24 Weeks
Metabolism and nutrition disorders Impaired Blood Sugar	33.3% 3/9 • 24 Weeks

Additional Information

Daniel Culver, DO

Cleveland Clinic Foundation

Phone: 216-444-6508

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place