Trial Outcomes & Findings for Treatment of CNS Sarcoidosis With H.P. Acthar Gel (NCT NCT02298491)

Last Updated: 2026-04-17

Results Overview

The Karnofsky Performance Scale score allows patients to be classified as to their functional impairment. This can be used to compare the effectiveness of different therapies and to assess the prognosis in individual patients. The scale ranges from 0 to 100. Higher scores are associated with better outcomes.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

4 participants

Primary outcome timeframe

12 months (intention to treat)

Results posted on

2026-04-17

Participant Flow

Participant milestones

Participant milestones
Measure	H.P. Acthar Gel H.P. Acthar Gel: 80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 12
Overall Study STARTED	4
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

4 subjects were enrolled; 3 subjects completed the assessments

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	H.P. Acthar Gel n=4 Participants H.P. Acthar Gel: 80 IU subcutaneously daily for 10 days then followed by 80 IU subcutaneously three times per week through Month 12
Age, Categorical <=18 years	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=4 Participants
Age, Continuous	46.3 years n=4 Participants
Sex: Female, Male Female	3 Participants n=4 Participants
Sex: Female, Male Male	1 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	4 Participants n=4 Participants
Race (NIH/OMB) White	0 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=4 Participants
Region of Enrollment United States	4 participants n=4 Participants
Karnofsky Performance Status (KPS) score	76.67 units on a scale STANDARD_DEVIATION 15.28 • n=3 Participants • 4 subjects were enrolled; 3 subjects completed the assessments

PRIMARY outcome

Timeframe: 12 months (intention to treat)

Population: The population consisted of individuals with neurosarcoidosis before and after treatment with Acthar Gel

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=3 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment n=3 Participants Study patient after treatment with Acthar Gel
Karnofsky Performance Status (KPS) Score	76.67 score on a scale Standard Deviation 15.28	76.67 score on a scale Standard Deviation 5.77

PRIMARY outcome

Timeframe: 1 year

Change in the total number of lesions assessed at 1 year

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=3 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment Study patient after treatment with Acthar Gel
Change in Total Number of New Lesions	4 lesions	—

SECONDARY outcome

Timeframe: 12 months (intention to treat)

Population: The analysis population consists of all patients pre- and post-treatment with Acthar Gel

A patient-reported measure of disability which consists of a scale with 9 levels of disability ranging from 0 to 8 (higher scores are associated with worse disability).

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=3 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment n=3 Participants Study patient after treatment with Acthar Gel
Patient-Determined Disease Steps (PDDS)	3 score on a scale Standard Deviation 2.65	2.67 score on a scale Standard Deviation 2.31

SECONDARY outcome

Timeframe: 12 months

Population: All patient pre- and post-treatment (intention to treat)

The MoCA is a 30-point test that screens for cognitive impairment. Scores range from 0 to 30, with higher scores associated with a better outcome.

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=3 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment n=3 Participants Study patient after treatment with Acthar Gel
Montreal Cognitive Assessment (MoCA)	26.33 score on a scale Standard Deviation 3.79	26.33 score on a scale Standard Deviation 3.51

SECONDARY outcome

Timeframe: 12 months (intention to treat)

Population: Study patient pre- and post-treatment with Acthar Gel

Assesses cognitive function. The patient is given a set of symbols, and each symbol is paired with a number. The patient is given 90 seconds to write down the number that corresponds to each symbol.Scores range from 0 to 110. A higher score is associated with a better outcome.

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=2 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment n=2 Participants Study patient after treatment with Acthar Gel
Symbol-Digit Modalities Test (SDMT)	34.5 score on a scale Standard Deviation 20.51	58 score on a scale Standard Deviation 19.80

SECONDARY outcome

Timeframe: 12 months (intention to treat)

Population: Patients pre-treatment and post-treatment with Acthar Gel

The WPAI-GH consists of six questions: 1 = currently employed; 2 = hours missed due to health problems; 3 = hours missed for other reasons; 4 = hours actually worked; 5 = degree that the patient's health affected productivity while working (using a 0 to 10 Visual Analogue Scale (VAS)); 6 = degree health affected productivity in regular unpaid activities (VAS). The result for question 5 is used for the outcome measure, for which a higher number is associated with a worse outcome.

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=3 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment n=3 Participants Study patient after treatment with Acthar Gel
Work Productivity and Activities Impairment -General Health (WPAI-GH)	2.33 score on a scale Standard Deviation 2.52	3.33 score on a scale Standard Deviation 2.89

SECONDARY outcome

Timeframe: 12 months (intent to treat)

Population: The analysis population consists of patients before and after each patient was treated with Acthar Gel.

The Beck Depression Inventory measures the severity of depression in adolescents and adults. It consists of 21 subscales, and the score for each individual subscale is added to give a total score of between 0 to 63. Total scores \<= 10 are considered normal. Scores \> 10 are considered abnormal, with higher scores above that cutoff being associated with a worse outcome.

Outcome measures

Outcome measures
Measure	H.P. Acthar Gel Pre-treatment n=3 Participants Study patient before treatment with Acthar Gel	H.P. Acthar Gel Post-treatment n=3 Participants Study patient after treatment with Acthar Gel
Beck Depression Inventory	6 score on a scale Standard Deviation 2.65	5 score on a scale Standard Deviation 2

Adverse Events

H.P. Acthar Gel

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Walter Royal, III, MD

Morehouse School of Medicine

Phone: 404-756-5222

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place