Trial Outcomes & Findings for ACTH Gel Therapy in Rheumatoid Arthritis (NCT NCT02030028)

NCT ID: NCT02030028

Last Updated: 2023-09-06

Results Overview

percentage of participants achieving \>20% improvement in CDAI after 12 weeks of therapy (\>20% reduction in CDAI relative to baseline based on ratio of CDAI at week 12 to CDAI at week 0: CDAI Week 12/CDAI Week 0 \< 0.8 meets criteria for improvement, as lower CDAI scores represent lower disease activity)

Recruitment status

COMPLETED

Study phase

Target enrollment

18 participants

Primary outcome timeframe

12 weeks

Results posted on

2023-09-06

Participant Flow

No patients were excluded from the study based on enrollment criteria. Because ACTHAR administration was add on therapy, no wash out period was required.

Participant milestones

Participant milestones
Measure	ACTHAR Gel Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Overall Study STARTED	18
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	ACTHAR Gel Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Overall Study Withdrawal by Subject	4
Overall Study lack of transportation	1

Baseline Characteristics

ACTH Gel Therapy in Rheumatoid Arthritis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ACTHAR Gel n=18 Participants Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
CDAI (Clinical Disease Activity Index)	27 units on a scale n=5 Participants
DAS28-ESR (Disease Activity Score 28--ESR)	5.6 units on a scale STANDARD_DEVIATION 1.6 • n=5 Participants
Age, Continuous	55.3 years n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	18 participants n=5 Participants
Erythrocyte Sedimentation Rate (ESR)	37.2 mm/hr STANDARD_DEVIATION 33.8 • n=5 Participants
C-Reactive Protein (CRP)	0.42 mg/dl STANDARD_DEVIATION 0.33 • n=5 Participants
Tender Joints	6 joints n=5 Participants
Swollen Joints	6 joints n=5 Participants
Physician Global Assessment of Disease Activity	5 units on a scale n=5 Participants
Patient Global Assessment of Disease Activity	7 units on a scale n=5 Participants
DAS28-CRP (Disease Activity Score 28--CRP)	4.2 units on a scale STANDARD_DEVIATION 0.6 • n=5 Participants
fatigue severity scale--functional well-being subscale	13 units on a scale n=5 Participants
FACIT (Functional Assessment of Chronic Illness Therapy) Fatigue Scale	26 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Study completers (9/13 had evaluable data at baseline and week 12)

Outcome measures

Outcome measures
Measure	ACTHAR Gel n=9 Participants Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Changes in Clinical Disease Activity Index (CDAI) Between Week 0 and Week 12	8 Participants

SECONDARY outcome

Timeframe: 12 weeks

Population: Study completers--5/13 with evaluable data

Change in the Disease Activity Score 28-CRP (DAS28-CRP) at week 12 relative to baseline (week 0), where lower DAS28-CRP scores at week 12 represent improvement. Scale: 0-8.61 (lower scores indicate reduced disease activity)

Outcome measures

Outcome measures
Measure	ACTHAR Gel n=5 Participants Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Change in the Disease Activity Score (DAS28-CRP) Between Week 0 and Week 12	-0.87 units on a scale Standard Deviation 1.75

SECONDARY outcome

Timeframe: 12 weeks

Population: Study completers--7/13 with evaluable data

Changes in C-reactive protein value (mg/dl): reduction in CRP values at week 12 relative to week 0 represents improvement (week 12 CRP-baseline CRP \< 0))

Outcome measures

Outcome measures
Measure	ACTHAR Gel n=7 Participants Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Changes in Acute Phase Reactants (CRP) Between Week 0 and Week 12	0.36 mg/dl Standard Deviation 1.33

SECONDARY outcome

Timeframe: 12 weeks

Population: Study completers--8/13 with evaluable data

Change in ESR--reduction in ESR at week 12 relative to baseline (week 0) represents improvement (week 12 ESR - baseline ERS \< 0)

Outcome measures

Outcome measures
Measure	ACTHAR Gel n=8 Participants Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Change in Acute Phase Reactants (ESR) Between Week 0 and Week 12	-7.38 mm/hr Standard Deviation 28.30

SECONDARY outcome

Timeframe: 12 weeks

Population: Study completers--13/13 with evaluable data

The FACIT-F (Functional Assessment of Chronic Illness Therapy-Fatigue) scoring metric assesses fatigue using 13 questions rated on a Likert scale of 0 (no fatigue)-4 (severe fatigue); total scores range from 0-52, with higher total scores representing more severe fatigue. The difference in FACIT-F scores between weeks 12 and week 0 (calculated as FACIT-F score week 12 minus FACIT-F score at week 0) indicates changes in fatigue relative to baseline, where reduction of FACIT-F score at week 12 represents improvement in fatigue.

Outcome measures

Outcome measures
Measure	ACTHAR Gel n=13 Participants Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Patient Reported Changes in Fatigue Between Week 0 and Week 12	-3.8 units on a scale Interval -23.1 to 5.4

Adverse Events

ACTHAR Gel

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	ACTHAR Gel n=18 participants at risk Open label Adrenocorticotropic Hormone (ACTH) Gel for 12 weeks, 80u (1mL), given subcutaneously twice each week. ACTHAR gel: Subjects are given the ACTHAR gel subcutaneously twice per week for 12 weeks.
Musculoskeletal and connective tissue disorders rib fractures	5.6% 1/18 • Number of events 1 • Adverse events were collected over a 16 week period spanning from baseline to 16 weeks No deviation from standard adverse events definitions

Additional Information

Dr. Dana Ascherman (Principal Investigator)

University of Pittsburgh

Phone: 14123838123

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60