Trial Outcomes & Findings for Efficacy and Safety of H.P. Acthar Gel for the Treatment of Refractory Cutaneous Manifestations of Dermatomyositis (NCT NCT02245841)

Results Overview

Change between baseline at 6 months in modified CDASI-A (Cutaneous Dermatomyositis Disease Area and Severity Index) scores at these timepoints. The CDASI-A score ranges from 0 to 100 with higher scores reflecting more severe disease activity.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

19 participants

Primary outcome timeframe

6 months

Results posted on

2024-06-11

Participant Flow

Participant milestones

Participant milestones
Measure	H.P Acthar Gel 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Overall Study STARTED	19
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	H.P Acthar Gel n=19 Participants 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Age, Continuous	56 years n=19 Participants
Sex: Female, Male Female	14 Participants n=19 Participants
Sex: Female, Male Male	5 Participants n=19 Participants
Region of Enrollment United States	19 participants n=19 Participants
Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI-A)	19 units on a scale n=19 Participants

PRIMARY outcome

Timeframe: 6 months

Change between baseline at 6 months in modified CDASI-A (Cutaneous Dermatomyositis Disease Area and Severity Index) scores at these timepoints. The CDASI-A score ranges from 0 to 100 with higher scores reflecting more severe disease activity.

Outcome measures

Outcome measures
Measure	H.P Acthar Gel n=15 Participants 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Change From Baseline in Cutaneous Dermatomyositis at 6 Months	-9 units on a scale Interval -18.0 to -5.0

PRIMARY outcome

Timeframe: 6 months

Change from baseline to 6 months in Physician's Global Assessment (PGA) visual acuity score. Scores range from 0-10 with Higher scores reflecting severe disease activity.

Outcome measures

Outcome measures
Measure	H.P Acthar Gel n=15 Participants 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Change in Physician's Global Assessment (PGA) Visual Acuity Score From Baseline to 6 Months	-1.9 units on a scale Interval -2.5 to -1.2

SECONDARY outcome

Timeframe: 6 months

Change between baseline and 6 months in patient assessed "Global Skin Score" at these timepoints. The Global Skin Score ranges from 0 to 10 with lower 0 representing "worst sign condition imaginable" and 10 representing "perfect health".

Outcome measures

Outcome measures
Measure	H.P Acthar Gel n=15 Participants 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Change From Baseline in Patient Assessment of Dermatomyositis at 6 Months.	2 units on a scale Interval 0.5 to 6.0

SECONDARY outcome

Timeframe: 6 months

Change between baseline and 6 months in patient assessed "Global Itch Score" at these timepoints. The Global Itch score ranges from 0 to 10 with higher scores reflect more severe itching.

Outcome measures

Outcome measures
Measure	H.P Acthar Gel n=15 Participants 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Change From Baseline in Patient Global Itch Score of Dermatomyositis at 6 Months	-1 units on a scale Interval -5.0 to 1.0

SECONDARY outcome

Timeframe: 6 months

Change between baseline and 6 months in patient assessed Dermatology Life Quality Index (DLQI) scores at these timepoints. The DLQI ranges from 0 to 30 with higher scores implying more significant impact on quality of life.

Outcome measures

Outcome measures
Measure	H.P Acthar Gel n=15 Participants 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
Change From Baseline in Patient Assessment of DLQI Dermatomyositis at 6 Months	-5 units on a scale Interval -11.0 to -4.0

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Safety and tolerability of H.P. Acthar gel based on frequency and types of adverse effects.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Median/mean change in dose of systemic corticosteroids and/or steroid-sparing immunosuppressive agents from initiation to completion of study

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Median/mean change in HbA1c

Outcome measures

Outcome data not reported

Adverse Events

H.P Acthar Gel

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	H.P Acthar Gel n=19 participants at risk 80 U (1 mL) of H.P. Acthar gel via subcutaneous injection twice weekly for 24 weeks
General disorders Difficulty Sleeping	31.6% 6/19 • 6 Months Standard definition
General disorders Weight Gain	57.9% 11/19 • 6 Months Standard definition

Additional Information

Anthony Fernandez, MD, PhD

Cleveland Clinic

Phone: 216-445-8776

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place