Trial Outcomes & Findings for Acthar in Treatment of Refractory Dermatomyositis and Polymyositis (NCT NCT01906372)
NCT ID: NCT01906372
Last Updated: 2017-09-01
Results Overview
3 of any of the 6 core set measures (CSM) improved by ≥ 20%, with no more than 2 CSM worsening by ≥25% (worsening measure cannot include the MMT). The DOI should be met at least once on any of the 6 follow up visits and maintained until week 24. Subjects not meeting DOI during the trial are treatment failures.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
Primary end point: IMACS preliminary definition of improvement (DOI)
Results posted on
2017-09-01
Participant Flow
Subjects with active and refractory polymyositis or dermatomyositis were recruited at two clinical centers, The University of Pittsburgh and North Shore Long Island Jewish Center over a 16 month recruitment period. Two subjects did not reach the week 8 time point prior to withdrawing from the trial therefore additional subjects were enrolled.
If the enrolling physician planned to discontinue a specific immunosuppressive agent or glucocorticoid prior to study visit 1, a pre-defined washout period was required.
Participant milestones
| Measure |
Acthar Gel
Acthar Gel (Adrenocorticotropic Hormone Gel) 80 units (1 ml) twice a week for a period of six months.
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
Visit 7: Treatment Phase Completed
|
10
|
|
Overall Study
Visit 10: Follow up Phase Completed
|
8
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Acthar Gel
Acthar Gel (Adrenocorticotropic Hormone Gel) 80 units (1 ml) twice a week for a period of six months.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
One additional patient dropped out of the study at 6 weeks due to worsening of conduction abnormalities (heart block unrelated to the study drug), since the patient had not completed minimum 8 weeks of study drug required for outcome assessment as per study protocol, he was not included in primary analysis.
Baseline characteristics by cohort
| Measure |
Acthar Gel
n=11 Participants
Acthar Gel (Adrenocorticotropic Hormone Gel)in refractory PM and DM patients using an open label design for 6 months. Study subjects will self-administer subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week for a period of six months.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=11 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=11 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=11 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=11 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=11 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=11 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=11 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=11 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=11 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=11 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=11 Participants
|
|
Diagnosis
Dermatomyositis
|
6 Participants
n=11 Participants
|
|
Diagnosis
Polymyositis
|
5 Participants
n=11 Participants
|
|
Disease Duration
|
2.0 years
STANDARD_DEVIATION 2.0 • n=10 Participants • One additional patient dropped out of the study at 6 weeks due to worsening of conduction abnormalities (heart block unrelated to the study drug), since the patient had not completed minimum 8 weeks of study drug required for outcome assessment as per study protocol, he was not included in primary analysis.
|
|
Autoantibody Status
|
8 Participants
n=10 Participants • One additional patient dropped out of the study at 6 weeks due to worsening of conduction abnormalities (heart block unrelated to the study drug), since the patient had not completed minimum 8 weeks of study drug required for outcome assessment as per study protocol, he was not included in primary analysis.
|
PRIMARY outcome
Timeframe: Primary end point: IMACS preliminary definition of improvement (DOI)Population: Total of 10 PM/DM patients completed the study.One additional patient dropped out of the study at 6 weeks due to worsening of conduction abnormalities (heart block unrelated to the study drug).The patient had not completed minimum 8 weeks of study drug required for outcome assessment as per study protocol, and was not included in primary analysis.
3 of any of the 6 core set measures (CSM) improved by ≥ 20%, with no more than 2 CSM worsening by ≥25% (worsening measure cannot include the MMT). The DOI should be met at least once on any of the 6 follow up visits and maintained until week 24. Subjects not meeting DOI during the trial are treatment failures.
Outcome measures
| Measure |
Acthar Gel
n=10 Participants
Acthar Gel (Adrenocorticotropic Hormone Gel) in active and refractory PM and DM patients using an open label design for 6 months. Study subjects self-administered subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week subcutaneously for a period of six months.
|
|---|---|
|
Specific Aim 1: Number of Subjects Meeting IMACS Preliminary Definition of Improvement (DOI).
|
7 Participants
|
SECONDARY outcome
Timeframe: Steroid sparing effect and safety and tolerability at 24 weeks compared to baselineMean change in glucocorticoid dose (equivalent prednisone dose) at 24 weeks compared to baseline.
Outcome measures
| Measure |
Acthar Gel
n=10 Participants
Acthar Gel (Adrenocorticotropic Hormone Gel) in active and refractory PM and DM patients using an open label design for 6 months. Study subjects self-administered subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week subcutaneously for a period of six months.
|
|---|---|
|
Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients.
Follow Up 24 weeks
|
2.3 mg
Standard Deviation 3.2
|
|
Steroid-sparing Effect of H.P. Acthar Gel in Refractory Adult PM and DM Patients.
Baseline
|
18.5 mg
Standard Deviation 15.7
|
Adverse Events
Acthar Gel
Serious events: 3 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Acthar Gel
n=10 participants at risk
Acthar Gel (Adrenocorticotropic Hormone Gel)in refractory PM and DM patients using an open label design for 6 months. 10 active and refractory PM/DM patients were evaluated over a 15 month period, followed by 6 months of additional follow-up for each subject. Study subjects will self-administer subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week for a period of six months.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Avascular Necrosis
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Infections and infestations
Local infection with moderate symptoms; oral intervention indicated (e.g., antibiotic, antifungal, a
|
20.0%
2/10 • Number of events 2 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Cardiac disorders
Atrioventricular Block
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
General disorders
Non-cardiac chest pain
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
Other adverse events
| Measure |
Acthar Gel
n=10 participants at risk
Acthar Gel (Adrenocorticotropic Hormone Gel)in refractory PM and DM patients using an open label design for 6 months. 10 active and refractory PM/DM patients were evaluated over a 15 month period, followed by 6 months of additional follow-up for each subject. Study subjects will self-administer subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week for a period of six months.
|
|---|---|
|
Cardiac disorders
Sinus Tachycardia
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
General disorders
General disorders and administration site conditions
|
40.0%
4/10 • Number of events 4 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Infections and infestations
Sinusitis
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Infections and infestations
Upper Respiratory Infections
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Metabolism and nutrition disorders
Cholesterol high
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
30.0%
3/10 • Number of events 3 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Psychiatric disorders
Anxiety
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Psychiatric disorders
Agitation
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Psychiatric disorders
Insomnia
|
20.0%
2/10 • Number of events 2 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Psychiatric disorders
Depression
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Respiratory, thoracic and mediastinal disorders
Lung nodules
|
10.0%
1/10 • Number of events 1 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Skin and subcutaneous tissue disorders
Calcinosis
|
20.0%
2/10 • Number of events 2 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
|
Vascular disorders
Hypertension
|
20.0%
2/10 • Number of events 2 • Adverse events were collected during the overall duration of the study (12 months).
Adverse events with a causality of possibly, probably or definitely related to medicinal product at included in this reporting data.
|
Additional Information
Rohit Aggarwal, MD
University of Pittsburgh, Division of Rheumatology
Phone: 412-647-2840
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place