A Pilot Study of Etanercept in Dermatomyositis

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
153 patients were screened and we enrolled and randomized 16 subjects (F 10, M 6) from until February 2006 to May 2009. Of these, 5 were new DM patients and 11 were refractory dermatomyositis patients. Etanercept: 11 subjects (New 3, Refractory 8) Placebo: 5 subjects (New 2, Refractory 3)

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Most patient were initially ineligible because they were not new dermatomyositis patients. Due to slow recruitment, we modified the protocol. We allowed enrollment of refractory dermatomyositis patients receiving prednisone for greater than 2 months. These subjects could also receive second line agents, as long as they were on a stable dose.

Reporting Groups

	Description
Etanercept	Subjects randomized to Etanercept will be provided with syringes contain 50 mg and will be injected SQ once a week for 52 wks
Placebo	Subjects will be given syringes containing placebo

Participant Flow:   Overall Study

	Etanercept	Placebo
STARTED	11	5
COMPLETED	10	4
NOT COMPLETED	1	1
Lost to Follow-up	1	0
Withdrawal by Subject	0	1

Reporting Groups

	Description
Etanercept	Subjects randomized to Etanercept will be provided with syringes contain 50 mg and will be injected SQ once a week for 52 wks
Placebo	Subjects will be given syringes containing placebo
Total	Total of all reporting groups

Baseline Measures

	Etanercept	Placebo	Total
Number of Participants   [units: participants]	11	5	16
Age   [units: participants]
< 18 years	0	0	0
Between 18 and 65 years	11	5	16
>=65 years	0	0	0
Age   [units: years] Mean ± Standard Deviation	43.36  ± 14.8	44.2  ± 11.9	43.6  ± 13.39
Gender   [units: participants]
Female	6	4	10
Male	5	1	6
Region of Enrollment   [units: participants]
Canada	0	1	1
United States	11	4	15

1.  Primary:

Occurrence of at Least One Adverse Event   [ Time Frame: at each visit during the 12 month study ]

2.  Primary:

Tolerability   [ Time Frame: At any point between Baseline (week 0) and the end of the study (Week 52) ]

3.  Primary:

Average Change in Oral Temperature From Baseline to Week 52   [ Time Frame: At Baseline (Week 0) and Week 52 ]

4.  Primary:

Average Change in Respiration Rate From Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

5.  Primary:

Average Change in Systolic Blood Pressure From Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

6.  Primary:

Average Change in Diastolic Blood Pressure Comparing Baseline to Week 52.   [ Time Frame: At Baseline (Week0) and Week 52 ]

7.  Primary:

Average Change in Pulse Comparing Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

8.  Primary:

Average Change in Body Weight in Kilograms (kg) Comparing Baseline to Week 52.   [ Time Frame: At Baseline (Week0) and Week 52 ]

9.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Creatine Kinase (CK) Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

10.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Alanine Aminotransferase (ALT) Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

11.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Gamma-glutamyl Transpeptidase (GGT) Laboratory Values From Baseline to Week 52   [ Time Frame: Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

12.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Aldolase Laboratory Values From Baseline to Week 52   [ Time Frame: Screening, Baseline (Week0), Week 4, 8,12, 16, 20, 24, 32, 40, and 52 ]

13.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Glucose Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

14.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Potassium Laboratory Values From Baseline to Week 52   [ Time Frame: Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

15.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal White Blood Cell Count (WBC) Values From Baseline to Week 52   [ Time Frame: Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

16.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Hemoglobin Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

17.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Hematocrit Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

18.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Platelet Counts From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

19.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Urine Leukocyte Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

20.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Urine Protein Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

21.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Urine Glucose Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

22.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Urine Ketone Laboratory Values From Baseline to Week 52   [ Time Frame: At Screening, Baseline (Week0), Week 4, 8, 12, 16, 20, 24, 32, 40, and 52 ]

23.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Serum 25-hydroxyvitamin D (25-OH VitD) Laboratory Values From the Screening Visit to Week 52   [ Time Frame: Screening visit and Week 52 ]

24.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Antinuclear Antibody Test (ANA) Values From the Screening Visit to Week 52   [ Time Frame: At Screening, Week 12, 24, 40, and 52 ]

25.  Primary:

Frequency of Subjects With Treatment Emergent, Clinically Significant, Abnormal Monoclonal Protein Detection by Serum Protein Electrophoresis (SPEP) From the Screening Visit to Week 52   [ Time Frame: Screening visit, Week 12, 24, 40, and 52 ]

26.  Primary:

Average Cumulative Dosage of Prednisone Over the One Year Study Period   [ Time Frame: Baseline until week 52 ]

27.  Secondary:

Average Prednisone Dosage After Week 24   [ Time Frame: from week 24 to 52 ]

28.  Secondary:

Average Daily Dose of Prednisone From Baseline to Week 52   [ Time Frame: Baseline through Week 52 ]

29.  Other Pre-specified:

The Number of Participants Who Were Classified as Treatment Failures   [ Time Frame: At any point during the 52 week study ]

30.  Other Pre-specified:

Change in the Average Manual Muscle Testing (MMT) Score From Baseline to Week 52   [ Time Frame: At Baseline (Week 0) and Week 52 ]

31.  Other Pre-specified:

Average Change in Time to Rise From a Chair From Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

32.  Other Pre-specified:

Average Change in Time (Seconds) to Walk 30 Feet Comparing Performance at Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

33.  Other Pre-specified:

Average Change in Z-score for Dual-emission X-ray Absorptiometry (DEXA) of the Femur From the Screening Visit to Week 52   [ Time Frame: Screening and Week 52 ]

34.  Other Pre-specified:

Average Change in Z-score for Dual-emission X-ray Absorptiometry (DEXA) of the Lumbar Spine From the Screening Visit to Week 52   [ Time Frame: Screening visit and Week 52. ]

35.  Other Pre-specified:

Average Change in Physician Global Activity Assessment From Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

36.  Other Pre-specified:

Average Change in Patient Global Activity Assessment Score From Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

37.  Other Pre-specified:

Average Change in Cutaneous Disease Activity and Severity Index (CDASI) Score From Week 52 to Baseline   [ Time Frame: At Baseline (Week0) and Week 52 ]

38.  Other Pre-specified:

Change in Pruritis Rating From Baseline to Week 52   [ Time Frame: At Baseline (Week0), and Week 52 ]

39.  Other Pre-specified:

Change in Health Assessment Questionnaire (HAQ) Score From Baseline to Week 52   [ Time Frame: At Baseline (Week0) and Week 52 ]

40.  Other Pre-specified:

Forced Vital Capacity (FVC) Average Change in Percent Predicted From Screening to Week 52.   [ Time Frame: Screening Visit and Week 52. ]

41.  Other Pre-specified:

Average Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) From the Screening Visit to Week 52   [ Time Frame: Screening Visit and Week 52 ]

42.  Other Pre-specified:

Average Change in Percent Predicted Diffusion Capacity (DLCO)From the Screening Visit to Week 52   [ Time Frame: Screening visit and Week 52 ]