A Pilot Study of Etanercept in Dermatomyositis

This study has been completed.
Sponsor:
Collaborator:
Amgen
Information provided by:
Brigham and Women's Hospital
ClinicalTrials.gov Identifier:
NCT00112385
First received: June 2, 2005
Last updated: May 23, 2011
Last verified: May 2011
Results First Received: April 25, 2011  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Condition: Dermatomyositis
Interventions: Drug: Etanercept
Drug: Placebo

  Participant Flow
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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  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

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  



  Outcome Measures
  Show All Outcome Measures

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 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: A Pilot Study of Etanercept in Dermatomyositis
Organization: Brigham and Women's Hospital
phone: 617-732-8046
e-mail: aamato@partners.org


No publications provided by Brigham and Women's Hospital

Publications automatically indexed to this study:

Responsible Party: Anthony A. Amato, MD, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT00112385     History of Changes
Obsolete Identifiers: NCT00282880
Other Study ID Numbers: 1 R01 NS049639-01A2, 1 R01 NS049639-01A2
Study First Received: June 2, 2005
Results First Received: April 25, 2011
Last Updated: May 23, 2011
Health Authority: United States: Food and Drug Administration