A Pilot Study of Etanercept in Dermatomyositis

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00112385
• Date of registration: 02/06/2005
• Prospective Registration: Yes
• Primary sponsor: Brigham and Women's Hospital
• Public title: A Pilot Study of Etanercept in Dermatomyositis
• Scientific title: A Pilot Study of Etanercept in Dermatomyositis
• Date of first enrolment: March 2006
• Target sample size: 16
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00112385
• Study type: Interventional
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Anthony A Amato, MD
• Affiliation: Brigham and Women's Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

Study subjects must meet the following criteria:

1. Meet the diagnostic criteria for DM (a-c; a,b,d; or a,c,d)

1. Subjects must have symmetric proximal greater than distal weakness

2. Characteristic DM rash consisting of any or all of the following: heliotrope,
shawl sign, V-sign, Gottron's sign, Gottron's papules, periungual telangiectasias

3. Laboratory evidence of myopathy with at least one of the following: an elevated
serum CK or aldolase level, myositis-specific antibody, electromyography (EMG)
demonstrating myopathic features (e.g., muscle membrane instability, myopathic
units, or early recruitment), or an abnormal skeletal muscle MRI showing diffuse
or patchy edema within the muscles.

4. A muscle biopsy will be optional if the patient fulfills criteria a-c. The
subject must demonstrate symmetric proximal weakness (criteria a) for entry into
the study. If the subject does not have a definite rash (criteria b) or
laboratory evidence of a myopathy (criteria c), a muscle biopsy will be required.
The muscle biopsy must demonstrate one of the following: perifascicular atrophy,
expression of MHC 1 on perifascicular muscle fibers, MAC deposition on small
blood vessels, tubuloreticular inclusions in endothelial cells on EM, or MXA
expression on muscle fibers of blood vessels

2. Newly diagnosed subjects should be able to walk independently 30 feet (cane, walkers,
orthoses allowed). However, subjects with refractory dermatomyositis may be
non-ambulatory.

3. Age > 18 years

4. Patients must not use topical skin ointments for treatment of the dermatological
manifestations as it will interfere with skin assessment.

5. Men and women of childbearing age must be willing to use a method of birth control.

6. Able to give informed consent

7. Subject or designee must have the ability to self-inject investigational product or
have a care giver at home who can administer subcutaneous injections

Exclusion Criteria

The presence of any of the following excludes subject participation in the study:

1. Presence of any one of the following medical conditions: active infection,
uncontrolled diabetes mellitus, MI, CVA or TIA within 3 months of screening visit,
symptomatic cardiomyopathy (congestive heart failure), symptomatic coronary artery
disease, uncontrolled hypertension (sitting systolic BP <80 mm Hg or > 160 or
diastolic BP > 100 mm Hg), oxygen-dependent severe pulmonary disease, systemic lupus
erythematosus (SLE), cancer (other than basal cell skin cancer) less than 5 years
previously, HIV or other immunosuppressing disease, positive PPD test or any history
of mycobacterial disease, chronic hepatitis B or hepatitis C, history of multiple
sclerosis, transverse myelitis, optic neuritis, chronic inflammatory demyelinating
neuropathy, epilepsy, or other chronic serious medical illnesses

2. Presence of any of the following on routine blood screening: WBC<3000, Platelets <
100,000, hematocrit < 30%, BUN > 30 mg %, symptomatic liver disease with serum albumin
< 3 G/DL, PT or PTT > upper range of control values

3. Forced Vital Capacity < 50% of predicted

4. History of non-compliance with other therapies

5. Any prior or concurrent cyclophosphamide, or current use of any immunosuppressive
agent besides methotrexate (e.g., azathioprine, mycophenolate, or cyclosporine)

6. Coexistence of other neuromuscular disease that may complicate interpretation of the
results of the study

7. Drug or alcohol abuse within last 3 months

8. Pregnancy or breast feeding

9. Juvenile DM

10. Subjects who have known hypersensitivity to Enbrel or any of its components or who is
known to have antibodies to etanercept

11. Use of a live vaccine 90 days prior to, or during this study.

12. Subject is currently enrolled in another investigational device or drug trial(s), or
subject has received other investigational agent(s) within 28 days of baseline visit.

13. Concurrent sulfasalazine therapy



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Dermatomyositis

Intervention(s)

Drug: Placebo

Drug: Etanercept

Primary Outcome(s)

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

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]

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]

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]

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]

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]

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]

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

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]

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]

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

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]

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]

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]

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

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]

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

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

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

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]

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

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

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]

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]

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]

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]

Secondary Outcome(s)

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

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

Secondary ID(s)

1R01NS049639-01A2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Amgen

Results

• Results available: Yes
• Date Posted: 21/06/2011
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00112385