Clinical trial: A Pilot Study of Etanercept in Dermatomyositis (NCT00112385)

Eligibility 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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 65 Years

Patient description (medical language): A 50-year-old woman comes to the clinic complaining of difficulty swallowing both liquids and solid foods, as well as occasional cough while eating.  She also has difficulty lifting her arms above her head and getting up from a chair. The weakness seems to get worse gradually. The patient has no prior medical problems and takes no medications.  Vital signs are normal.  Physical examination shows an erythematous rash on the upper eyelids. There are some red papules over joints of her hands. The rest of the physical examination is unremarkable. Antinuclear antibodies, anti-Jo-1 antibodies and anti-MDA5 antibody are positive.  Muscle biopsy shows perifascicular inflammation and atrophy of the fascicle and surrounding blood vessels.

Patient description (patient's own words): I had to go to the clinic because of difficulty swallowing all kinds of food, liquid and solid. I also sometimes cough when I eat my dinner. At only 50 year old, I had some difficulties lifting up my arms and getting up from a chair! And it's only getting worse! I never had any medical issues in the past and I'm not under any medication, I just don't get it! My vitals were normal. The physical exam showed a red rash on my upper eyelids. I also have red papules on my hands' joints. After a blood test, they told me that my antinuclear antibodies, anti-Jo-1 antibodies, and anti-MDA5 antibodies were positive.  I also did a muscle biopsy and it showed that I had an atrophy of my blood vessels around my muscle fibers and that I have an inflammation of these.

Does the patient match the trial criteria? 1