Efficacy and Safety Study of GB-0998 for Treatment of Steroid-resistant Polymyositis and Dermatomyositis (PM/DM)

Study Overview
=================
Brief Summary
-----------------
This randomized, double-blind, placebo-controlled trial will carry out to assess the efficacy of GB-0998 in the treatment of the steroid-resistant polymyositis and dermatomyositis based on the changes in manual muscle strength (MMT) scores as primary endpoint, and in addition, to assess the safety of GB-0998.

Official Title
-----------------
A Randomized, Double-blind, Controlled Clinical Study of GB-0998 for the Steroid-resistant Polymyositis and Dermatomyositis

Conditions
-----------------
Polymyositis, Dermatomyositis

Intervention / Treatment
-----------------
* Drug: GB-0998
* Drug: Placebo of GB-0998


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patients who have been defined as definite based on the criteria of Bohan and Peter for polymyositis and dermatomyositis. Patients who are defined as steroid-resistant ones based on the changes of scores on manual muscle testing (MMT) and serum levels of creatine kinase (CK) during observation period before administration of drug. Exclusion Criteria: Patients with malignant tumors. Patients with acute interstitial pneumonia, including acute exacerbation of chronic. Patients with severe muscular atrophy for a long period. Patients with severe infectious disease. Patients who have the anamnesis of shock or hypersensitivity to this drug. Patients with severe hepatic disorder or severe renal disorder. Patients who have the anamnesis of cerebral infarction or ischemic heart disease, or who having symptom of these diseases. Patients who have been diagnosed as IgA deficiency in their past history. Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant. Patients who were administered immunoglobulin dosage within 6 weeks before consent.

Ages Eligible for Study
-----------------
Minimum Age: 16 Years
Maximum Age: 75 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: GB-0998<br>* 8 mL (400 mg of GB-0998)/kg per day is intravenously administered for five successive days<br>* Other names: Venoglobulin-IH;|
| Placebo Comparator: 2<br> | Drug: Placebo of GB-0998<br>* 8 mL/kg per day is intravenously administered for five successive days<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in manual muscle strength (MMT) scores |  | 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in the serum levels of creatine kinase (CK) and total scores of activities of daily living (ADL) |  | 8 weeks | 
| Adverse events and laboratory tests |  | 8 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
polymyositis, dermatomyositis, High-dose intravenous immunoglobulin, Venoglobulin-IH, Steroid-resistant polymyositis(PM) and dermatomyositis(DM)