Trial Assessing Safety and Efficacy of Gammanorm® in Autoimmune Diseases

Study Overview
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Brief Summary
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This observational trial assesses the safety and efficacy of Gammanorm® in autoimmune diseases.

Official Title
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A Prospective, Multicenter Observational Trial Assessing the Safety and Efficacy of Gammanorm® in Autoimmune Diseases

Conditions
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Autoimmune Diseases

Intervention / Treatment
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* Drug: Gammanorm


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult man or woman older than 18 years, Patient with autoimmune disease such as CIDP, MMN, PM, DM, MI, ITP, NAM or any other off-label use of Gammanorm®, Patient who has a prescription of Gammanorm® treatment for immunomodulation, Patient accepting to participate in the study by oral consent after having received oral and written information on the study. Exclusion Criteria: Patient treated with Gammanorm® for immune substitution, or other authorized use Patient who refuses to participate in the study.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients Receiving Gammanorm®<br>Patients Receiving Gammanorm® | Drug: Gammanorm<br>* Patients already receiving Gammanorm will be observed over the course of 2 years every 3 months. Gammanorm will be given standard of care.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation. | Evaluate the safety of Gammanorm® in terms of adverse event rate in patients receiving the off-label treatment in immunomodulation. | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| SMOG score | For patients with idiopathic thrombocytopenic purpura, efficacy will be assessed using the SMOG score. SMOG score : Bleeding manifestations are grouped into three major domains: skin (S), visible mucosae (M), and organs (O), with gradation of severity (SMOG). Each bleeding manifestation is assessed at the time of examination. Severity is graded from 0 to 3 or 4, with grade 5 for any fatal bleeding. | 24 months | 
| Rankin score | For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the Rankin score | 24 months | 
| MRC | For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the MRC. MRC (Medical research council) scale for testing muscle : The muscle scale grades muscle power on a scale of 0 to 5 in relation to the maximum expected for that muscle (5= Normal power , 0= No contraction visible or palpable). | 24 months | 
| ONLS | For patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) and Multifocal motor neuropathy (MMN), efficacy will be assessed using the ONLS (overall neuropathy limitations score). Score ONLS (Overall Neuropathy Limitation Scale) : The OLNS focuses on upper and lower limb functions, and consists of a checklist for interviewing patients. It is scored from 0 to 5 on the upper limb section and from 0 to 7 on the lower limb section. A score of 0 indicates no limitations (the ceiling of the scale) and a score of 5 or 7 indicates no purposeful movement. | 24 months | 
| Muscular Testings and Myositis Functional Rating Scale | For patients with inflammatory myopathies including dermatomyositis (DM), polymyositis (PM), inclusion body myositis (IBM), and autoimmune necrotizing myopathy (AINM), efficacy will be assessed using the muscular testings and myositis functional rating scale | 24 months | 
| Evaluate treatment compliance | Evaluate treatment compliance in terms of number of infusions per week | 24 months | 
| Evaluate reasons for stopping the treatment | Evaluate reasons for stopping the treatment by categorizing across the study the reasons subjects stopped treatment. | 24 months | 
| Patient satisfaction regarding Gammanorm | Evaluate patient satisfaction with use of patient diaries where the patient will complete a questionnaire on Life Quality Index (LQI) | 24 months | 
| Patient Quality of Life regarding Gammanorm | Evaluate patient quality of life with use of patient diaries where the patient will complete a questionnaire on quality of life (SF-12) | 24 months |