Subcutaneous Immunoglobulin for CIDP

Study Overview
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Brief Summary
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The investigators are using self administered subcutaneous IG in patients with CIDP who require IVIG. Safety, efficacy, and patient satisfaction will be examined.

Detailed Description
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Chronic inflammatory demyelinating polyneuropathy (CIDP) is an autoimmune neurological disorder that causes limb weakness and numbness. Many patients require immunosuppressants and plasma exchange (PLEX) to control their symptoms. Intravenous immunoglobulin (IVIG)is also an effective treatment (Hughes et al, 2006 & 2008; Hughes, 2009; Cocito et al, 2010), and the American Academy of Neurology (AAN) guideline recommended that it should be offered in the long-term treatment of CIDP (Patwa et al, 2012). While effective, IVIG causes systemic side effects in about 5% of patients. These side effects include rash, pruritus, myalgia, fever, chills, headache, low back pain, nausea, vomiting, changes in blood pressure or heart rate, renal failure, and aseptic meningitis (Berger, 2008). For many patients who are chronically treated with IVIG, venous access may be a problem over time. An alternative is the subcutaneous (SC) route, which has been in use since 1980 for primary immune deficiency disorders and is the treatment of choice for this condition in Scandinavia and England (Radinsky et al, 2003). As compared to IV route, SC route maintains higher trough levels of immunoglobulins, increases patient independence, reduces systemic side-effects, and is better tolerated in those who are pregnant or sensitized to IgA (Radinsky et al, 2003). In a review of side effects associated with 33,168 SCIG infusions, no severe or anaphylactoid reactions occurred (Gardulf et al, 1995). Patients can self-administer medication, and hence, overall cost may be reduced. A retrospective study of 28 children with primary immunodeficiency in Canada showed that the mean difference in costs between IVIG and SCIG during the study period (1 year on IVIG and 1 year on SCIG) was $4,346 in favor of SCIG (Ducruet et al, 2011). A US$10,100 reduction in cost per year per patient associated with SCIG use was also reported by Gardulf et al (1995) in Sweden. Disadvantages of SCIG include more frequent infusions and local reactions at sites of infusion (transient swelling, soreness, redness, induration, local heat, and itching) in about 1% of patients.

Official Title
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A Study of Subcutaneous Immunoglobulin as Chronic Treatment for Patients With Chronic Inflammatory Demyelinating Polyneuropathy

Conditions
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Chronic Inflammatory Demyelinating Polyneuropathy

Intervention / Treatment
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* Drug: Immune Globulin Subcutaneous (Human)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: To qualify, a patient must have CIDP and persistence of significant symptoms (having 2 or more of the following): Weakness in any limb, Motor fatigue significant to interfere with ADL or work, Paresthesia of sufficient severity to require a medication, Sensory impairment, Walking impairment, AND requires IVIG to control symptoms. Exclusion Criteria: Thrombocytopenia or other bleeding disorders, Anticoagulation therapy, Severe or anaphylactoid reactions to IVIG, Cancer, Pregnancy, Breast-feeding, Renal insufficiency or failure, Congestive heart failure, Psychiatric illness.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 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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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Immune globulin subcutaneous (Human)<br>lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly | Drug: Immune Globulin Subcutaneous (Human)<br>* Patients who have CIDP and are on IVIG will be allowed in the study to try subcutaneous immune Globulin (SCIG) as part of an open label study.<br>* Other names: Hizentra;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Relapse of CIDP Symptoms | This is defined as a 20% decrease in force (as detected on Hand-Held Dynamometry (HHD)) in greater that 50% of the muscles tested compared to baseline values | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Short Form 36 | This questionnaire evaluates the patient's health status | Monthly for six months | 
| Rasch-built Overall Disability Scale | The Rasch-built Overall Disability Scale (R-ODS) is an instrument answered by the patient to assess overall disability | Monthly for 6 months | 
| CIP-PRO20 | The CIP-PRO20 is used to evaluate quality of life in patients with polyneuropathy | Monthly for 6 months | 
| Treatment Satisfaction Questionnaire for Medication | The TSQM is the Treatment Satisfaction Questionnaire for Medication will be used to assess the patient's satisfaction with IVIg treatment compared to the use of SCIg treatment | 2-6 weeks prior to Day 1 of treatment and then monthly for 6 months |