Clinical trial NCT02465359: Subcutaneous Immunoglobulin for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Official title: A Study of Subcutaneous Immunoglobulin as Chronic Treatment for Patients With Chronic Inflammatory Demyelinating Polyneuropathy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Chronic Inflammatory Demyelinating Polyneuropathy
Lead sponsor: University of South Florida
Sponsor class: OTHER
Collaborators: name: CSL Behring
First posted: 2015-06-08
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: The investigators are using self administered subcutaneous immunoglobulin (SCIG) in patients with CIDP who require Intravenous immunoglobulin (IVIG). Safety, efficacy, and patient satisfaction will be examined.

Detailed description: 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 intravenous (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 Immunoglobulin A (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.

Eligibility criteria: 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 activities of daily living (ADL) or work,
* Paresthesia of sufficient severity to require a medication,
* Sensory impairment,
* Walking impairment,

AND requires IVIG to control symptoms.

Exclusion Criteria:

1. Thrombocytopenia or other bleeding disorders,
2. Anticoagulation therapy,
3. Severe or anaphylactoid reactions to IVIG,
4. Cancer,
5. Pregnancy,
6. Breast-feeding,
7. Renal insufficiency or failure,
8. Congestive heart failure,
9. Psychiatric illness.

Primary outcomes:
measure: Relapse of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Symptoms
timeFrame: 24 weeks
Secondary outcomes:
measure: Mean Change in Short Form 36 (SF-36) Domain: Physical Functioning Between Week 24 and Screening
timeFrame: 24 weeks
measure: Mean Change in Inflammatory Rasch-built Overall Disability Scale (I-RODS) From Screening to Week 24
timeFrame: 24 weeks
measure: Mean Change in Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) Between Week 24 and Screening
timeFrame: 24 weeks
measure: Treatment Satisfaction Questionnaire for Medication (TSQM)
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain: Role Limitations-physical Between Screening and Week 24
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain: Role Limitations - Emotional (Between Week 24 and Screening)
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain: Energy/Fatigue Between Screening and Week 24
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain: Emotional Well-being Between Screening and 24 Weeks
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain: Social Functioning Between Screening and Week 24
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain: Pain Between Screening and 24 Weeks
timeFrame: 24 weeks
measure: Mean Change in Short Form 36 Domain "General Health" Between Screening and Week 24
timeFrame: 24 weeks
measure: Mean Change in Limb Motor Strength Testing (LMST) Over 24 Weeks
timeFrame: 24 weeks
measure: Mean Change in Timed 25-foot Walk (T25-FW) Between Screening and Week 24
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Tampa
country: United States
facility: USF Dept of Neurology
state: Florida
Study officials:
affiliation: University of South Florida
name: Tuan Vu, MD
role: PRINCIPAL_INVESTIGATOR
References: