Patients receiving intravenous immunoglobulin (IVIG) therapy for primary immunodeficiency and neurologic conditions may experience adverse drug reactions (ADRs). The mechanism of the ADR is unknown. Currently, the standard practice for these patients is to change from IV to subcutaneous IG (SCIG) but because of the need of immunomodulation or patient preference, SCIG may not be an option. Data has shown that some levels of complement decrease from pre- to post-infusion of IVIG. This study is to determine if replacing this complement protein may ameliorate ADRs.
This is a single-site, pilot study conducted in the US to determine the benefit of human C1-esterase inhibitor \[recombinant\] (C1-INH-R) therapy to ameliorate ADRs in subjects receiving IVIG therapy experience ADRs post-infusion. Subjects who are currently receiving IVIG for immunodeficiency or neurologic conditions and experience ADRs will be enrolled. In part 1, subjects will continue to receive IVIG for two infusions. Complement proteins will be measured pre- and post-infusion. Quality of life and other questionnaires will be administered. In part 2, subjects will receive C1-INH-R infusion prior to receiving IVIG infusion for three infusions. Complement proteins will be measured pre- and post- infusion. Quality of life and other questionnaires will be administered.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
PREVENTION
Masking
NONE
Enrollment
20
C1-INH-R is FDA approved and indicated for the treatment of acute attacks of angioedema in adolescent and adult patients with Hereditary Angioedema (HAE) as a replacement for low levels of C1-esterase inhibitor or low function of C1-esterase inhibitor
IMMUNOe Research Centers
Centennial, Colorado, United States
The change in Modified Fatigue Severity Scale (mFSS)
Subject-rated Rasch-built 7-item modified fatigue survey scale. Scale rated 0 - 3 (3 = agree, 0 = less agree)
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Modified Fatigue Impact Scale (MFIS)
Subject-rated 24 item questionnaire measuring the impact of fatigue rated 0 - 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always)
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Migraine Disability Assessment (MIDAS)
Headache severity measurement of number of days affected after infusion
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Headache Impact Scale (HIT-6)
Subject-rated measurement of the impact headaches have on the ability to function on the job, at school, at home and in social situations. 6 item questionnaire rated from never (6 points each), rarely (8 points each), sometimes (10 points each), very often (11 points each), always (13 points each). The higher number the more the impact.
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Activities of Daily Living Sliding Scale
Subject-rated 10 point scale measuring level of activity with 1 being the worst (least) and 10 being the best (greatest)
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Activities of Daily Living Questionnaire
Calculates the number of days missed from work, school/daycare/activities, housework, and regular exercise
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Energy Sliding Scale
Subject-rated10 point scale measuring energy level with 1 being the worst (Lowest) and 10 being the best (highest)
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Infection Questionnaire
Subject-rated 7 item infection questionnaire measured from 1 to 10 with 1 being the least affected and 10 being the most affected.
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in Perceived Deficits Questionnaire - cognitive assessment
Subject-rated 20 item questionnaire measuring memory, attention and concentration rated 0 to 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always) with the lower score being least impact.
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
The change in 36 item short form survey (SF-36)
Health Survey asks 36 questions to measure functional health and well-being from the patient's point of view.
Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)
Change in the number of ADRs
Adverse reactions to infusions
Time frame: Measured at each infusion (every 3 - 4 weeks)
Change in levels of C1-INH pre- and post-infusion
Laboratory levels of C1-INH total and functional
Time frame: Measurement at each infusion (every 3 - 4 weeks)
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