Trial Outcomes & Findings for Subcutaneous Immunoglobulin for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (NCT NCT02465359)

Last Updated: 2024-10-08

Results Overview

This outcome measure, referred to as the relapse of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) symptoms, is a measure of how many participants experiences CIDP symptoms causing them to withdraw prematurely from the study. This is a count of participant withdrawals compared to the number of participants who completed the study.

Recruitment status

COMPLETED

Study phase

Target enrollment

15 participants

Primary outcome timeframe

24 weeks

Results posted on

2024-10-08

Participant Flow

Participant milestones

Participant milestones
Measure	Immune Globulin Subcutaneous (Human) lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): Patients who have CIDP and are on intravenous immunoglobulin (IVIG) will be allowed in the study to try subcutaneous immune Globulin (SCIG) as part of an open label study.
Overall Study STARTED	15
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Immune Globulin Subcutaneous (Human) lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): Patients who have CIDP and are on intravenous immunoglobulin (IVIG) will be allowed in the study to try subcutaneous immune Globulin (SCIG) as part of an open label study.
Overall Study Lack of Efficacy	1
Overall Study Withdrawal by Subject	1
Overall Study Adverse Event	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Age, Categorical <=18 years	0 Participants n=15 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=15 Participants
Age, Categorical >=65 years	1 Participants n=15 Participants
Sex: Female, Male Female	7 Participants n=15 Participants
Sex: Female, Male Male	8 Participants n=15 Participants
Region of Enrollment United States	15 participants n=15 Participants
Duration of IVIg use before SCIg (months)	25.8 Months STANDARD_DEVIATION 24.01 • n=15 Participants

PRIMARY outcome

Timeframe: 24 weeks

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Relapse of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Symptoms withdrew prematurely	3 Participants
Relapse of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) Symptoms completed study	12 Participants

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to physical functioning is shown, listed as the mean change from screening to 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 (SF-36) Domain: Physical Functioning Between Week 24 and Screening	0 score on a scale Interval -0.12 to 0.12

SECONDARY outcome

Timeframe: 24 weeks

The Inflammatory Rasch-built Overall Disability Scale (I-RODS) is an instrument answered by the patient to assess overall disability through assessing activity and social participation limitations in patients with inflammatory neuropathies. It is a 24-item scale, with each item representing a common, daily activity. The patient assigns a score between 0 and 2 to each item as follows: 0 = impossible to perform 1. = performed with difficulty 2. = easily performed The total raw score on the I-RODS (0-48 max) is transformed to a final score (0-100), with a lower score indicating worse outcomes. Here the outcome measure is the mean change between screening and week 24, as calculated from transformed total I-RODS score. A negative change between week 24 and screening, or a reduction in I-RODS score, indicates a worsening of the patient's perceived daily functioning, as measured by the I-RODS.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=14 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Inflammatory Rasch-built Overall Disability Scale (I-RODS) From Screening to Week 24	-1.5 units on a scale Interval -4.6 to 1.6

SECONDARY outcome

Timeframe: 24 weeks

The Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) is used to evaluate quality of life in patients with polyneuropathy. The scale consists of 15 items, resulting in a single-score with 4 life domains (physical, social, pain and emotional well-being). Each item assessed is scored 0-2 (0= not at all, 1 = a little bit, 2 = a lot). Max score possible is 30 with a higher score indicating worse outcomes. This outcome measure was evaluated at screening and week 24 by determining the mean change between these two time points. A negative score would indicate an improvement as perceived by the participant.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI) Between Week 24 and Screening	-4.3 units on a scale Interval -7.7 to -0.9

SECONDARY outcome

Timeframe: 24 weeks

The Treatment Satisfaction Questionnaire for Medication (TSQM) is used to assess the patient's treatment satisfaction with intravenous immune globulin (IVIg) compared to the use of subcutaneous immune globulin (SCIg). There are 14 items and the TSQM addresses four domains: effectiveness, side effects, convenience, and global satisfaction. Questions are scored from one (least satisfied) to five or seven (most satisfied), with the exception of item 4 relating to side effects (scored yes/no, 1 or 0, respectively). Item scores are summed to give four domain scores, which are in turn transformed to a scale of 0-100. Higher scores indicate higher treatment satisfaction.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=11 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Treatment Satisfaction Questionnaire for Medication (TSQM)	10.3 units on a scale Interval 4.3 to 16.2

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "role limitations-physical" is shown, listed as the mean change between screening to 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain: Role Limitations-physical Between Screening and Week 24	0.24 score on a scale Interval 0.03 to 0.46

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) measure health status of patients. Per Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "role limitations-emotional" is shown, listed as the mean change from screening to 24 weeks. A negative value for this measure indicates a worsening of role limitations related to emotional health.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain: Role Limitations - Emotional (Between Week 24 and Screening)	-0.01 score on a scale Interval -0.27 to 0.24

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "energy/fatigue" is shown, listed as the mean change between screening and 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain: Energy/Fatigue Between Screening and Week 24	0.09 score on a scale Interval 0.0 to 0.18

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "emotional well-being" is shown, listed as the mean change between screening and 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain: Emotional Well-being Between Screening and 24 Weeks	0.03 score on a scale Interval -0.04 to 0.09

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "social functioning" is shown, listed as the mean change between screening and 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain: Social Functioning Between Screening and Week 24	0.06 score on a scale Interval -0.07 to 0.19

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "pain" is shown, listed as the mean change between screening and 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain: Pain Between Screening and 24 Weeks	0.08 score on a scale Interval 0.0 to 0.16

SECONDARY outcome

Timeframe: 24 weeks

The short form 36 (SF-36) is a health status profile designed to measure health status and outcomes of patients. Per the paper from Burholt and Nash (2011) scoring and domains are as follows:" The SF-36 is a 36 item scale, which measures eight domains of health status: physical functioning (10 items); physical role limitations (four items); bodily pain (two items); general health perceptions (five items); energy/vitality (four items); social functioning (two items); emotional role limitations (three items) and mental health (five items). A scoring algorithm is used to convert the raw scores into the eight dimensions listed above. The scores are transformed to range from zero where the respondent has the worst possible health to 100 where the respondent is in the best possible health." For this particular outcome measure, only the domain pertaining to "general health" is shown, listed as the mean change between screening and 24 weeks.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Short Form 36 Domain "General Health" Between Screening and Week 24	0.01 score on a scale Interval -0.07 to 0.1

SECONDARY outcome

Timeframe: 24 weeks

LMST is a discrete variable that tests muscle strength in upper and lower limbs. It is a sum of the strength of 16 muscle groups tested at a given time point. LMST is measured and reported in pounds (lbs). For this outcome measure, the mean change, in pounds, between screening and week 24 LMST is reported.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Limb Motor Strength Testing (LMST) Over 24 Weeks	5.6 pounds Interval 2.3 to 8.9

SECONDARY outcome

Timeframe: 24 weeks

The T25-FW is a quantitative mobility and leg function performance test based on a timed 25-foot walk. A lower time indicates greater mobility and leg performance, thus suggesting better outcomes. Time is measured in seconds. This outcome is the mean change between timed 25 foot walk times between screening and week 24.

Outcome measures

Outcome measures
Measure	Immune Globulin Subcutaneous (Human) n=15 Participants lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
Mean Change in Timed 25-foot Walk (T25-FW) Between Screening and Week 24	-1.0 seconds Interval -2.9 to 0.8

Adverse Events

Immune Globulin Subcutaneous (Human)

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Immune Globulin Subcutaneous (Human) n=15 participants at risk lmmune Globulin Subcutaneous(Human) 20% Liquid (Hizentra) will be given weekly Immune Globulin Subcutaneous (Human): 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.
General disorders Pain at infusion site	86.7% 13/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Transient swelling at site of infusion	80.0% 12/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Redness at site of infusion	73.3% 11/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Induration at site of infusion	46.7% 7/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Itching at site of infusion	46.7% 7/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Numbness	33.3% 5/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Bruising at site of infusion	33.3% 5/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Increased Exhaustion	26.7% 4/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Nervous system disorders Hedache/Migrane	26.7% 4/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Skin and subcutaneous tissue disorders Local heat at site of infusion	20.0% 3/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Musculoskeletal and connective tissue disorders Increased weakness	20.0% 3/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Skin and subcutaneous tissue disorders Tingling sensation	20.0% 3/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Skin and subcutaneous tissue disorders Burning sensation at site of infusion	20.0% 3/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Fever	13.3% 2/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Musculoskeletal and connective tissue disorders Pain in the extremities	13.3% 2/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Fell/fainted	13.3% 2/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Skin and subcutaneous tissue disorders Generalized itching	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Gastrointestinal disorders Bloating	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Stabbing Pain	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Musculoskeletal and connective tissue disorders Backache	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Chills	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Musculoskeletal and connective tissue disorders Twitching	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Gastrointestinal disorders Constipation	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Nervous system disorders CIDP exacerbation	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Heavy legs	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Bleeding at site of infusion	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Respiratory, thoracic and mediastinal disorders Cough	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Musculoskeletal and connective tissue disorders Stiffness	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Eye disorders Eyes burning	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
General disorders Shingles	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Musculoskeletal and connective tissue disorders Severe hip osteoarthritis	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.
Psychiatric disorders Depression	6.7% 1/15 • 24 Weeks Definitions do not differ to ClinicalTrials.gov adverse event definitions.

Additional Information

Tuan H. Vu, MD

University of South Florida

Phone: 813-974-9413

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place