Trial Outcomes & Findings for Immune Globulin Intravenous (IGIV) For Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (NCT NCT00220740)
NCT ID: NCT00220740
Last Updated: 2016-03-23
Results Overview
The primary efficacy objective was the comparison of IGIV-C and Placebo group Responder rates. An Efficacy Period Responder was defined as a subject with ≥ 1 point improvement in the adjusted Inflammatory Neuropathy Case And Treatment (INCAT) score, with the improvement maintained through the end of Week 24 in the Efficacy Period. Measurements are reported in INCAT scale of 0-5 in both lower and upper extremities, for a total score of 0 to 10. INCAT scores for arm disability: 0 = no upper limb problems; 5 = inability to use either arm for any purposeful movement. INCAT scores for leg disability: 0= walking not affected; 5 = restricted to wheelchair, unable to stand and walk a few steps with help
COMPLETED
PHASE3
117 participants
6 months
2016-03-23
Participant Flow
The study was conducted at 31 study centers in USA, Poland, Argentina, Czech Republic, Canada, Mexico, Italy, Israel, Germany, and Serbia.
Participant milestones
| Measure |
IGIV-C
2 g/kg loading dose, followed by 1 g/kg maintenance dose
|
Placebo
.1% albumin; 2g/kg loading dose and 1 g/kg maintenance dose
|
|---|---|---|
|
Efficacy Period
STARTED
|
59
|
58
|
|
Efficacy Period
COMPLETED
|
33
|
12
|
|
Efficacy Period
NOT COMPLETED
|
26
|
46
|
|
Rescue Treatment
STARTED
|
45
|
23
|
|
Rescue Treatment
COMPLETED
|
26
|
5
|
|
Rescue Treatment
NOT COMPLETED
|
19
|
18
|
|
Withdrawal Period
STARTED
|
43
|
31
|
|
Withdrawal Period
COMPLETED
|
37
|
16
|
|
Withdrawal Period
NOT COMPLETED
|
6
|
15
|
Reasons for withdrawal
| Measure |
IGIV-C
2 g/kg loading dose, followed by 1 g/kg maintenance dose
|
Placebo
.1% albumin; 2g/kg loading dose and 1 g/kg maintenance dose
|
|---|---|---|
|
Efficacy Period
Entered rescue treatment with placebo
|
23
|
0
|
|
Efficacy Period
Adverse Event
|
1
|
1
|
|
Efficacy Period
Withdrawal by Subject
|
1
|
0
|
|
Efficacy Period
Protocol Violation
|
1
|
0
|
|
Efficacy Period
Entered Rescue Treatment with IGIV-C
|
0
|
45
|
Baseline Characteristics
Immune Globulin Intravenous (IGIV) For Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Baseline characteristics by cohort
| Measure |
IGIV-C
n=59 Participants
2 g/kg loading dose, followed by 1 g/kg maintenance dose
|
Placebo
n=58 Participants
.1% albumin, 2 g/kg loading dose, followed by 1 g/kg maintenance dose
|
Total
n=117 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
44 Participants
n=5 Participants
|
40 Participants
n=7 Participants
|
84 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
|
Age, Continuous
|
49.9 years
STANDARD_DEVIATION 17.31 • n=5 Participants
|
53.3 years
STANDARD_DEVIATION 15.63 • n=7 Participants
|
51.6 years
STANDARD_DEVIATION 16.51 • n=5 Participants
|
|
Sex: Female, Male
Female
|
28 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
31 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
77 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
2 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Czech Republic
|
7 participants
n=5 Participants
|
6 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Region of Enrollment
Mexico
|
3 participants
n=5 Participants
|
5 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Argentina
|
8 participants
n=5 Participants
|
7 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
15 participants
n=5 Participants
|
15 participants
n=7 Participants
|
30 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
8 participants
n=5 Participants
|
7 participants
n=7 Participants
|
15 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=5 Participants
|
3 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Macedonia, The Former Yugoslav Republic of
|
5 participants
n=5 Participants
|
6 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
Baseline INCAT Score
Total Overall Disability Score
|
4.2 units on a scale
STANDARD_DEVIATION 1.4 • n=5 Participants
|
4.1 units on a scale
STANDARD_DEVIATION 1.5 • n=7 Participants
|
4.2 units on a scale
STANDARD_DEVIATION 1.4 • n=5 Participants
|
|
Baseline INCAT Score
Upper Extremity Disability Score
|
2.3 units on a scale
STANDARD_DEVIATION 1.0 • n=5 Participants
|
2.1 units on a scale
STANDARD_DEVIATION 1.0 • n=7 Participants
|
2.2 units on a scale
STANDARD_DEVIATION 1.0 • n=5 Participants
|
|
Baseline INCAT Score
Lower Extremity Disability Score
|
1.9 units on a scale
STANDARD_DEVIATION 0.8 • n=5 Participants
|
1.9 units on a scale
STANDARD_DEVIATION 1.1 • n=7 Participants
|
1.9 units on a scale
STANDARD_DEVIATION 0.9 • n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: The Intent-to-Treat Population was defined as all randomized subjects. This population was the primary efficacy population to be analyzed.
The primary efficacy objective was the comparison of IGIV-C and Placebo group Responder rates. An Efficacy Period Responder was defined as a subject with ≥ 1 point improvement in the adjusted Inflammatory Neuropathy Case And Treatment (INCAT) score, with the improvement maintained through the end of Week 24 in the Efficacy Period. Measurements are reported in INCAT scale of 0-5 in both lower and upper extremities, for a total score of 0 to 10. INCAT scores for arm disability: 0 = no upper limb problems; 5 = inability to use either arm for any purposeful movement. INCAT scores for leg disability: 0= walking not affected; 5 = restricted to wheelchair, unable to stand and walk a few steps with help
Outcome measures
| Measure |
IGIV-C
n=59 Participants
|
Placebo
n=58 Participants
|
|---|---|---|
|
Comparison of the Responder Rates Between Two Treatment Groups in the Efficacy Period
|
54.2 percentage of responders
|
20.7 percentage of responders
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Intent-to-treat
Mean changes in amplitude \[mV\] measured at most proximal site in the most severely affected motor nerve from baseline to endpoint during the Efficacy Period (Intent to treat population)
Outcome measures
| Measure |
IGIV-C
n=59 Participants
|
Placebo
n=58 Participants
|
|---|---|---|
|
Mean Change in the Amplitude (Millivolts) in the Most Severely Affected Motor Nerve During the Efficacy Period
|
0.69 Millivolts
Standard Deviation 1.856
|
0.47 Millivolts
Standard Deviation 2.291
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: Intent-to-treat
Outcome measures
| Measure |
IGIV-C
n=59 Participants
|
Placebo
n=58 Participants
|
|---|---|---|
|
Mean Change in Grip Strength During the Efficacy Period
Dominant hand (IGIV-C n=57, Placebo n=58)
|
13.175 kilopascal
Standard Deviation 19.2935
|
1.489 kilopascal
Standard Deviation 15.5742
|
|
Mean Change in Grip Strength During the Efficacy Period
Non-dominant hand (IGIV-C n=58, Placebo n=58)
|
13.316 kilopascal
Standard Deviation 17.3526
|
4.261 kilopascal
Standard Deviation 14.8959
|
SECONDARY outcome
Timeframe: 6 monthsPopulation: In the Randomized Withdrawal Period, 43 subjects were randomized to IGIV-C, but only 31 out of the 41 subjects were prior IGIV-C responders or rescue successes. Similarly, 31 subjects were randomized to Placebo, but only 26 out of the 31 subjects were prior IGIV-C responders or rescue successes.
Outcome measures
| Measure |
IGIV-C
n=31 Participants
|
Placebo
n=26 Participants
|
|---|---|---|
|
Time to Relapse for Subjects Who Were IGIV-C Responders or IGIV-C Rescue Successes, During the Randomized Withdrawal Period
|
21.89 weeks
Standard Deviation 6.181
|
16.56 weeks
Standard Deviation 8.543
|
Adverse Events
IGIV-C
Placebo
Serious adverse events
| Measure |
IGIV-C
n=113 participants at risk
2 g/kg loading dose, followed by 1 g/kg maintenance dose.
A total of 113 subjects were exposed to IGIV-C across all three treatments/periods.
|
Placebo
n=95 participants at risk
.1% albumin; 2g/kg loading dose and 1 g/kg maintenance dose.
A total of 95 subjects were exposed to Placebo across all three treatments/periods.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
Nervous system disorders
Dizziness
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
Cardiac disorders
Palpitations
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
General disorders
Pyrexia
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
Nervous system disorders
Headache
|
1.8%
2/113 • Number of events 2
|
0.00%
0/95
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
Gastrointestinal disorders
Vomiting
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
Infections and infestations
Bronchopneumonia
|
0.88%
1/113 • Number of events 1
|
0.00%
0/95
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Nervous system disorders
Demyelinating polyneuropathy
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Investigations
Medical observation (for asthma)
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Vascular disorders
Deep vein thrombosis
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
|
Nervous system disorders
CIDP (relapse)
|
0.00%
0/113
|
1.1%
1/95 • Number of events 1
|
Other adverse events
| Measure |
IGIV-C
n=113 participants at risk
2 g/kg loading dose, followed by 1 g/kg maintenance dose.
A total of 113 subjects were exposed to IGIV-C across all three treatments/periods.
|
Placebo
n=95 participants at risk
.1% albumin; 2g/kg loading dose and 1 g/kg maintenance dose.
A total of 95 subjects were exposed to Placebo across all three treatments/periods.
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
6.2%
7/113 • Number of events 9
|
3.2%
3/95 • Number of events 3
|
|
General disorders
Pyrexia
|
12.4%
14/113 • Number of events 26
|
0.00%
0/95
|
|
General disorders
Asthenia
|
8.0%
9/113 • Number of events 10
|
3.2%
3/95 • Number of events 4
|
|
General disorders
Chills
|
8.0%
9/113 • Number of events 10
|
0.00%
0/95
|
|
Infections and infestations
Influenza
|
5.3%
6/113 • Number of events 6
|
2.1%
2/95 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.1%
8/113 • Number of events 9
|
3.2%
3/95 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.1%
8/113 • Number of events 11
|
1.1%
1/95 • Number of events 1
|
|
Nervous system disorders
Headache
|
30.1%
34/113 • Number of events 55
|
8.4%
8/95 • Number of events 15
|
|
Nervous system disorders
Dizziness
|
5.3%
6/113 • Number of events 6
|
1.1%
1/95 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.1%
8/113 • Number of events 13
|
1.1%
1/95 • Number of events 1
|
|
Vascular disorders
Hypertension
|
8.8%
10/113 • Number of events 20
|
4.2%
4/95 • Number of events 6
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator must send a draft manuscript of the publication or abstract to the sponsor thirty days in advance of submission in order to obtain approval prior to submission of the final version for publication. This will be reviewed promptly and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the investigator(s), the contents of the publication will be discussed in order to find a solution that satisfies both parties.
- Publication restrictions are in place
Restriction type: OTHER