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Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy (CIDP)

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ClinicalTrials.gov Identifier: NCT02638207
Recruitment Status : Completed
First Posted : December 23, 2015
Results First Posted : February 16, 2021
Last Update Posted : February 16, 2021
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Chronic Inflammatory Demyelinating Poly(Radiculo)Neuropathy
Intervention Drug: NewGam
Enrollment 142
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Period Title: Overall Study
Started 35 69 38
Completed 28 61 34
Not Completed 7 8 4
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam Total
Hide Arm/Group Description All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days). Total of all reporting groups
Overall Number of Baseline Participants 35 69 38 142
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 35 participants 69 participants 38 participants 142 participants
52.49  (14.449) 56.32  (14.616) 58.05  (13.764) 55.84  (14.398)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 69 participants 38 participants 142 participants
Female
13
  37.1%
31
  44.9%
14
  36.8%
58
  40.8%
Male
22
  62.9%
38
  55.1%
24
  63.2%
84
  59.2%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 35 participants 69 participants 38 participants 142 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
35
 100.0%
69
 100.0%
38
 100.0%
142
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 35 participants 69 participants 38 participants 142 participants
84.09  (16.511) 81.74  (16.333) 77.68  (75.50) 81.23  (79.00)
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 35 participants 69 participants 38 participants 142 participants
173.46  (9.506) 172.81  (8.304) 171.61  (9.030) 172.65  (8.770)
BMI  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 35 participants 69 participants 38 participants 142 participants
27.92  (4.873) 27.27  (4.588) 26.33  (5.231) 27.18  (4.837)
1.Primary Outcome
Title Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
Hide Description Efficacy - Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease of at least 1 point on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score (a scale from 0 to 10, from healthy to unable to make any purposeful movements with arms and/or legs)
Time Frame at Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 1.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 69
Measure Type: Number
Unit of Measure: proportion of subjects
.7971
2.Secondary Outcome
Title Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
Hide Description Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
Time Frame at Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Measure Type: Number
Unit of Measure: Proportion of responders
.6471 .7971 .9167
3.Secondary Outcome
Title Grip Strength Score
Hide Description Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kilopascal (kPa)
Time Frame at Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Measure Type: Number
Unit of Measure: Proportion of responders
.5588 .6522 .8333
4.Secondary Outcome
Title Inflammatory Rasch-built Overall Disability Scale (I-RODS Score)
Hide Description Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the Inflammatory Rasch-built overall disability scale (I-RODS Score) using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated
Time Frame at Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Measure Type: Number
Unit of Measure: Proportion of responders
.3824 .5507 .7222
5.Secondary Outcome
Title Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score
Hide Description Time to first confirmed worsening on the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability scale by at least 1 point from the value at baseline (Week 0)
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
There was only 1 patient with worsening (in the 1.0 g/kg group), thus an analysis of the time to first worsening was not possible.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Median (95% Confidence Interval)
Unit of Measure: days
NA [1] 
(NA to NA)
NA [2] 
(NA to NA)
NA [1] 
(NA to NA)
[1]
No patients with worsening in this group, analysis not possible.
[2]
There was only 1 patient with worsening (in the 1.0 g/kg group), thus an analysis of the time to first worsening was not possible.
6.Secondary Outcome
Title Mean Change in Grip Strength
Hide Description Mean change from baseline (Week 0) to Termination Visit in grip strength of both hands (assessed by Martin vigorimeter). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Mean (Standard Deviation)
Unit of Measure: kPa
Dominant Hand 23.91  (25.290) 19.38  (20.377) 26.06  (25.030)
Non-dominant Hand 23.94  (24.600) 17.43  (19.916) 24.53  (22.247)
7.Secondary Outcome
Title Inflammatory Rasch-built Overall Disability Scale (I-RODS)
Hide Description

Mean change from baseline (Week 0) to Termination Visit in Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported) and number of improvers. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.

A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome.

Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Mean (Standard Deviation)
Unit of Measure: score on a scale
11.38  (12.485) 10.32  (10.836) 13.86  (11.981)
8.Secondary Outcome
Title Motor Nerves
Hide Description Mean change from baseline (Week 0) to Termination Visit in sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.
Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 68 36
Mean (Standard Deviation)
Unit of Measure: mV
2.16  (6.332) 2.69  (6.688) 3.93  (9.298)
9.Secondary Outcome
Title Mean Change in Pain Intensity Numerical Rating Scale (PI-NRS Scale)
Hide Description

Mean change from baseline (Week 0) to Termination Visit in Pain Intensity Numeric Rating Scale (PI-NRS). The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point.

The Pain Intensity Numeric Rating Scale (PI-NRS, a numeric scale where 0 = no pain and 10 = worst possible pain) is an 11-point scale for patient self-reporting of pain. A higher value represents a worse outcome.

Time Frame Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Mean (Standard Deviation)
Unit of Measure: score on a scale
-2.29  (3.040) -2.19  (2.907) -2.17  (3.256)
10.Secondary Outcome
Title Worsening on the Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)
Hide Description

Time to first confirmed worsening on the I-RODS scale. The reported change was calculated from two time points as the value at the later time point minus the value at the earlier time point. A scale from 0 to 48, from "Not possible to perform" to "Possible without any difficulty". Higher values represent a better outcome.

Worsening is determined using the concept of MCID (minimum clinically important difference) using the individually obtained standard errors (MCID-SE).

Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
There was 1 patient with worsening in the 0.5 g/kg group and 2 in the 1.0 g/kg group; an analysis of the time to first worsening was not possible
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Median (95% Confidence Interval)
Unit of Measure: days
NA [1] 
(NA to NA)
NA [2] 
(NA to NA)
NA [3] 
(NA to NA)
[1]
There was only 1 patient with worsening in this group, thus an analysis of the time to first worsening was not possible.
[2]
There were only 2 patients with worsening in this group, thus an analysis of the time to first worsening was not possible.
[3]
No patients with worsening in this group, analysis not possible.
11.Secondary Outcome
Title 1 Point Decrease in the INCAT Disability Score
Hide Description Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Median (95% Confidence Interval)
Unit of Measure: days
22.0
(22.0 to 24.0)
26.0
(22.0 to 43.0)
23.0
(22.0 to 43.0)
12.Secondary Outcome
Title Decrease in Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)
Hide Description Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores
Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Intention-to-treat Set (ITTS): The ITTS consist of all patients of the Safety Data Set for whom any data was collected post infusion of IMP. Three patients (one in the 0.5 g/kg group and two in the 2.0 g/kg group) were excluded from the ITTS as no data were collected post-infusion of IMP. Therefore n=139 for the ITTS. Every treated subject will be considered in the analysis according to his randomized treatment/dose assignment.
Arm/Group Title 0.5 g/kg NewGam 1.0 g/kg NewGam 2.0 g/kg NewGam
Hide Arm/Group Description:
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (0.5g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (1.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
All patients will receive a loading dose of 2.0 g/kg Newgam (administered over two consecutive days), followed by seven infusions of the maintenance dose the patient has been randomized to (2.0g/kg NewGam), also administered over two consecutive days every 3 weeks (±4 days).
Overall Number of Participants Analyzed 34 69 36
Median (95% Confidence Interval)
Unit of Measure: days
NA [1] 
(NA to NA)
NA [2] 
(NA to NA)
NA [1] 
(NA to NA)
[1]
There were no patients with worsening in this group thus an analysis of the time to first worsening was not possible.
[2]
There was only 1 patient with worsening in this group thus an analysis of the time to first worsening was not possible.
Time Frame Up to 36 weeks
Adverse Event Reporting Description

An AE is any untoward medical occurrence in a patient receiving an IMP and which does not have to have a causal relationship with treatment.

At each visit AEs will be elicited using a standard non-leading question like "How have you been since the last visit / during the previous study period?" Patient diaries will also be checked.

Safety Data Set (SDS): The SDS was based on all randomized patients who received at least part of one infusion of IMP. Therefore n=142 for the SDS.

 
Arm/Group Title NGAM 0.5 g/kg NGAM 1.0 g/kg NGAM 2.0 g/kg Total
Hide Arm/Group Description NGAM 0.5 g/kg NGAM 1.0 g/kg NGAM 2.0 g/kg Total of all three treatment arms.
All-Cause Mortality
NGAM 0.5 g/kg NGAM 1.0 g/kg NGAM 2.0 g/kg Total
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/35 (0.00%)      1/69 (1.45%)      1/38 (2.63%)      2/142 (1.41%)    
Hide Serious Adverse Events
NGAM 0.5 g/kg NGAM 1.0 g/kg NGAM 2.0 g/kg Total
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/35 (2.86%)      4/69 (5.80%)      1/38 (2.63%)      6/142 (4.23%)    
Cardiac disorders         
Cardio-respiratory Arrest  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Ear and labyrinth disorders         
Deafness unilateral  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Gastrointestinal disorders         
Vomiting  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Infections and infestations         
Osteomyelitis  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Pneumonia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Encephalitis  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Musculoskeletal and connective tissue disorders         
Osteonecrosis  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Meningioma  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Nervous system disorders         
Headache  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Respiratory, thoracic and mediastinal disorders         
Aspiration  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
1
Term from vocabulary, MedDRA (Unspecified)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
NGAM 0.5 g/kg NGAM 1.0 g/kg NGAM 2.0 g/kg Total
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   20/35 (57.14%)      45/69 (65.22%)      24/38 (63.16%)      88/142 (61.97%)    
Blood and lymphatic system disorders         
Leukopenia  1  1/35 (2.86%)  2 5/69 (7.25%)  5 0/38 (0.00%)  0 6/142 (4.23%)  7
Anaemia  1  0/35 (0.00%)  0 2/69 (2.90%)  2 1/38 (2.63%)  2 3/142 (2.11%)  4
Thrombocytopenia  1  0/35 (0.00%)  0 2/69 (2.90%)  2 0/38 (0.00%)  0 2/142 (1.41%)  2
Eosinophilia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Lymphadenitis  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Neutropenia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Thrombocytosis  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Cardiac disorders         
Tachycardia  1  1/35 (2.86%)  1 2/69 (2.90%)  4 1/38 (2.63%)  1 4/142 (2.82%)  6
Bradycardia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Left atrial dilatation  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Left ventricular hypertrophy  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Ear and labyrinth disorders         
Tinnitus  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Eye disorders         
Conjunctival oedema  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Gastrointestinal disorders         
Nausea  1  1/35 (2.86%)  1 1/69 (1.45%)  2 3/38 (7.89%)  5 5/142 (3.52%)  8
Diarrhoea  1  0/35 (0.00%)  0 2/69 (2.90%)  2 2/38 (5.26%)  2 4/142 (2.82%)  4
Vomiting  1  0/35 (0.00%)  0 1/69 (1.45%)  2 1/38 (2.63%)  1 2/142 (1.41%)  3
Abdominal pain  1  0/35 (0.00%)  0 1/69 (1.45%)  2 0/38 (0.00%)  0 1/142 (0.70%)  2
Abdominal pain lower  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Abdominal pain upper  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Dysphagia  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Gastrooesophageal reflux disease  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Hiatus hernia  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
General disorders         
Pyrexia  1  3/35 (8.57%)  4 6/69 (8.70%)  6 2/38 (5.26%)  3 11/142 (7.75%)  13
Chills  1  3/35 (8.57%)  4 3/69 (4.35%)  3 1/38 (2.63%)  1 7/142 (4.93%)  8
Influenza like illness  1  0/35 (0.00%)  0 3/69 (4.35%)  3 0/38 (0.00%)  0 3/142 (2.11%)  3
Asthenia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 1/38 (2.63%)  1 2/142 (1.41%)  2
Chest pain  1  0/35 (0.00%)  0 1/69 (1.45%)  1 1/38 (2.63%)  1 2/142 (1.41%)  2
Catheter site pain  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Chest discomfort  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  2 1/142 (0.70%)  2
Infusion site coldness  1  0/35 (0.00%)  0 1/69 (1.45%)  2 0/38 (0.00%)  0 1/142 (0.70%)  2
Infusion site discomfort  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Oedema peripheral  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Peripheral swelling  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Hepatobiliary disorders         
Autoimmune hepatitis  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Gallbladder disorders  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Infections and infestations         
Nasopharyngitis  1  1/35 (2.86%)  1 2/69 (2.90%)  2 2/38 (5.26%)  2 5/142 (3.52%)  5
Respiratory tract infection viral  1  1/35 (2.86%)  1 2/69 (2.90%)  2 1/38 (2.63%)  1 4/142 (2.82%)  4
Urinary tract infection  1  1/35 (2.86%)  1 2/69 (2.90%)  2 1/38 (2.63%)  1 4/142 (2.82%)  4
Eczema infected  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Infective exacerbation of bronchiectasis  1  0/35 (0.00%)  0 1/69 (1.45%)  2 0/38 (0.00%)  0 1/142 (0.70%)  2
Influenza  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Pharyngitis  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Sinusitis  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Upper respiratory tract infection  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Viral pharyngitis  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Viral upper respiratory tract infection  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Injury, poisoning and procedural complications         
Contusion  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Fall  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Laceration  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Ligament injury  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Investigations         
Blood pressure increased  1  3/35 (8.57%)  7 6/69 (8.70%)  7 0/38 (0.00%)  0 9/142 (6.34%)  14
Body temperature increased  1  2/35 (5.71%)  2 4/69 (5.80%)  7 3/38 (7.89%)  3 9/142 (6.34%)  12
Blood lactate dehydrogenase increased  1  0/35 (0.00%)  0 5/69 (7.25%)  5 0/38 (0.00%)  0 5/142 (3.52%)  5
Heart rate decreased  1  0/35 (0.00%)  0 2/69 (2.90%)  2 1/38 (2.63%)  1 3/142 (2.11%)  3
Transaminases increased  1  1/35 (2.86%)  1 2/69 (2.90%)  2 0/38 (0.00%)  0 3/142 (2.11%)  3
Alanine aminotransferase increased  1  0/35 (0.00%)  0 2/69 (2.90%)  3 0/38 (0.00%)  0 2/142 (1.41%)  3
Aspartate aminotransferase increased  1  0/35 (0.00%)  0 2/69 (2.90%)  3 0/38 (0.00%)  0 2/142 (1.41%)  3
Blood uric acid increased  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Fibrin D dimer increased  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Haemoglobin decreased  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Weight decreased  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Metabolism and nutrition disorders         
Dehydration  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Diabetes mellitus inadequate control  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Dyslipidaemia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Electrolyte imbalance  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Type 2 diabetes mellitus  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Musculoskeletal and connective tissue disorders         
Back pain  1  0/35 (0.00%)  0 4/69 (5.80%)  4 2/38 (5.26%)  2 6/142 (4.23%)  6
Pain in extremity  1  1/35 (2.86%)  1 2/69 (2.90%)  2 0/38 (0.00%)  0 3/142 (2.11%)  3
Arthralgia  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Nervous system disorders         
Headache  1  1/35 (2.86%)  1 9/69 (13.04%)  11 8/38 (21.05%)  12 19/142 (13.38%)  25
Somnolence  1  2/35 (5.71%)  3 0/69 (0.00%)  0 1/38 (2.63%)  1 3/142 (2.11%)  4
Dizziness  1  1/35 (2.86%)  1 0/69 (0.00%)  0 1/38 (2.63%)  1 2/142 (1.41%)  2
Tension headache  1  0/35 (0.00%)  0 1/69 (1.45%)  4 1/38 (2.63%)  3 2/142 (1.41%)  7
Carpal tunnel syndrome  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Neuralgia  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Paraesthesia  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Restless legs syndrome  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Renal and urinary disorders         
Micturition urgency  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Respiratory, thoracic and mediastinal disorders         
Cough  1  0/35 (0.00%)  0 1/69 (1.45%)  1 1/38 (2.63%)  1 2/142 (1.41%)  2
Dyspnoea  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Tachypnoea  1  0/35 (0.00%)  0 1/69 (1.45%)  4 0/38 (0.00%)  0 1/142 (0.70%)  4
Skin and subcutaneous tissue disorders         
Dermatitis allergic  1  4/35 (11.43%)  4 5/69 (7.25%)  10 4/38 (10.53%)  5 13/142 (9.15%)  19
Skin exfoliation  1  1/35 (2.86%)  1 2/69 (2.90%)  3 1/38 (2.63%)  1 4/142 (2.82%)  5
Urticaria  1  3/35 (8.57%)  4 0/69 (0.00%)  0 0/38 (0.00%)  0 3/142 (2.11%)  4
Pruritus  1  0/35 (0.00%)  0 0/69 (0.00%)  0 2/38 (5.26%)  3 2/142 (1.41%)  3
Rash  1  0/35 (0.00%)  0 1/69 (1.45%)  1 1/38 (2.63%)  5 2/142 (1.41%)  6
Seborrhoeic dermatitis  1  1/35 (2.86%)  1 0/69 (0.00%)  0 1/38 (2.63%)  1 2/142 (1.41%)  2
Decubitis ulcer  1  0/35 (0.00%)  0 1/69 (1.45%)  1 0/38 (0.00%)  0 1/142 (0.70%)  1
Dermatitis atopic  1  0/35 (0.00%)  0 0/69 (0.00%)  0 1/38 (2.63%)  1 1/142 (0.70%)  1
Erythema  1  1/35 (2.86%)  2 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  2
Rash erythematous  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
Vascular disorders         
Hypertension  1  0/35 (0.00%)  0 3/69 (4.35%)  3 0/38 (0.00%)  0 3/142 (2.11%)  3
Hypotension  1  1/35 (2.86%)  1 0/69 (0.00%)  0 0/38 (0.00%)  0 1/142 (0.70%)  1
1
Term from vocabulary, MedDRA (Unspecified)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Mikaela Raymond
Organization: CRMG
Phone: 866-337-1868
EMail: ctgov@clinicalresearchmgt.com
Layout table for additonal information
Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT02638207    
Other Study ID Numbers: NGAM-08
First Submitted: December 16, 2015
First Posted: December 23, 2015
Results First Submitted: September 3, 2020
Results First Posted: February 16, 2021
Last Update Posted: February 16, 2021