Trial Outcomes & Findings for Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy (NCT NCT02638207)

Last Updated: 2021-02-16

Results Overview

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)

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

142 participants

Primary outcome timeframe

at Week 24

Results posted on

2021-02-16

Participant Flow

Participant milestones

Participant milestones
Measure	0.5 g/kg NewGam 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).	1.0 g/kg NewGam 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).	2.0 g/kg NewGam 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 Study STARTED	35	69	38
Overall Study COMPLETED	28	61	34
Overall Study NOT COMPLETED	7	8	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	0.5 g/kg NewGam n=35 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=38 Participants 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 n=142 Participants Total of all reporting groups
Age, Continuous	52.49 years STANDARD_DEVIATION 14.449 • n=99 Participants	56.32 years STANDARD_DEVIATION 14.616 • n=107 Participants	58.05 years STANDARD_DEVIATION 13.764 • n=206 Participants	55.84 years STANDARD_DEVIATION 14.398 • n=7 Participants
Sex: Female, Male Female	13 Participants n=99 Participants	31 Participants n=107 Participants	14 Participants n=206 Participants	58 Participants n=7 Participants
Sex: Female, Male Male	22 Participants n=99 Participants	38 Participants n=107 Participants	24 Participants n=206 Participants	84 Participants n=7 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) White	35 Participants n=99 Participants	69 Participants n=107 Participants	38 Participants n=206 Participants	142 Participants n=7 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Weight	84.09 kg STANDARD_DEVIATION 16.511 • n=99 Participants	81.74 kg STANDARD_DEVIATION 16.333 • n=107 Participants	77.68 kg STANDARD_DEVIATION 75.50 • n=206 Participants	81.23 kg STANDARD_DEVIATION 79.00 • n=7 Participants
Height	173.46 cm STANDARD_DEVIATION 9.506 • n=99 Participants	172.81 cm STANDARD_DEVIATION 8.304 • n=107 Participants	171.61 cm STANDARD_DEVIATION 9.030 • n=206 Participants	172.65 cm STANDARD_DEVIATION 8.770 • n=7 Participants
BMI	27.92 kg/m^2 STANDARD_DEVIATION 4.873 • n=99 Participants	27.27 kg/m^2 STANDARD_DEVIATION 4.588 • n=107 Participants	26.33 kg/m^2 STANDARD_DEVIATION 5.231 • n=206 Participants	27.18 kg/m^2 STANDARD_DEVIATION 4.837 • n=7 Participants

PRIMARY outcome

Timeframe: at Week 24

Population: 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.

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=69 Participants 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).	1.0 g/kg NewGam 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).	2.0 g/kg NewGam 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).
Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score	.7971 proportion of subjects	—	—

SECONDARY outcome

Timeframe: at Week 24

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

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Decrease in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score	.6471 Proportion of responders	.7971 Proportion of responders	.9167 Proportion of responders

SECONDARY outcome

Timeframe: at Week 24

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)

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Grip Strength Score	.5588 Proportion of responders	.6522 Proportion of responders	.8333 Proportion of responders

SECONDARY outcome

Timeframe: at Week 24

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

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Inflammatory Rasch-built Overall Disability Scale (I-RODS Score)	.3824 Proportion of responders	.5507 Proportion of responders	.7222 Proportion of responders

SECONDARY outcome

Timeframe: Week 24

Population: 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.

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)

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Worsening in the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score	NA days No patients with worsening in this group, analysis not possible.	NA days 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.	NA days No patients with worsening in this group, analysis not possible.

SECONDARY outcome

Timeframe: Up to 24 weeks

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.

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Mean Change in Grip Strength Dominant Hand	23.91 kPa Standard Deviation 25.290	19.38 kPa Standard Deviation 20.377	26.06 kPa Standard Deviation 25.030
Mean Change in Grip Strength Non-dominant Hand	23.94 kPa Standard Deviation 24.600	17.43 kPa Standard Deviation 19.916	24.53 kPa Standard Deviation 22.247

SECONDARY outcome

Timeframe: Up to 24 weeks

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.

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Inflammatory Rasch-built Overall Disability Scale (I-RODS)	11.38 score on a scale Standard Deviation 12.485	10.32 score on a scale Standard Deviation 10.836	13.86 score on a scale Standard Deviation 11.981

SECONDARY outcome

Timeframe: Up to 24 weeks

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.

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=68 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Motor Nerves	2.16 mV Standard Deviation 6.332	2.69 mV Standard Deviation 6.688	3.93 mV Standard Deviation 9.298

SECONDARY outcome

Timeframe: Up to 24 weeks

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.

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Mean Change in Pain Intensity Numerical Rating Scale (PI-NRS Scale)	-2.29 score on a scale Standard Deviation 3.040	-2.19 score on a scale Standard Deviation 2.907	-2.17 score on a scale Standard Deviation 3.256

SECONDARY outcome

Timeframe: 24 weeks

Population: 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

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).

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Worsening on the Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)	NA days There was only 1 patient with worsening in this group, thus an analysis of the time to first worsening was not possible.	NA days There were only 2 patients with worsening in this group, thus an analysis of the time to first worsening was not possible.	NA days No patients with worsening in this group, analysis not possible.

SECONDARY outcome

Timeframe: 24 weeks

Time to 1 point decrease (improvement of disability) in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
1 Point Decrease in the INCAT Disability Score	22.0 days Interval 22.0 to 24.0	26.0 days Interval 22.0 to 43.0	23.0 days Interval 22.0 to 43.0

SECONDARY outcome

Timeframe: 24 weeks

Time to decrease in Inflammatory Rasch-built overall disability scale (I-RODS) scores

Outcome measures

Outcome measures
Measure	1.0 g/kg NewGam n=34 Participants 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).	1.0 g/kg NewGam n=69 Participants 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).	2.0 g/kg NewGam n=36 Participants 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).
Decrease in Inflammatory Rasch-built Overall Disability Scale (I-RODS Scale)	NA days There were no patients with worsening in this group thus an analysis of the time to first worsening was not possible.	NA days There was only 1 patient with worsening in this group thus an analysis of the time to first worsening was not possible.	NA days There were no patients with worsening in this group thus an analysis of the time to first worsening was not possible.

Adverse Events

NGAM 0.5 g/kg

Serious events: 1 serious events

Other events: 20 other events

Deaths: 0 deaths

NGAM 1.0 g/kg

Serious events: 4 serious events

Other events: 45 other events

Deaths: 1 deaths

NGAM 2.0 g/kg

Serious events: 1 serious events

Other events: 24 other events

Deaths: 1 deaths

Total

Serious events: 6 serious events

Other events: 88 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	NGAM 0.5 g/kg n=35 participants at risk NGAM 0.5 g/kg	NGAM 1.0 g/kg n=69 participants at risk NGAM 1.0 g/kg	NGAM 2.0 g/kg n=38 participants at risk NGAM 2.0 g/kg	Total n=142 participants at risk Total of all three treatment arms.
Infections and infestations Osteomyelitis	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Infections and infestations Pneumonia	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Cardiac disorders Cardio-respiratory Arrest	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Respiratory, thoracic and mediastinal disorders Aspiration	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Ear and labyrinth disorders Deafness unilateral	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Musculoskeletal and connective tissue disorders Osteonecrosis	2.9% 1/35 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/69 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Nervous system disorders Headache	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Gastrointestinal disorders Vomiting	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Meningioma	0.00% 0/35 • Up to 36 weeks 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.	1.4% 1/69 • Number of events 1 • Up to 36 weeks 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.	0.00% 0/38 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.
Infections and infestations Encephalitis	0.00% 0/35 • Up to 36 weeks 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.	0.00% 0/69 • Up to 36 weeks 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.	2.6% 1/38 • Number of events 1 • Up to 36 weeks 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.	0.70% 1/142 • Number of events 1 • Up to 36 weeks 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.

Other adverse events

Additional Information

Mikaela Raymond

CRMG

Phone: 866-337-1868

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place