Trial Outcomes & Findings for A Safety and Tolerability Study of ILB® in Patients With Amyotrophic Lateral Sclerosis (ALS) (NCT NCT03705390)

NCT ID: NCT03705390

Last Updated: 2025-06-26

Results Overview

Measured by the number of serious adverse events (SAEs) and adverse events (AEs) using CTCAE grading v4.0.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 11 participants
• Primary outcome timeframe: From informed consent up to 30 days after last administration of trial treatment
• Results posted on: 2025-06-26

Participant Flow

Weekly dosing for 10 weeks (in the first instance) during out-patient visits at site. Dosing beyond 10 weeks (initially to 24 weeks and then up to a maximum of up to 48 weeks) was dependent upon a formal review of the patient's eligibility, their wishes and the most suitable treatment options. Following suspension of the trial due to the COVID-19 pandemic, patients were asked to re-consent for an additional single point long-term remote follow up visit in Quarter 1 of 2021 via video call.

Participant milestones

Measure	ILB® Arm ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
10 Week Initial Treatment Period STARTED	11
10 Week Initial Treatment Period COMPLETED	9
10 Week Initial Treatment Period NOT COMPLETED	2
Treatment Extension STARTED	9
Treatment Extension COMPLETED	8
Treatment Extension NOT COMPLETED	1

Reasons for withdrawal

Measure	ILB® Arm ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
10 Week Initial Treatment Period Trial suspended prior to completion	2
Treatment Extension Trial closed early due to COVID-19 pandemic	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Age, Categorical <=18 years	0 Participants n=11 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=11 Participants
Age, Categorical >=65 years	2 Participants n=11 Participants
Age, Continuous	58 years STANDARD_DEVIATION 8.77 • n=11 Participants
Sex: Female, Male Female	4 Participants n=11 Participants
Sex: Female, Male Male	7 Participants n=11 Participants
Region of Enrollment United Kingdom	11 participants n=11 Participants
Time from ALS diagnosis to trial entry (months)	7.27 months STANDARD_DEVIATION 5.34 • n=11 Participants
Family history of motor neurone disease (n(%)) No	10 Participants n=11 Participants
Family history of motor neurone disease (n(%)) Yes	1 Participants n=11 Participants
Family history of fronto-temporal dementia (n(%)) No	11 Participants n=11 Participants
Family history of fronto-temporal dementia (n(%)) Yes	0 Participants n=11 Participants

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Measured by the number of serious adverse events (SAEs) and adverse events (AEs) using CTCAE grading v4.0.

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by SAEs and AEs - Measured by Incidence Serious Adverse Events	1 Adverse events
Safety Assessed by SAEs and AEs - Measured by Incidence Adverse Events	270 Adverse events

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Grade refers to the severity of the AE as follows:

Grade 1 - Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated.

Grade 2 - Moderate; minimal, local or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living.

Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living.

Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE.

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by AEs - Summarised by Grade Grade 1	265 number of AEs
Safety Assessed by AEs - Summarised by Grade Grade 2	4 number of AEs
Safety Assessed by AEs - Summarised by Grade Grade 3	1 number of AEs
Safety Assessed by AEs - Summarised by Grade Grade 4	0 number of AEs
Safety Assessed by AEs - Summarised by Grade Grade 5	0 number of AEs

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Relatedness categories: 1 = unrelated, 2 = unlikely to be related, 3 = possibly related, 4 = probably related, 5 = definitely related

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by AEs - Summarised by Relatedness Definitely related	93 number of AEs
Safety Assessed by AEs - Summarised by Relatedness Unrelated	127 number of AEs
Safety Assessed by AEs - Summarised by Relatedness Unlikely to be related	45 number of AEs
Safety Assessed by AEs - Summarised by Relatedness Possibly related	4 number of AEs
Safety Assessed by AEs - Summarised by Relatedness Probably related	1 number of AEs

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Grade refers to the severity of the admitting event as follows:

Grade 1 - Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated.

Grade 2 - Moderate; minimal, local or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living.

Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living.

Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE.

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by SAEs - Summarised by Admitting Event Grade Grade 1	0 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Grade Grade 2	0 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Grade Grade 3	1 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Grade Grade 4	0 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Grade Grade 5	0 number of SAEs

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Relatedness categories: 1 = unrelated, 2 = unlikely to be related, 3 = possibly related, 4 = probably related, 5 = definitely related

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by SAEs - Summarised by Admitting Event Relatedness Unrelated	1 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Relatedness Unlikely to be related	0 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Relatedness Possibly related	0 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Relatedness Probably related	0 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Relatedness Definitely related	0 number of SAEs

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Description of the main event type - primary cause of admission (body system, Adverse event term and grade)

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by SAEs - Summarised by Admitting Event Type Musculoskeletal and connective tissue disorder - Other, specify: generalised muscle weakness	1 number of SAEs
Safety Assessed by SAEs - Summarised by Admitting Event Type Other admitting event types	0 number of SAEs

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Population: Expectedness of SAEs is only ascertained in the instance of the event being a Serious Adverse Reaction (SAR), therefore as the only SAE was unrelated, expectedness was not applicable

Serious Adverse Events only will be defined as expected or unexpected based on information provided in the Quick Reference Document

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

Outcome of serious adverse events only: Resolved with sequelae or Resolved without sequelae

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Safety Assessed by SAEs - Summarised by Sequelae Resolved with Sequelae	0 number of SAEs
Safety Assessed by SAEs - Summarised by Sequelae Resolved without Sequelae	1 number of SAEs

PRIMARY outcome

Timeframe: From informed consent up to 30 days after last administration of trial treatment

An intolerable adverse event will satisfy all of the following criteria:

1. Associated with a serious adverse event or a drug discontinuation of greater than three weeks;

2. Grade 3, 4 or 5 in severity according to CTCAE version 4;

3. In the opinion of the Investigator is i) definitely related or ii) probably related or iii) possibly related to the study drug treatment.

Adverse events which are considered unrelated or probably not related will not be classed as intolerable events.

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Tolerability Assessed by the Incidence of Intolerable Adverse Events	0 Number of intolerable adverse events

PRIMARY outcome

Timeframe: From baseline to final treatment visit

Total drug administered over the study period (measured in milligrams)

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Quantity of Study Drug Administered - Total Drug Administered	4200 mg Interval 1902.0 to 5754.0

PRIMARY outcome

Timeframe: From baseline to final treatment visit

Numerical count of the number of study drug injections given whilst on the trial

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Quantity of Study Drug Administered - Number of Administrations	24 number of drug administrations Interval 14.0 to 35.0

PRIMARY outcome

Timeframe: From baseline to final treatment visit

Numerical count of the number of study drug injections missed whilst on the trial

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Quantity of Study Drug Administered - Number of Interruptions	1 number of interruptions Interval 0.0 to 3.0

PRIMARY outcome

Timeframe: From baseline to final treatment visit

Population: 6 patients experienced a treatment interruption whilst on the trial

Length of interruptions in weeks between study drug injections for those participants that experienced a treatment interruption whilst on the trial

Outcome measures

Measure	ILB® Arm n=6 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Quantity of Study Drug Administered - Duration of Interruptions	1.5 weeks Interval 1.0 to 2.0

PRIMARY outcome

Timeframe: From baseline to final treatment visit

numerical count of patients who have discontinued study drug treatment

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Quantity of Study Drug Administered - Number of Discontinuations	0 number of treatment discontinuations

SECONDARY outcome

Timeframe: From baseline to final treatment visit

Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R). This patient reported outcome measures the subjective well-being of patients. There are 5 scales which are calculated and scored: physical mobility, independence,eating and drinking, communication,emotional functioning. Each is scored between 0-100. An improved condition is represented by a decreasing sub-scale score. These sub scales are then averaged to make a summary index score. The range of the summary index is 0-100 and an improved condition is represented by decreasing summary index score. For each of the sub-scales and summary index. Interpretation is as follows: 0-19 Never or very rarely, 20-39 rarely experience problems, 40-59 sometimes experience problems,60-79 often experience, 80-100 problems (nearly) always or unable to do at all. The scale of the summary score is also 0-100 with analogous interpretation to the sub scales. An increase in score is a worse outcome.

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Score Change	2.0 score on a scale Interval 0.5 to 3.0

SECONDARY outcome

Timeframe: From baseline to final treatment visit

Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 . A functional rating scale including assessments of communication, mobility, dressing and respiration. the total score range is 0-40. An improved condition is represented by decreasing sub-scale score. Interpretation is as follows: 0 being the best outcome and 40 being the worst. Minimum value is 0 and Maximum value is 40 per time-point / questionnaire completion

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change Physical mobility	-5.0 change in ALSAQ-40 score from baseline Interval -15.0 to -3.8
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change Activities of daily living	-2.5 change in ALSAQ-40 score from baseline Interval -17.5 to 2.5
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change Eating and drinking	0 change in ALSAQ-40 score from baseline Interval -12.5 to 0.0
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change Communication	0 change in ALSAQ-40 score from baseline Interval -8.9 to 0.0
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change Emotional functioning	-7.5 change in ALSAQ-40 score from baseline Interval -18.5 to -5.0
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change Summary index score	-6.2 change in ALSAQ-40 score from baseline Interval -15.0 to -1.9

SECONDARY outcome

Timeframe: From baseline to final treatment visit

Urinary p75 extracellular domain (p75ECD) is a biological fluid-based biomarker of ALS disease progression

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Urinary p75ECD Change	-0.62 ng/mmol creatinine Interval -0.98 to 0.22

SECONDARY outcome

Timeframe: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration

This outcome measure quantifies the amount of drug detectable in the blood after administration over time

Outcome measures

Measure	ILB® Arm n=6 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 0.5 hours	3.73 μg/mL Standard Deviation 1.41
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 1 hours	4.86 μg/mL Standard Deviation 1.18
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 2 hours	5.56 μg/mL Standard Deviation 1.45
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 2.5 hours	5.66 μg/mL Standard Deviation 1.5
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 3.0 hours	5.4 μg/mL Standard Deviation 1.03
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 4.0 hours	4.76 μg/mL Standard Deviation 1.17
Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration 6.0 hours	3.72 μg/mL Standard Deviation 0.76

SECONDARY outcome

Timeframe: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration

Tmax (the time the peak concentration occurred) was calculated to characterise the kinetic profile of ILB® in plasma post-administration

Outcome measures

Measure	ILB® Arm n=6 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Pharmacokinetics (PK; Tmax) Statistics of ILB® in Plasma Following Administration	2.42 hours Standard Deviation 0.38

SECONDARY outcome

Timeframe: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration

Cmax (peak concentration of ILB® in plasma post-administration) was calculated to characterise the kinetic profile of ILB® in plasma post-administration

Outcome measures

Measure	ILB® Arm n=6 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Pharmacokinetics (PK; Cmax) Statistics of ILB® in Plasma Following Administration	6.03 μg/mL Standard Deviation 1.16

SECONDARY outcome

Timeframe: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration

AUC0-last (area under the curve time 0 (time of administration) to the last value above the limit of quantification) was calculated to characterise the kinetic profile of ILB® in plasma post-administration

Outcome measures

Measure	ILB® Arm n=6 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Pharmacokinetics (PK; AUC0-last) Statistics of ILB® in Plasma Following Administration	27.18 μg*h/mL Standard Deviation 5.62

SECONDARY outcome

Timeframe: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration

Population: The terminal half-life t1/2 could not be calculated for 3 out of the 6 patients as the concentration of ILB® in plasma did not drop below half its maximal value.

t1/2 (terminal half-life of ILB® in plasma post-administration) was calculated to characterise the kinetic profile of ILB® in plasma post-administration

Outcome measures

Measure	ILB® Arm n=3 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Pharmacokinetics (PK; t1/2) Statistics of ILB® in Plasma Following Administration	3.74 hours Standard Deviation 1.63

SECONDARY outcome

Timeframe: from baseline to final treatment visit

Plasma neurofilament light chain (NfL) is a blood-based biomarker for neurodegeneration

Outcome measures

Measure	ILB® Arm n=11 Participants ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
NfL in Plasma Change	1.5 ng/L Interval -3.0 to 4.5

Adverse Events

ILB® Arm

Serious events: 1 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Measure

ILB® Arm n=11 participants at risk ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks

Musculoskeletal and connective tissue disorders Generalised muscle weakness

9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.

Other adverse events

Measure	ILB® Arm n=11 participants at risk ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Blood and lymphatic system disorders Not clinically significant abnormal blood creatinine	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Blood and lymphatic system disorders Not clinically significant creatinine kinase, 286, (normal range 30-200)	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Blood and lymphatic system disorders Not clinically significant monocyte count, 0.9, (normal range 0.2 - 0.8)	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Blood and lymphatic system disorders Not clinically significant raised red cell distribution, 14.8 (normal range 11-14)	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Eye disorders 'Sticky' left eye	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Gastrointestinal disorders Constipation	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Gastrointestinal disorders Diarrhea	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Gastrointestinal disorders Rectal bleeding	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
General disorders Chesty cough	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
General disorders Cold chills down left arm	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
General disorders Cramps in the chest and abdomen	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
General disorders Discomfort at injection site when touched	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
General disorders Patient felt faint	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Infections and infestations Bilateral ear infection	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Infections and infestations Chest infection	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Infections and infestations Common cold	27.3% 3/11 • Number of events 4 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Infections and infestations Patient visited the dentist and had a filling	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Bruising	81.8% 9/11 • Number of events 92 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Fall	45.5% 5/11 • Number of events 9 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Bruising to right hip from fall	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Bruising to right shoulder following fall	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Bruising to the face following fall	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Fall up the stairs leading to flat due to increasing leg weakness	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Injury, poisoning and procedural complications Fall while getting out of bed, due to increase in leg weakness	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Activated partial thromboplastin time prolonged	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Alanine aminotransferase increased	18.2% 2/11 • Number of events 3 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Alkaline phosphatase increased	18.2% 2/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Aspartate aminotransferase increased	27.3% 3/11 • Number of events 6 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Blood bilirubin increased	9.1% 1/11 • Number of events 4 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Cholesterol high	45.5% 5/11 • Number of events 8 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Hemoglobin increased	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal CRP blood results	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal eosinophil blood results	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal glucose levels (3.1, normal results 3.5-11.0)	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal hdl level, clinically insignificant.	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal mean cell haemoglobin level, clinically insignificant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal platelet distribution width result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal ptt blood result (elevated)	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Abnormal red cell distribution levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Creatinine level decreased	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations CRP increased	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased aptt result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased basophil count	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased basophil level	18.2% 2/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased basophil result	36.4% 4/11 • Number of events 4 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased creatinine	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased creatinine levels	9.1% 1/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased creatinine result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased creatinine value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased haematocrit value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased haemoglobin value	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased hdl cholesterol result	18.2% 2/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased hdl cholesterol value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased hdl cholesterol value -not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased hdl value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased igm result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased monocyte result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased potassium level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased rbc dist width	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased rbc distribution width	18.2% 2/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased red blood cell value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Decreased sodium levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated aptt ratio	9.1% 1/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated ast blood level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated c- reactive protein blood results	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated calcim level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated calcium level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated ck levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated creatine kinase result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated crp result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated crp value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated eosinophil result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated haematocrit blood level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated hdl cholesterol blood levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated mean cell haemoglobin	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated mean cell hb concentration	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated mean cell hb level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated mean cell hb result	18.2% 2/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated monocyte result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated red blood cell count	27.3% 3/11 • Number of events 4 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated sodium levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Elevated total protein result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Hdl cholesterol levels high	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased ck value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased crp level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased eosinophils count - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased esr value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased iga value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased monocyte count - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased neutrophil count - not clinically significant	9.1% 1/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased platelet dist width	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased platelet dist. width	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased rbc distribution width	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Increased white blood cell value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Low albumin level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Low haematocrit	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Low sodium	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Mean cell hb concentration level elevated	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Prolonged pr interval on ecg	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised ast blood levels	9.1% 1/11 • Number of events 2 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised ast levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised blood glucose level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised crp result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised eosinophil count	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised eosinophils	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised hdl cholesterol blood levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised sodium level	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Raised total protein result	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Reduced aptt ratio value - not clinically significant	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Reduced basophil count	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Reduced free thyroxine levels	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Lymphocyte count decreased	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations Neutrophil count decreased	18.2% 2/11 • Number of events 3 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Investigations White blood cell decreased	27.3% 3/11 • Number of events 5 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Back pain	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Fractured nasal bone following fall	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Increased leg weakness leading to inability to stand resulting in admission to hospital	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Muscle spasm in legs	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Occasional spasm in neck when yawning	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Painful left shoulder blade	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Musculoskeletal and connective tissue disorders Right hip pain	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Nervous system disorders Headache	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Psychiatric disorders Insomnia	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Respiratory, thoracic and mediastinal disorders Allergic rhinitis	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Respiratory, thoracic and mediastinal disorders Pneumonia	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Respiratory, thoracic and mediastinal disorders Sore throat	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Skin and subcutaneous tissue disorders Eczema	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Skin and subcutaneous tissue disorders Rash on abdomen around injection site	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Skin and subcutaneous tissue disorders Rash on both feet	9.1% 1/11 • Number of events 1 • Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study. All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.

Additional Information

D3B Trial Mangement Team Leader

CRCTU, University of Birmingham

Phone: +44 (0) 121 371 8027

Email: ALS@trials.bham.ac.uk

Results disclosure agreements

• Principal investigator is a sponsor employee The Clinical Centre shall not publish or otherwise disseminate the conclusions of the Study, including all or any part of the Results of the Study without the prior written consent of the Sponsor, such consent not to be unreasonably withheld or delayed. Any publication or other dissemination of the conclusions of the Study by the National Health Organisation (NHS) Organisation shall not' occur until the Sponsor has published the conclusions of the Study. Results published- PubMed ID: 38990927
• Publication restrictions are in place

Restriction type: OTHER