Trial Outcomes & Findings for Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome (NCT NCT02057692)

NCT ID: NCT02057692

Last Updated: 2019-03-26

Results Overview

Pruritus was assessed using Itch report outcome measure (ItchRO\[Obs\]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 37 participants
• Primary outcome timeframe: Baseline, Week 13/Early Termination
• Results posted on: 2019-03-26

Participant Flow

Study was conducted at 13 study centers in the United States and Canada, between 24 November 2014 (first participant first visit) and 16 November 2016 (last participant last visit).

Overall, 37 participants were enrolled to dose escalation phase started with 14 microgram per kilogram per day (mcg/kg/day) per week on Week 1 up to 280 mcg/kg/day for a maximum up to 5 weeks, until they reached the planned stable dose of 70, 140 and 280 mcg/kg/day and continued up to Week 13.

Participant milestones

Measure	Maralixibat 70 mcg/kg/Day Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Overall Study STARTED	8	11	6	12
Overall Study COMPLETED	7	11	6	11
Overall Study NOT COMPLETED	1	0	0	1

Reasons for withdrawal

Measure	Maralixibat 70 mcg/kg/Day Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Overall Study Adverse Event	1	0	0	0
Overall Study Lost to Follow-up	0	0	0	1

Baseline Characteristics

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Baseline characteristics by cohort

Measure	Maralixibat 70 mcg/kg/Day n=8 Participants Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 Participants Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 Participants Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).	Total n=37 Participants Total of all reporting groups
Age, Continuous	7.4 Years STANDARD_DEVIATION 4.75 • n=5 Participants	8.6 Years STANDARD_DEVIATION 4.32 • n=7 Participants	5.5 Years STANDARD_DEVIATION 4.76 • n=5 Participants	5.5 Years STANDARD_DEVIATION 4.19 • n=4 Participants	6.8 Years STANDARD_DEVIATION 4.48 • n=21 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants	6 Participants n=4 Participants	16 Participants n=21 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	7 Participants n=7 Participants	2 Participants n=5 Participants	6 Participants n=4 Participants	21 Participants n=21 Participants

PRIMARY outcome

Timeframe: Baseline, Week 13/Early Termination

Population: Modified Intent-to-Treat Population (mITT) population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.

Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.

Outcome measures

Measure	Maralixibat 70 mcg/kg/Day n=8 Participants Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 Participants Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 Participants Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).	Placebo Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus	-1.470 Score on a scale Standard Error 0.3034	-1.486 Score on a scale Standard Error 0.2597	-0.620 Score on a scale Standard Error 0.3575	-0.580 Score on a scale Standard Error 0.2453	—

SECONDARY outcome

Timeframe: Baseline, Week 13/Early Termination

Population: mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.

Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.

Outcome measures

Measure	Maralixibat 70 mcg/kg/Day n=8 Participants Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 Participants Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 Participants Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).	Placebo Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level	-117.401 Micro moles per liter (mcmol/L) Standard Error 46.2353	-40.358 Micro moles per liter (mcmol/L) Standard Error 34.8650	-27.437 Micro moles per liter (mcmol/L) Standard Error 46.2823	-10.442 Micro moles per liter (mcmol/L) Standard Error 32.6776	—

SECONDARY outcome

Timeframe: Baseline, Week 13/Early Termination

Population: mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.

Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.

Outcome measures

Measure	Maralixibat 70 mcg/kg/Day n=8 Participants Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 Participants Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 Participants Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).	Placebo Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels Alanine Aminotransferase	14.616 Unit per liter (U/L) Standard Error 17.9442	13.236 Unit per liter (U/L) Standard Error 14.9424	29.539 Unit per liter (U/L) Standard Error 19.6083	-11.928 Unit per liter (U/L) Standard Error 13.9736	—
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels Aspartate Aminotransferase	20.241 Unit per liter (U/L) Standard Error 16.8467	11.851 Unit per liter (U/L) Standard Error 13.9367	16.539 Unit per liter (U/L) Standard Error 18.3472	-3.691 Unit per liter (U/L) Standard Error 13.0114	—
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels Gamma Glutamyl Transferase	-48.124 Unit per liter (U/L) Standard Error 46.2867	48.053 Unit per liter (U/L) Standard Error 36.1507	-10.937 Unit per liter (U/L) Standard Error 48.8614	-37.757 Unit per liter (U/L) Standard Error 34.7766	—
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels Alkaline Phosphatase	46.276 Unit per liter (U/L) Standard Error 38.9056	-9.570 Unit per liter (U/L) Standard Error 30.8065	-3.280 Unit per liter (U/L) Standard Error 42.1582	32.502 Unit per liter (U/L) Standard Error 29.9885	—

SECONDARY outcome

Timeframe: Baseline, Week 13/Early Termination

Population: mITT population included all participants randomly assigned to study treatment, receiving at least 1 dose of treatment, and having at least 1 post baseline observer itch reported outcome (ItchRO\[Obs\]) average daily score.

Liver enzyme levels of total bilirubin and direct bilirubin were reported here.

Outcome measures

Measure	Maralixibat 70 mcg/kg/Day n=8 Participants Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 Participants Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 Participants Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).	Placebo Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations Total Bilirubin	-0.291 Milligrams per deciliter (mg/dL) Standard Error 0.3777	-0.353 Milligrams per deciliter (mg/dL) Standard Error 0.3014	-0.804 Milligrams per deciliter (mg/dL) Standard Error 0.4021	0.104 Milligrams per deciliter (mg/dL) Standard Error 0.2847	—
Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations Direct Bilirubin	-0.333 Milligrams per deciliter (mg/dL) Standard Error 0.2086	-0.305 Milligrams per deciliter (mg/dL) Standard Error 0.1672	-0.508 Milligrams per deciliter (mg/dL) Standard Error 0.2243	0.011 Milligrams per deciliter (mg/dL) Standard Error 0.1591	—

OTHER_PRE_SPECIFIED outcome

Timeframe: From the start of study drug administration up to Week 17

Population: The Safety Population included all participants who were randomly assigned to study treatment and received at least one dose of the study drug.

An AE was any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life -threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.TEAEs were defined as AEs/SAEs that started or worsened after the study drug treatment.

Outcome measures

Measure	Maralixibat 70 mcg/kg/Day n=1 Participants Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=7 Participants Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=11 Participants Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=6 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).	Placebo n=12 Participants Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) TEAE	1 Participants	7 Participants	9 Participants	5 Participants	12 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) Serious TEAE	0 Participants	1 Participants	0 Participants	0 Participants	0 Participants

Adverse Events

Maralixibat 14 mcg/kg/Day

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Maralixibat 70 mcg/kg/Day

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Maralixibat 140 mcg/kg/Day

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Maralixibat 280 mcg/kg/Day

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Measure	Maralixibat 14 mcg/kg/Day n=1 participants at risk Participants received 14 mcg/kg/day of maralixibat oral solution for 1 week. One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant using this arm.	Maralixibat 70 mcg/kg/Day n=7 participants at risk Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 participants at risk Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 participants at risk Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 participants at risk Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Gastrointestinal disorders Vomiting	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.

Other adverse events

Measure	Maralixibat 14 mcg/kg/Day n=1 participants at risk Participants received 14 mcg/kg/day of maralixibat oral solution for 1 week. One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant using this arm.	Maralixibat 70 mcg/kg/Day n=7 participants at risk Participants received low dose maralixibat 70 mcg/kg/day oral solution for 10 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35 and 70 mcg/kg/day dose per each week respectively up to 3 weeks.	Maralixibat 140 mcg/kg/Day n=11 participants at risk Participants received mid dose maralixibat 140 mcg/kg/day oral solution for 9 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70 and 140 mcg/kg/day dose per each week respectively up to 4 weeks.	Maralixibat 280 mcg/kg/Day n=6 participants at risk Participants received high dose maralixibat 280 mcg/kg/day oral solution for 8 weeks during the stable dosing period, which was administered after a dose escalation period, in which participants received 14, 35, 70, 140 and 280 mcg/kg/day dose per each week respectively up to 5 weeks.	Placebo n=12 participants at risk Participants received placebo matched to maralixibat oral solution for 13 weeks (dose escalation and stable dosing period).
Cardiac disorders Left ventricular hypertrophy	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Acarodermatitis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Ear infection	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	18.2% 2/11 • Number of events 3 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
General disorders Irritability	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
General disorders Pyrexia	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	28.6% 2/7 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	18.2% 2/11 • Number of events 3 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	33.3% 2/6 • Number of events 5 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	25.0% 3/12 • Number of events 5 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Musculoskeletal and connective tissue disorders Growing pains	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 4 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Musculoskeletal and connective tissue disorders Muscle spasms	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Musculoskeletal and connective tissue disorders Neck pain	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Musculoskeletal and connective tissue disorders Pain in extremity	100.0% 1/1 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 2/12 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Vascular disorders Pallor	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Eye disorders Eye pain	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Eye disorders Eye swelling	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Abdominal pain	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	28.6% 2/7 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 2/12 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	18.2% 2/11 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 2/12 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Constipation	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Diarrhoea	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	54.5% 6/11 • Number of events 6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	33.3% 2/6 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	50.0% 6/12 • Number of events 8 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Haematemesis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Haematochezia	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Lip swelling	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Nausea	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Tooth crowding	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Gastrointestinal disorders Vomiting	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
General disorders Fatigue	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
General disorders Influenza like illness	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Herpes simplex	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Influenza	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Nasopharyngitis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	33.3% 2/6 • Number of events 3 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Oral herpes	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Otitis media	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Pertussis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Pharyngitis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Pharyngitis streptococcal	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Upper respiratory tract infection	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	18.2% 2/11 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 2/12 • Number of events 3 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Infections and infestations Urinary tract infection	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Injury, poisoning and procedural complications Overdose	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Acoustic stimulation tests abnormal	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Alanine aminotransferase increased	100.0% 1/1 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Aspartate aminotransferase increased	100.0% 1/1 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Blood alkaline phosphatase increased	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Blood bicarbonate decreased	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Blood pressure systolic increased	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Gamma-Glutamyltransferase increased	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations International normalised ratio increased	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Investigations Neutrophil count decreased	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Renal and urinary disorders Enuresis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Renal and urinary disorders Pollakiuria	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Metabolism and nutrition disorders Dehydration	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Metabolism and nutrition disorders Hypoglycaemia	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Metabolism and nutrition disorders Vitamin e deficiency	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Nervous system disorders Convulsion	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Nervous system disorders Dizziness	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Nervous system disorders Headache	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	28.6% 2/7 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Nervous system disorders Migraine	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Nervous system disorders Peripheral sensory neuropathy	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Psychiatric disorders Mood altered	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	28.6% 2/7 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	18.2% 2/11 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	33.3% 2/6 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 1/6 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Respiratory, thoracic and mediastinal disorders Sinus congestion	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	8.3% 1/12 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Skin and subcutaneous tissue disorders Rash	0.00% 0/1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	14.3% 1/7 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	9.1% 1/11 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	16.7% 2/12 • Number of events 2 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.
Skin and subcutaneous tissue disorders Rash maculo-papular	100.0% 1/1 • Number of events 1 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/7 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/11 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/6 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.	0.00% 0/12 • From the start of study drug administration up to Week 17 One participant assigned to the 70μg/kg/day arm, was withdrawn from the study after receiving a single dose of 14 mcg/kg/day dose. TEAE was reported separately for this participant in a separate arm.

Additional Information

Study Physician

Mirum

Phone: 1 650-667-4085

Email: medinfo@mirumpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
• Publication restrictions are in place

Restriction type: OTHER