Trial Outcomes & Findings for A Study to Evaluate Emapalumab in Japanese Healthy Volunteers. (NCT NCT04765553)

Results Overview

The maximum observed concentration of emapalumab (Cmax)

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

Day 1 preinfusion, 1hr, 2hrs, 4hrs, 8hrs, 10hrs post dose, day 2, 3, 5, 8, week 2, 4, 6, 8, 10, 12, study completion week 14 or Withdrawal

Results posted on

2023-11-30

Participant Flow

Participant milestones

Participant milestones
Measure	Emapalumab Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Overall Study STARTED	6	2
Overall Study COMPLETED	6	2
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion	Total n=8 Participants Total of all reporting groups
Age, Continuous	35.0 years n=5 Participants	45 years n=7 Participants	40 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	6 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2hrs, 4hrs, 8hrs, 10hrs post dose, day 2, 3, 5, 8, week 2, 4, 6, 8, 10, 12, study completion week 14 or Withdrawal

The maximum observed concentration of emapalumab (Cmax)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
The Maximum Observed Concentration of Emapalumab	23980 ng/mL Interval 22000.0 to 30600.0	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

The time at which the maximum concentration of emapalumab is observed (Tmax)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
The Time at Which the Maximum Concentration of Emapalumab is Observed	2.000 Hours Interval 2.0 to 2.0	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Concentration of emapalumab at end of infusion (CEnd of inf))

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Concentration of Emapalumab at End of Infusion	23230 ng/mL Interval 21800.0 to 26800.0	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Area under the plasma concentration-time curve from emapalumab injection to time of last measurable concentration (AUClast)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Area Under the Plasma Concentration-time Curve	11690000 ng*h/mL Interval 10300000.0 to 13400000.0	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Area under the plasma concentration-time curve from emapalumab injection extrapolated to infinity (AUCinf)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Area Under the Concentration-time Curve Extrapolated to Infinity	12430000 ng*h/mL Interval 10800000.0 to 14600000.0	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Emapalumab elimination half-life (t1/2)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Emapalumab Elimination Half-life	634.9 Hours Interval 489.0 to 683.0	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Apparent total body clearance of emapalumab from plasma (CL)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Apparent Total Body Clearance of Emapalumab From Plasma	0.006136 L/h Interval 0.00395 to 0.00732	—

PRIMARY outcome

Timeframe: Day 1 preinfusion, 1hr, 2 hrs, 4hrs, 8hrs, 10hrs, Day 2, 3, 5, 8, Week 2, 4, 6, 8, 10, 12, study completion week 14 or at Withdrawal

Apparent volume of distribution at steady state (Vss)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Steady State Volume of Distribution	4.953 L Interval 3.66 to 5.38	—

SECONDARY outcome

Timeframe: Continuously from start of emapalumab infusion up to 14 weeks

Total number of reported adverse events

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Overall Summary of Adverse Events All Treatment emergent adverse events (TEAEs)	3 number of events	1 number of events
Overall Summary of Adverse Events Adverse Drug Reactions (ADRs)	3 number of events	0 number of events
Overall Summary of Adverse Events Serious TEAEs	0 number of events	0 number of events
Overall Summary of Adverse Events Serious ADRs	0 number of events	0 number of events
Overall Summary of Adverse Events TEAEs leading to drug withdrawn	0 number of events	0 number of events
Overall Summary of Adverse Events ADRs leading to drug withdrawn	0 number of events	0 number of events
Overall Summary of Adverse Events TEAEs leading to dose reduced	0 number of events	0 number of events
Overall Summary of Adverse Events ADRs leading to dose reduced	0 number of events	0 number of events
Overall Summary of Adverse Events TEAEs leading to drug interrupted	0 number of events	0 number of events
Overall Summary of Adverse Events ADRs leading to drug interrupted	0 number of events	0 number of events
Overall Summary of Adverse Events TEAEs leading to death	0 number of events	0 number of events
Overall Summary of Adverse Events ADRs leading to death	0 number of events	0 number of events
Overall Summary of Adverse Events Infusion Related Reactions, (IRRs)	2 number of events	0 number of events

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples.

Change from baseline in levels of Aspartate aminotransferase (AST)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Aspartate Aminotransferase Day 1	0.5 IU/L Interval -4.0 to 2.0	2.5 IU/L Interval 0.0 to 5.0
Change in Levels of Aspartate Aminotransferase Day 2	-0.5 IU/L Interval -5.0 to 1.0	5.0 IU/L Interval -1.0 to 11.0
Change in Levels of Aspartate Aminotransferase Day 3	-2.5 IU/L Interval -7.0 to 0.0	1.5 IU/L Interval -4.0 to 7.0
Change in Levels of Aspartate Aminotransferase Day 5	-1.5 IU/L Interval -5.0 to 1.0	9.0 IU/L Interval -4.0 to 22.0
Change in Levels of Aspartate Aminotransferase Day 8	1.0 IU/L Interval -3.0 to 9.0	11.5 IU/L Interval -1.0 to 24.0
Change in Levels of Aspartate Aminotransferase Week 2	-2 IU/L Interval -5.0 to 1.0	7.0 IU/L Interval 7.0 to 7.0
Change in Levels of Aspartate Aminotransferase Week 4	-0.5 IU/L Interval -4.0 to 18.0	6.5 IU/L Interval 6.0 to 7.0
Change in Levels of Aspartate Aminotransferase Week 6	-2.0 IU/L Interval -5.0 to 2.0	0.5 IU/L Interval 0.0 to 1.0
Change in Levels of Aspartate Aminotransferase Week 10	-1.5 IU/L Interval -3.0 to 4.0	6.5 IU/L Interval 4.0 to 9.0
Change in Levels of Aspartate Aminotransferase Week 14	-2.0 IU/L Interval -6.0 to 8.0	5.0 IU/L Interval 0.0 to 10.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Alanine aminotransferase (ALT)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Alanine Aminotransferase Day 1	0.5 IU/L Interval -4.0 to 5.0	1.0 IU/L Interval -1.0 to 3.0
Change in Levels of Alanine Aminotransferase Day 2	0.0 IU/L Interval -5.0 to 4.0	2.5 IU/L Interval -2.0 to 7.0
Change in Levels of Alanine Aminotransferase Day 3	-2.0 IU/L Interval -7.0 to 0.0	0.5 IU/L Interval -6.0 to 7.0
Change in Levels of Alanine Aminotransferase Day 5	-0.5 IU/L Interval -4.0 to 5.0	12.0 IU/L Interval -7.0 to 31.0
Change in Levels of Alanine Aminotransferase Day 8	4.5 IU/L Interval -5.0 to 19.0	18.5 IU/L Interval -6.0 to 43.0
Change in Levels of Alanine Aminotransferase Week 2	-3.0 IU/L Interval -11.0 to 4.0	15.0 IU/L Interval 15.0 to 15.0
Change in Levels of Alanine Aminotransferase Week 4	1.0 IU/L Interval -8.0 to 12.0	19.5 IU/L Interval 10.0 to 29.0
Change in Levels of Alanine Aminotransferase Week 6	-4.0 IU/L Interval -7.0 to 6.0	0.0 IU/L Interval -3.0 to 3.0
Change in Levels of Alanine Aminotransferase Week10	2.0 IU/L Interval -5.0 to 8.0	8.5 IU/L Interval 7.0 to 10.0
Change in Levels of Alanine Aminotransferase Week 14	0.0 IU/L Interval -9.0 to 17.0	5.5 IU/L Interval -1.0 to 12.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Direct Bilirubin

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Direct Bilirubin Day 1	0.15 mg/dL Interval 0.0 to 0.3	0.05 mg/dL Interval 0.0 to 0.1
Change in Levels of Direct Bilirubin Day 2	0.15 mg/dL Interval 0.0 to 0.5	0.00 mg/dL Interval -0.1 to 0.1
Change in Levels of Direct Bilirubin Day 3	0.10 mg/dL Interval -0.1 to 0.2	-0.05 mg/dL Interval -0.1 to 0.0
Change in Levels of Direct Bilirubin Day 5	0.05 mg/dL Interval -0.1 to 0.1	-0.05 mg/dL Interval -0.1 to 0.0
Change in Levels of Direct Bilirubin Day 8	0.10 mg/dL Interval -0.1 to 0.1	0.00 mg/dL Interval 0.0 to 0.0
Change in Levels of Direct Bilirubin Week 2	0.00 mg/dL Interval -0.1 to 0.1	-0.10 mg/dL Interval -0.1 to -0.1
Change in Levels of Direct Bilirubin Week 4	0.05 mg/dL Interval 0.0 to 0.1	0.00 mg/dL Interval 0.0 to 0.0
Change in Levels of Direct Bilirubin Week 6	0.10 mg/dL Interval -0.1 to 0.1	0.00 mg/dL Interval 0.0 to 0.0
Change in Levels of Direct Bilirubin Week 10	0.05 mg/dL Interval 0.0 to 0.2	-0.05 mg/dL Interval -0.1 to 0.0
Change in Levels of Direct Bilirubin Week 14	0.00 mg/dL Interval 0.0 to 0.1	-0.05 mg/dL Interval -0.1 to 0.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Total Bilirubin

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Total Bilirubin Day 8	0.20 mg/dL Interval -0.5 to 0.6	0.05 mg/dL Interval -0.1 to 0.2
Change in Levels of Total Bilirubin Week 2	-0.05 mg/dL Interval -0.5 to 0.2	-0.20 mg/dL Interval -0.2 to -0.2
Change in Levels of Total Bilirubin Week 4	0.15 mg/dL Interval -0.3 to 0.5	-0.05 mg/dL Interval -0.1 to 0.0
Change in Levels of Total Bilirubin Week 6	0.10 mg/dL Interval -0.4 to 0.5	0.05 mg/dL Interval -0.1 to 0.2
Change in Levels of Total Bilirubin Week 10	0.15 mg/dL Interval -0.2 to 0.6	0.00 mg/dL Interval -0.1 to 0.1
Change in Levels of Total Bilirubin Week 14	0.10 mg/dL Interval -0.3 to 0.2	-0.15 mg/dL Interval -0.5 to 0.2
Change in Levels of Total Bilirubin Day 1	0.35 mg/dL Interval 0.1 to 1.3	0.30 mg/dL Interval 0.2 to 0.4
Change in Levels of Total Bilirubin Day 2	0.55 mg/dL Interval 0.0 to 1.7	0.10 mg/dL Interval -0.1 to 0.3
Change in Levels of Total Bilirubin Day 3	0.15 mg/dL Interval -0.5 to 0.8	-0.10 mg/dL Interval -0.3 to 0.1
Change in Levels of Total Bilirubin Day 5	0.10 mg/dL Interval -0.4 to 0.4	-0.20 mg/dL Interval -0.3 to -0.1

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Uric acid

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Uric Acid Day 3	0.40 mg/dL Interval -0.1 to 0.8	0.15 mg/dL Interval -0.4 to 0.7
Change in Levels of Uric Acid Day 5	0.35 mg/dL Interval -0.1 to 1.6	0.20 mg/dL Interval -0.2 to 0.6
Change in Levels of Uric Acid Day 8	0.25 mg/dL Interval -0.4 to 1.2	0.20 mg/dL Interval 0.0 to 0.4
Change in Levels of Uric Acid Week 2	-0.10 mg/dL Interval -0.6 to 1.1	-0.40 mg/dL Interval -0.4 to -0.4
Change in Levels of Uric Acid Week 4	0.25 mg/dL Interval -0.6 to 0.5	-0.05 mg/dL Interval -0.5 to 0.4
Change in Levels of Uric Acid Week 6	0.00 mg/dL Interval -0.6 to 0.7	0.10 mg/dL Interval -0.2 to 0.4
Change in Levels of Uric Acid Week 10	0.40 mg/dL Interval -0.2 to 2.2	0.55 mg/dL Interval 0.3 to 0.8
Change in Levels of Uric Acid Week 14	0.10 mg/dL Interval -0.7 to 0.5	0.85 mg/dL Interval 0.3 to 1.4
Change in Levels of Uric Acid Day 1	0.15 mg/dL Interval -0.2 to 0.6	0.10 mg/dL Interval -0.1 to 0.3
Change in Levels of Uric Acid Day 2	0.40 mg/dL Interval 0.1 to 0.8	0.15 mg/dL Interval -0.3 to 0.6

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Alkaline phosphatase

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Alkaline Phosphatase Day 1	-34.0 U/L Interval -45.0 to 13.0	16.0 U/L Interval 15.0 to 17.0
Change in Levels of Alkaline Phosphatase Day 2	-26.5 U/L Interval -46.0 to 25.0	16.0 U/L Interval 13.0 to 19.0
Change in Levels of Alkaline Phosphatase Day 3	-23.5 U/L Interval -42.0 to 30.0	22.0 U/L Interval 10.0 to 34.0
Change in Levels of Alkaline Phosphatase Day 5	-28.0 U/L Interval -53.0 to 31.0	17.0 U/L Interval 4.0 to 30.0
Change in Levels of Alkaline Phosphatase Day 8	-6.5 U/L Interval -20.0 to 27.0	35.0 U/L Interval 22.0 to 48.0
Change in Levels of Alkaline Phosphatase Week 2	9.5 U/L Interval -44.0 to 26.0	26.0 U/L Interval 26.0 to 26.0
Change in Levels of Alkaline Phosphatase Week 4	4.0 U/L Interval -9.0 to 25.0	33.5 U/L Interval 17.0 to 50.0
Change in Levels of Alkaline Phosphatase Week 6	8.0 U/L Interval -40.0 to 42.0	22.0 U/L Interval 19.0 to 25.0
Change in Levels of Alkaline Phosphatase Week 10	-0.5 U/L Interval -25.0 to 22.0	40.0 U/L Interval 19.0 to 61.0
Change in Levels of Alkaline Phosphatase Week 14	-15.0 U/L Interval -51.0 to 45.0	33.0 U/L Interval 17.0 to 49.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Total protein

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Total Protein Day 1	0.25 g/dL Interval -0.2 to 0.4	0.30 g/dL Interval 0.2 to 0.4
Change in Levels of Total Protein Day 2	0.25 g/dL Interval -0.2 to 0.4	0.25 g/dL Interval 0.2 to 0.3
Change in Levels of Total Protein Day 3	0.20 g/dL Interval -0.4 to 0.7	0.35 g/dL Interval 0.2 to 0.5
Change in Levels of Total Protein Day 5	0.15 g/dL Interval -0.5 to 0.6	0.00 g/dL Interval -0.1 to 0.1
Change in Levels of Total Protein Day 8	0.50 g/dL Interval 0.2 to 0.8	0.65 g/dL Interval 0.4 to 0.9
Change in Levels of Total Protein Week 2	-0.05 g/dL Interval -0.2 to 0.4	0.10 g/dL Interval 0.1 to 0.1
Change in Levels of Total Protein Week 4	0.05 g/dL Interval -0.4 to 0.6	-0.10 g/dL Interval -0.4 to 0.2
Change in Levels of Total Protein Week 6	-0.10 g/dL Interval -0.4 to 0.2	0.05 g/dL Interval -0.4 to 0.5
Change in Levels of Total Protein Week 10	0.4 g/dL Interval 0.3 to 0.7	0.15 g/dL Interval -0.1 to 0.4
Change in Levels of Total Protein Week 14	0.10 g/dL Interval -0.2 to 0.4	0.55 g/dL Interval 0.2 to 0.9

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Albumin

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Albumin Day 1	0.10 g/dL Interval -0.2 to 0.2	0.35 g/dL Interval 0.3 to 0.4
Change in Levels of Albumin Day 2	0.05 g/dL Interval 0.0 to 0.2	0.30 g/dL Interval 0.3 to 0.3
Change in Levels of Albumin Day 3	-0.15 g/dL Interval -0.2 to 0.2	0.20 g/dL Interval 0.1 to 0.3
Change in Levels of Albumin Day 5	0.00 g/dL Interval -0.1 to 0.2	0.20 g/dL Interval 0.2 to 0.2
Change in Levels of Albumin Day 8	0.30 g/dL Interval 0.2 to 0.6	0.45 g/dL Interval 0.3 to 0.6
Change in Levels of Albumin Week 2	-0.05 g/dL Interval -0.3 to 0.1	0.00 g/dL Interval 0.0 to 0.0
Change in Levels of Albumin Week 4	0.10 g/dL Interval -0.6 to 0.3	0.10 g/dL Interval -0.1 to 0.3
Change in Levels of Albumin Week 6	-0.10 g/dL Interval -0.4 to 0.0	0.00 g/dL Interval -0.3 to 0.3
Change in Levels of Albumin Week 10	0.30 g/dL Interval 0.2 to 0.4	0.05 g/dL Interval -0.1 to 0.2
Change in Levels of Albumin Week 14	0.00 g/dL Interval -0.2 to 0.1	0.25 g/dL Interval 0.0 to 0.5

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Prothrombin Time/International Normalized Ratio (PTINR)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Prothrombin Time/International Normalized Ratio Day 1	0.065 Unitless Interval 0.02 to 0.1	-0.005 Unitless Interval -0.05 to 0.04
Change in Levels of Prothrombin Time/International Normalized Ratio Day 2	0.045 Unitless Interval -0.05 to 0.1	-0.035 Unitless Interval -0.08 to 0.01
Change in Levels of Prothrombin Time/International Normalized Ratio Day 3	0.075 Unitless Interval 0.0 to 0.11	0.010 Unitless Interval -0.05 to 0.07
Change in Levels of Prothrombin Time/International Normalized Ratio Day 5	0.015 Unitless Interval -0.03 to 0.06	-0.025 Unitless Interval -0.06 to 0.01
Change in Levels of Prothrombin Time/International Normalized Ratio Day 8	-0.005 Unitless Interval -0.03 to 0.08	-0.055 Unitless Interval -0.1 to -0.01
Change in Levels of Prothrombin Time/International Normalized Ratio Week 2	-0.020 Unitless Interval -0.07 to 0.07	-0.060 Unitless Interval -0.06 to -0.06
Change in Levels of Prothrombin Time/International Normalized Ratio Week 4	-0.040 Unitless Interval -0.1 to 0.09	-0.055 Unitless Interval -0.11 to 0.0
Change in Levels of Prothrombin Time/International Normalized Ratio Week 6	-0.050 Unitless Interval -0.09 to 0.07	-0.050 Unitless Interval -0.09 to -0.01
Change in Levels of Prothrombin Time/International Normalized Ratio Week 10	-0.030 Unitless Interval -0.08 to 0.06	-0.065 Unitless Interval -0.14 to 0.01
Change in Levels of Prothrombin Time/International Normalized Ratio Week 14	-0.030 Unitless Interval -0.05 to 0.05	-0.085 Unitless Interval -0.17 to 0.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Fibrinogen

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Fibrinogen Day 1	6.0 mg/dL Interval -48.0 to 18.0	49.5 mg/dL Interval 40.0 to 59.0
Change in Levels of Fibrinogen Day 2	53.0 mg/dL Interval -38.0 to 125.0	103.5 mg/dL Interval 92.0 to 115.0
Change in Levels of Fibrinogen Day 3	60.0 mg/dL Interval -184.0 to 190.0	-97.0 mg/dL Interval -110.0 to -84.0
Change in Levels of Fibrinogen Day 5	75.5 mg/dL Interval -7.0 to 174.0	6.0 mg/dL Interval -38.0 to 50.0
Change in Levels of Fibrinogen Day 8	93.5 mg/dL Interval -134.0 to 145.0	84.0 mg/dL Interval 53.0 to 115.0
Change in Levels of Fibrinogen Week 2	-5.5 mg/dL Interval -135.0 to 78.0	2.5 mg/dL Interval -5.0 to 10.0
Change in Levels of Fibrinogen Week 4	-5.5 mg/dL Interval -135.0 to 78.0	2.5 mg/dL Interval -5.0 to 10.0
Change in Levels of Fibrinogen Week 6	-14.0 mg/dL Interval -34.0 to 20.0	23.5 mg/dL Interval -3.0 to 50.0
Change in Levels of Fibrinogen Week 10	10.5 mg/dL Interval -6.0 to 102.0	-5.0 mg/dL Interval -19.0 to 9.0
Change in Levels of Fibrinogen Week 14	-43.0 mg/dL Interval -122.0 to 86.0	7.5 mg/dL Interval -9.0 to 24.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Complement C3

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Complement C3 Day 1	4.0 mg/dL Interval -2.0 to 13.0	11.5 mg/dL Interval 8.0 to 15.0
Change in Levels of Complement C3 Day 2	3.5 mg/dL Interval -1.0 to 16.0	4.5 mg/dL Interval 3.0 to 6.0
Change in Levels of Complement C3 Day 3	12.5 mg/dL Interval -3.0 to 30.0	9.0 mg/dL Interval 5.0 to 13.0
Change in Levels of Complement C3 Day 5	14.5 mg/dL Interval -4.0 to 40.0	3.0 mg/dL Interval 0.0 to 6.0
Change in Levels of Complement C3 Day 8	20.0 mg/dL Interval 4.0 to 32.0	16.5 mg/dL Interval 8.0 to 25.0
Change in Levels of Complement C3 Week 2	10.5 mg/dL Interval 0.0 to 18.0	3.0 mg/dL Interval 3.0 to 3.0
Change in Levels of Complement C3 Week 4	0.5 mg/dL Interval -8.0 to 17.0	3.0 mg/dL Interval -1.0 to 7.0
Change in Levels of Complement C3 Week 6	-1.0 mg/dL Interval -8.0 to 8.0	-0.5 mg/dL Interval -7.0 to 6.0
Change in Levels of Complement C3 Week 10	6.5 mg/dL Interval 2.0 to 20.0	4.5 mg/dL Interval -1.0 to 10.0
Change in Levels of Complement C3 Week 14	-9.0 mg/dL Interval -15.0 to 14.0	5.5 mg/dL Interval -5.0 to 16.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Creatinine

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Creatinine Day 1	0.030 mg/dL Interval -0.03 to 0.08	0.095 mg/dL Interval 0.05 to 0.14
Change in Levels of Creatinine Day 2	0.010 mg/dL Interval 0.01 to 0.02	0.020 mg/dL Interval -0.04 to 0.08
Change in Levels of Creatinine Day 3	0.010 mg/dL Interval -0.04 to 0.05	0.025 mg/dL Interval -0.05 to 0.1
Change in Levels of Creatinine Day 5	0.055 mg/dL Interval -0.02 to 0.07	0.035 mg/dL Interval 0.0 to 0.07
Change in Levels of Creatinine Day 8	-0.005 mg/dL Interval -0.03 to 0.04	-0.005 mg/dL Interval -0.04 to 0.03
Change in Levels of Creatinine Week 2	-0.035 mg/dL Interval -0.09 to 0.14	-0.060 mg/dL Interval -0.06 to -0.06
Change in Levels of Creatinine Week 4	-0.040 mg/dL Interval -0.12 to 0.07	0.025 mg/dL Interval -0.01 to 0.06
Change in Levels of Creatinine Week 6	-0.030 mg/dL Interval -0.11 to 0.17	0.010 mg/dL Interval -0.07 to 0.09
Change in Levels of Creatinine Week 10	-0.020 mg/dL Interval -0.1 to 0.13	-0.010 mg/dL Interval -0.07 to 0.05
Change in Levels of Creatinine Week 14	0.010 mg/dL Interval -0.13 to 0.12	0.125 mg/dL Interval 0.03 to 0.22

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of C-reactive protein (CRP)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of C-reactive Protein Day 1	0.000 mg/dL Interval -0.03 to 0.05	-0.015 mg/dL Interval -0.05 to 0.02
Change in Levels of C-reactive Protein Day 2	0.105 mg/dL Interval -0.01 to 0.94	0.030 mg/dL Interval -0.08 to 0.14
Change in Levels of C-reactive Protein Day 3	0.645 mg/dL Interval -0.01 to 2.28	0.025 mg/dL Interval -0.09 to 0.14
Change in Levels of C-reactive Protein Day 5	0.155 mg/dL Interval -0.01 to 0.54	-0.035 mg/dL Interval -0.08 to 0.01
Change in Levels of C-reactive Protein Day 8	0.050 mg/dL Interval 0.0 to 0.13	-0.025 mg/dL Interval -0.08 to 0.03
Change in Levels of C-reactive Protein Week 2	0.010 mg/dL Interval -0.02 to 0.04	-0.050 mg/dL Interval -0.05 to -0.05
Change in Levels of C-reactive Protein Week 4	0.000 mg/dL Interval -0.04 to 0.36	0.065 mg/dL Interval 0.0 to 0.13
Change in Levels of C-reactive Protein Week 6	0.030 mg/dL Interval -0.02 to 0.07	-0.060 mg/dL Interval -0.1 to -0.02
Change in Levels of C-reactive Protein Week10	0.005 mg/dL Interval -0.06 to 0.07	-0.025 mg/dL Interval -0.08 to 0.03
Change in Levels of C-reactive Protein Week 14	-0.010 mg/dL Interval -0.01 to 0.1	-0.025 mg/dL Interval -0.09 to 0.04

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Sodium

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Sodium Day 1	-1.0 mEq/L Interval -4.0 to 2.0	3.5 mEq/L Interval 3.0 to 4.0
Change in Levels of Sodium Day 2	-0.5 mEq/L Interval -2.0 to 1.0	1.5 mEq/L Interval 1.0 to 2.0
Change in Levels of Sodium Day 3	-1.5 mEq/L Interval -3.0 to 0.0	2.0 mEq/L Interval 2.0 to 2.0
Change in Levels of Sodium Day 5	0.00 mEq/L Interval -4.0 to 1.0	2.5 mEq/L Interval 2.0 to 3.0
Change in Levels of Sodium Day 8	0.0 mEq/L Interval -4.0 to 2.0	2.0 mEq/L Interval 2.0 to 2.0
Change in Levels of Sodium Week 2	-0.5 mEq/L Interval -3.0 to 3.0	1.0 mEq/L Interval 1.0 to 1.0
Change in Levels of Sodium Week 4	-0.5 mEq/L Interval -6.0 to 3.0	3.5 mEq/L Interval 3.0 to 4.0
Change in Levels of Sodium Week 6	0.0 mEq/L Interval -3.0 to 2.0	5.0 mEq/L Interval 3.0 to 7.0
Change in Levels of Sodium Week 10	-1.5 mEq/L Interval -5.0 to 2.0	0.5 mEq/L Interval 0.0 to 1.0
Change in Levels of Sodium Week 14	-1.0 mEq/L Interval -2.0 to 2.0	3.5 mEq/L Interval 0.0 to 7.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Potassium

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Potassium Day 1	0.00 mEq/L Interval -0.5 to 0.2	0.60 mEq/L Interval 0.5 to 0.7
Change in Levels of Potassium Day 2	0.00 mEq/L Interval -0.8 to 0.4	0.15 mEq/L Interval 0.0 to 0.3
Change in Levels of Potassium Day 3	0.15 mEq/L Interval -0.5 to 0.3	0.40 mEq/L Interval 0.0 to 0.8
Change in Levels of Potassium Day 5	0.15 mEq/L Interval 0.1 to 0.4	0.00 mEq/L Interval -0.1 to 0.1
Change in Levels of Potassium Day 8	-0.15 mEq/L Interval -0.6 to 0.2	-0.25 mEq/L Interval -0.4 to -0.1
Change in Levels of Potassium Week 2	0.15 mEq/L Interval -0.2 to 0.3	0.10 mEq/L Interval 0.1 to 0.1
Change in Levels of Potassium Week 4	0.00 mEq/L Interval -0.2 to 0.3	0.35 mEq/L Interval 0.2 to 0.5
Change in Levels of Potassium Week 6	0.00 mEq/L Interval -0.1 to 0.1	0.40 mEq/L Interval 0.1 to 0.7
Change in Levels of Potassium Week 10	0.05 mEq/L Interval -0.4 to 0.5	-0.20 mEq/L Interval -0.3 to -0.1
Change in Levels of Potassium Week 14	0.00 mEq/L Interval -0.02 to 0.3	0.30 mEq/L Interval 0.0 to 0.6

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Calcium

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Calcium Day 1	-0.05 mg/dL Interval -0.8 to 0.2	0.40 mg/dL Interval 0.3 to 0.5
Change in Levels of Calcium Day 2	0.00 mg/dL Interval -0.6 to 0.3	0.15 mg/dL Interval 0.1 to 0.2
Change in Levels of Calcium Day 3	-0.10 mg/dL Interval -0.6 to 0.7	0.30 mg/dL Interval 0.2 to 0.4
Change in Levels of Calcium Day 5	0.05 mg/dL Interval -0.5 to 0.5	0.20 mg/dL Interval 0.1 to 0.3
Change in Levels of Calcium Day 8	0.15 mg/dL Interval -0.1 to 0.4	0.10 mg/dL Interval -0.1 to 0.3
Change in Levels of Calcium Week 2	-0.05 mg/dL Interval -0.6 to 0.4	0.10 mg/dL Interval 0.1 to 0.1
Change in Levels of Calcium Week 4	-0.15 mg/dL Interval -0.9 to 0.3	-0.05 mg/dL Interval -0.1 to 0.0
Change in Levels of Calcium Week 6	-0.40 mg/dL Interval -1.0 to 0.5	0.15 mg/dL Interval -0.4 to 0.7
Change in Levels of Calcium Week 10	0.30 mg/dL Interval 0.0 to 0.6	0.00 mg/dL Interval -0.1 to 0.1
Change in Levels of Calcium Week 14	0.00 mg/dL Interval -0.3 to 0.5	0.55 mg/dL Interval 0.2 to 0.9

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Glucose

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Glucose Day 1	-9.0 mg/dL Interval -13.0 to 0.0	-3.0 mg/dL Interval -5.0 to -1.0
Change in Levels of Glucose Day 2	-1.0 mg/dL Interval -5.0 to 4.0	0.5 mg/dL Interval -1.0 to 2.0
Change in Levels of Glucose Day 3	0.5 mg/dL Interval -6.0 to 6.0	3.0 mg/dL Interval 0.0 to 6.0
Change in Levels of Glucose Day 5	-4.5 mg/dL Interval -7.0 to 0.0	-2.0 mg/dL Interval -5.0 to 1.0
Change in Levels of Glucose Day 8	-7.5 mg/dL Interval -14.0 to -4.0	0.0 mg/dL Interval -3.0 to 3.0
Change in Levels of Glucose Week 2	-2.0 mg/dL Interval -11.0 to 0.0	2.0 mg/dL Interval 2.0 to 2.0
Change in Levels of Glucose Week 4	-1.0 mg/dL Interval -6.0 to 10.0	3.0 mg/dL Interval 0.0 to 6.0
Change in Levels of Glucose Week 6	0.0 mg/dL Interval 0.0 to 5.0	4.5 mg/dL Interval 4.0 to 5.0
Change in Levels of Glucose Week 10	1.5 mg/dL Interval -4.0 to 4.0	0.5 mg/dL Interval -1.0 to 2.0
Change in Levels of Glucose Week 14	1.0 mg/dL Interval -5.0 to 9.0	0.5 mg/dL Interval -2.0 to 3.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of HDL

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of HDL Day 1	-2.0 mg/dL Interval -8.0 to 3.0	0.0 mg/dL Interval 0.0 to 0.0
Change in Levels of HDL Day 2	-3.5 mg/dL Interval -8.0 to 0.0	-1.0 mg/dL Interval -2.0 to 0.0
Change in Levels of HDL Day 3	-4.0 mg/dL Interval -11.0 to 0.0	1.0 mg/dL Interval -1.0 to 3.0
Change in Levels of HDL Day 5	-8.0 mg/dL Interval -14.0 to -2.0	-1.5 mg/dL Interval -4.0 to 1.0
Change in Levels of HDL Day 8	-7.5 mg/dL Interval -14.0 to -2.0	2.5 mg/dL Interval 0.0 to 5.0
Change in Levels of HDL Week 2	-7.0 mg/dL Interval -9.0 to 2.0	0.0 mg/dL Interval 0.0 to 0.0
Change in Levels of HDL Week 4	-2.0 mg/dL Interval -8.0 to 11.0	-2.0 mg/dL Interval -5.0 to 1.0
Change in Levels of HDL Week 6	-5.0 mg/dL Interval -7.0 to 6.0	-1.0 mg/dL Interval -2.0 to 0.0
Change in Levels of HDL Week 10	0.5 mg/dL Interval -4.0 to 6.0	-0.5 mg/dL Interval -3.0 to 2.0
Change in Levels of HDL Week 14	-5.0 mg/dL Interval -10.0 to -2.0	0.5 mg/dL Interval -2.0 to 3.0

SECONDARY outcome

Timeframe: Baselilne, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of LDL

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of LDL Day 1	11.0 mg/dL Interval -5.0 to 13.0	16.5 mg/dL Interval 15.0 to 18.0
Change in Levels of LDL Day 2	5.5 mg/dL Interval -13.0 to 17.0	17.0 mg/dL Interval 13.0 to 21.0
Change in Levels of LDL Day 3	3.5 mg/dL Interval -32.0 to 23.0	19.5 mg/dL Interval 14.0 to 25.0
Change in Levels of LDL Day 5	3.0 mg/dL Interval -40.0 to 15.0	7.5 mg/dL Interval 5.0 to 10.0
Change in Levels of LDL Day 8	7.0 mg/dL Interval -25.0 to 22.0	13.0 mg/dL Interval 1.0 to 25.0
Change in Levels of LDL Week 2	-7.0 mg/dL Interval -25.0 to 19.0	17.0 mg/dL Interval 17.0 to 17.0
Change in Levels of LDL Week 4	2.0 mg/dL Interval -22.0 to 19.0	0.0 mg/dL Interval -7.0 to 7.0
Change in Levels of LDL Week 6	3.0 mg/dL Interval -24.0 to 39.0	3.5 mg/dL Interval -3.0 to 10.0
Change in Levels of LDL Week 10	9.0 mg/dL Interval -2.0 to 32.0	3.5 mg/dL Interval -15.0 to 22.0
Change in Levels of LDL Week 14	-8.0 mg/dL Interval -11.0 to 8.0	19.5 mg/dL Interval 7.0 to 32.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of BUN/Urea haematology

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of BUN/Urea Haematology Day 1	-1.35 mg/dL Interval -3.6 to 0.0	-1.60 mg/dL Interval -2.1 to -1.1
Change in Levels of BUN/Urea Haematology Day 2	-0.55 mg/dL Interval -2.8 to 1.7	2.55 mg/dL Interval 0.9 to 4.2
Change in Levels of BUN/Urea Haematology Day 3	-1.05 mg/dL Interval -2.2 to -0.4	1.30 mg/dL Interval -1.1 to 3.7
Change in Levels of BUN/Urea Haematology Day 5	0.80 mg/dL Interval -1.7 to 1.6	2.00 mg/dL Interval 0.3 to 3.7
Change in Levels of BUN/Urea Haematology Day 8	-2.05 mg/dL Interval -5.3 to 2.3	-1.50 mg/dL Interval -3.0 to 0.0
Change in Levels of BUN/Urea Haematology Week 2	-1.90 mg/dL Interval -2.6 to 2.8	-2.30 mg/dL Interval -2.3 to -2.3
Change in Levels of BUN/Urea Haematology Week 4	-1.40 mg/dL Interval -6.6 to 4.4	3.10 mg/dL Interval 0.8 to 5.4
Change in Levels of BUN/Urea Haematology Week 6	-2.20 mg/dL Interval -4.4 to 4.9	-1.25 mg/dL Interval -2.7 to 0.2
Change in Levels of BUN/Urea Haematology Week 10	0.95 mg/dL Interval -6.2 to 2.4	-1.15 mg/dL Interval -4.8 to 2.5
Change in Levels of BUN/Urea Haematology Week 14	-1.70 mg/dL Interval -2.7 to -1.4	2.75 mg/dL Interval -0.9 to 6.4

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Hemoglobin

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Hemoglobin Day 1	0.4 g/dL Interval -0.6 to 0.6	0.75 g/dL Interval 0.6 to 0.9
Change in Levels of Hemoglobin Day 2	0.35 g/dL Interval -0.2 to 0.8	0.55 g/dL Interval 0.3 to 0.8
Change in Levels of Hemoglobin Day 3	0.20 g/dL Interval -0.5 to 0.7	0.45 g/dL Interval 0.0 to 0.9
Change in Levels of Hemoglobin Day 5	0.25 g/dL Interval -0.4 to 1.1	0.25 g/dL Interval 0.1 to 0.4
Change in Levels of Hemoglobin Day 8	0.75 g/dL Interval 0.5 to 0.9	0.7 g/dL Interval 0.4 to 1.0
Change in Levels of Hemoglobin Week 2	-0.25 g/dL Interval -0.7 to 0.7	0.10 g/dL Interval 0.1 to 0.1
Change in Levels of Hemoglobin Week 4	0.00 g/dL Interval -0.9 to 0.8	0.20 g/dL Interval -0.2 to 0.6
Change in Levels of Hemoglobin Week 6	-0.20 g/dL Interval -0.6 to 0.5	0.05 g/dL Interval -0.5 to 0.6
Change in Levels of Hemoglobin Week 10	0.90 g/dL Interval 0.3 to 1.5	0.65 g/dL Interval 0.5 to 0.8
Change in Levels of Hemoglobin Week 14	0.20 g/dL Interval -0.4 to 0.7	1.25 g/dL Interval 1.2 to 1.3

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Hematocrit

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Hematocrit Day 1	1.6 % of red blood cells in blood Interval -1.6 to 2.8	2.80 % of red blood cells in blood Interval 2.3 to 3.3
Change in Levels of Hematocrit Day 2	1.10 % of red blood cells in blood Interval -0.3 to 3.0	2.10 % of red blood cells in blood Interval 1.5 to 2.7
Change in Levels of Hematocrit Day 3	0.70 % of red blood cells in blood Interval -1.3 to 2.9	1.55 % of red blood cells in blood Interval 0.7 to 2.4
Change in Levels of Hematocrit Day 5	0.90 % of red blood cells in blood Interval -1.2 to 3.6	0.7 % of red blood cells in blood Interval 0.2 to 1.2
Change in Levels of Hematocrit Day 8	2.70 % of red blood cells in blood Interval 1.7 to 3.5	2.15 % of red blood cells in blood Interval 1.3 to 3.0
Change in Levels of Hematocrit Week 2	-0.40 % of red blood cells in blood Interval -1.9 to 4.2	0.3 % of red blood cells in blood Interval 0.3 to 0.3
Change in Levels of Hematocrit Week 4	0.15 % of red blood cells in blood Interval -2.2 to 2.6	0.95 % of red blood cells in blood Interval -0.1 to 2.0
Change in Levels of Hematocrit Week 6	1.00 % of red blood cells in blood Interval -1.1 to 1.9	0.85 % of red blood cells in blood Interval -1.6 to 3.3
Change in Levels of Hematocrit Week 10	3.05 % of red blood cells in blood Interval 1.2 to 4.0	2.70 % of red blood cells in blood Interval 2.1 to 3.3
Change in Levels of Hematocrit Week 14	0.1 % of red blood cells in blood Interval -1.4 to 1.4	2.45 % of red blood cells in blood Interval 2.4 to 2.5

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Platelet count

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Platelet Count Day 1	0.40 x10^10/L Interval 0.2 to 1.2	1.20 x10^10/L Interval 1.1 to 1.3
Change in Levels of Platelet Count Day 2	-0.15 x10^10/L Interval -1.3 to 0.9	1.10 x10^10/L Interval 0.9 to 1.3
Change in Levels of Platelet Count Day 3	-0.1 x10^10/L Interval -2.3 to 0.3	1.40 x10^10/L Interval 1.4 to 1.4
Change in Levels of Platelet Count Day 5	0.05 x10^10/L Interval -0.4 to 1.6	1.8 x10^10/L Interval 1.6 to 2.0
Change in Levels of Platelet Count Day 8	1.8 x10^10/L Interval 0.3 to 5.7	3.70 x10^10/L Interval 3.2 to 4.2
Change in Levels of Platelet Count Week 2	0.70 x10^10/L Interval -0.6 to 3.1	2.30 x10^10/L Interval 2.3 to 2.3
Change in Levels of Platelet Count Week 4	0.05 x10^10/L Interval -0.2 to 3.3	1.55 x10^10/L Interval 0.7 to 2.4
Change in Levels of Platelet Count Week 6	0.4 x10^10/L Interval -2.7 to 1.6	3.10 x10^10/L Interval 2.8 to 3.4
Change in Levels of Platelet Count Week 10	0.5 x10^10/L Interval -0.5 to 4.0	2.40 x10^10/L Interval 1.6 to 3.2
Change in Levels of Platelet Count Week 14	-0.10 x10^10/L Interval -3.2 to 1.7	4.65 x10^10/L Interval 4.1 to 5.2

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Neutrophils

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Neutrophils Day 1	-0.30 % of white blood cells Interval -4.5 to 3.6	0.35 % of white blood cells Interval -7.5 to 8.2
Change in Levels of Neutrophils Day 2	17.45 % of white blood cells Interval -0.5 to 21.1	-0.40 % of white blood cells Interval -6.0 to 5.2
Change in Levels of Neutrophils Day 3	9.70 % of white blood cells Interval -1.1 to 12.2	-0.85 % of white blood cells Interval -3.0 to 1.3
Change in Levels of Neutrophils Day 5	0.75 % of white blood cells Interval -6.4 to 9.2	-16.25 % of white blood cells Interval -30.8 to -1.7
Change in Levels of Neutrophils Day 8	1.65 % of white blood cells Interval -4.0 to 6.8	1.30 % of white blood cells Interval 0.7 to 1.9
Change in Levels of Neutrophils Week 2	2.50 % of white blood cells Interval -2.1 to 17.9	5.10 % of white blood cells Interval 5.1 to 5.1
Change in Levels of Neutrophils Week 4	-1.05 % of white blood cells Interval -4.1 to 7.4	2.10 % of white blood cells Interval -1.6 to 5.8
Change in Levels of Neutrophils Week 6	-4.10 % of white blood cells Interval -9.5 to 8.3	0.55 % of white blood cells Interval -1.9 to 3.0
Change in Levels of Neutrophils Week 10	1.45 % of white blood cells Interval -9.7 to 8.6	0.95 % of white blood cells Interval -1.4 to 3.3
Change in Levels of Neutrophils Week 14	-5.40 % of white blood cells Interval -10.5 to 1.7	1.65 % of white blood cells Interval -4.7 to 8.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Red blood cells

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Red Blood Cells Day 1	18 cellsx10^10/L Interval -20.0 to 30.0	27.0 cellsx10^10/L Interval 25.0 to 29.0
Change in Levels of Red Blood Cells Day 2	13.5 cellsx10^10/L Interval -7.0 to 29.0	20.0 cellsx10^10/L Interval 13.0 to 27.0
Change in Levels of Red Blood Cells Day 3	9.0 cellsx10^10/L Interval -15.0 to 33.0	15.0 cellsx10^10/L Interval 6.0 to 24.0
Change in Levels of Red Blood Cells Day 5	12.0 cellsx10^10/L Interval -14.0 to 43.0	8.5 cellsx10^10/L Interval 3.0 to 14.0
Change in Levels of Red Blood Cells Day 8	29.5 cellsx10^10/L Interval 21.0 to 33.0	23.5 cellsx10^10/L Interval 14.0 to 33.0
Change in Levels of Red Blood Cells Week 2	-4.0 cellsx10^10/L Interval -24.0 to 27.0	0.0 cellsx10^10/L Interval 0.0 to 0.0
Change in Levels of Red Blood Cells Week 4	-1.0 cellsx10^10/L Interval -27.0 to 24.0	2.0 cellsx10^10/L Interval -9.0 to 13.0
Change in Levels of Red Blood Cells Week 6	-6.0 cellsx10^10/L Interval -23.0 to 11.0	-0.5 cellsx10^10/L Interval -23.0 to 22.0
Change in Levels of Red Blood Cells Week 10	23.0 cellsx10^10/L Interval -1.0 to 32.0	20.0 cellsx10^10/L Interval 13.0 to 27.0
Change in Levels of Red Blood Cells Week 14	-6.0 cellsx10^10/L Interval -25.0 to -2.0	18.5 cellsx10^10/L Interval 16.0 to 21.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Immunoglobulin levels (IgG)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Immunoglobulin Levels Day 1	69.5 mg/dL Interval -60.0 to 100.0	119.5 mg/dL Interval 119.0 to 120.0
Change in Levels of Immunoglobulin Levels Day 2	28.5 mg/dL Interval 7.0 to 78.0	72.0 mg/dL Interval 66.0 to 78.0
Change in Levels of Immunoglobulin Levels Day 3	5.0 mg/dL Interval -41.0 to 67.0	78 mg/dL Interval 62.0 to 94.0
Change in Levels of Immunoglobulin Levels Day 5	6.0 mg/dL Interval -79.0 to 92.0	16.0 mg/dL Interval 5.0 to 27.0
Change in Levels of Immunoglobulin Levels Day 8	125.5 mg/dL Interval 105.0 to 148.0	156.5 mg/dL Interval 76.0 to 237.0
Change in Levels of Immunoglobulin Levels Week 2	14.5 mg/dL Interval -52.0 to 105.0	48.0 mg/dL Interval 48.0 to 48.0
Change in Levels of Immunoglobulin Levels Week 4	29.5 mg/dL Interval -63.0 to 182.0	34.5 mg/dL Interval 1.0 to 68.0
Change in Levels of Immunoglobulin Levels Week 6	2.0 mg/dL Interval -82.0 to 135.0	36.0 mg/dL Interval -40.0 to 112.0
Change in Levels of Immunoglobulin Levels Week 10	98.0 mg/dL Interval 37.0 to 267.0	49.5 mg/dL Interval -14.0 to 113.0
Change in Levels of Immunoglobulin Levels Week 14	1.0 mg/dL Interval -99.0 to 185.0	89.5 mg/dL Interval -24.0 to 203.0

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of patients analyzed are the number of available samples.

Change from baseline in Activated Partial Thromboplastin Clotting Time (APTT)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Coagulation Profile Day 1	0.85 sec Interval 0.4 to 2.1	0.4 sec Interval 0.2 to 0.6
Change in Levels of Coagulation Profile Day 2	-0.30 sec Interval -1.2 to 2.6	-0.65 sec Interval -0.9 to -0.4
Change in Levels of Coagulation Profile Day 3	0.4 sec Interval -0.9 to 2.2	1.80 sec Interval 1.2 to 2.4
Change in Levels of Coagulation Profile Day 5	-1.40 sec Interval -2.0 to 2.1	-0.80 sec Interval -1.4 to -0.2
Change in Levels of Coagulation Profile Day 8	1.40 sec Interval -0.8 to 3.5	0.30 sec Interval 0.0 to 0.6
Change in Levels of Coagulation Profile Week 2	-0.55 sec Interval -1.5 to 2.1	0.5 sec Interval 0.5 to 0.5
Change in Levels of Coagulation Profile Week 4	-0.15 sec Interval -2.0 to 2.8	-2.25 sec Interval -2.3 to -2.2
Change in Levels of Coagulation Profile Week 6	-0.90 sec Interval -2.4 to 1.3	-0.50 sec Interval -1.3 to 0.3
Change in Levels of Coagulation Profile Week 10	1.50 sec Interval -1.9 to 3.8	-2.60 sec Interval -3.5 to -1.7
Change in Levels of Coagulation Profile Week 14	-1.80 sec Interval -3.1 to 3.5	-1.25 sec Interval -3.4 to 0.9

SECONDARY outcome

Timeframe: From Day 1 to Week 14

Population: Number of analyzed are the number of available samples

Presence of anti-drug antibodies (ADA) and neutralizing antibodies (nAb)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Presence of Anti-drug Antibodies and Neutralizing Antibodies Baseline	0 Participants	—
Presence of Anti-drug Antibodies and Neutralizing Antibodies Week 14	0 Participants	—

SECONDARY outcome

Timeframe: Baseline, Days 1,2,3,5,8, Weeks 2,4,6,10, and Study Completion Visit (Week 14)

Population: Number of analyzed are the number of available samples

Change from baseline in levels of Complement C4

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 Participants Saline i.v. infusion Saline: Saline single i.v infusion
Change in Levels of Complement C4 Day 1	0.0 mg/dL Interval -1.0 to 2.0	2.5 mg/dL Interval 2.0 to 3.0
Change in Levels of Complement C4 Day 2	1.0 mg/dL Interval -1.0 to 4.0	2.0 mg/dL Interval 1.0 to 3.0
Change in Levels of Complement C4 Day 3	3.0 mg/dL Interval -1.0 to 8.0	3.0 mg/dL Interval 1.0 to 5.0
Change in Levels of Complement C4 Day 5	4.5 mg/dL Interval -1.0 to 12.0	2.5 mg/dL Interval 0.0 to 5.0
Change in Levels of Complement C4 Day 8	6.0 mg/dL Interval 1.0 to 11.0	6.0 mg/dL Interval 3.0 to 9.0
Change in Levels of Complement C4 Week 2	2.0 mg/dL Interval 1.0 to 4.0	4.0 mg/dL Interval 4.0 to 4.0
Change in Levels of Complement C4 Week 4	0.0 mg/dL Interval -4.0 to 3.0	-1.0 mg/dL Interval -3.0 to 1.0
Change in Levels of Complement C4 Week 6	-1.0 mg/dL Interval -2.0 to 3.0	1.5 mg/dL Interval -2.0 to 5.0
Change in Levels of Complement C4 Week 10	-1.5 mg/dL Interval -3.0 to 0.0	-1.5 mg/dL Interval -3.0 to 0.0
Change in Levels of Complement C4 Week 14	-4.0 mg/dL Interval -7.0 to 6.0	1.5 mg/dL Interval -3.0 to 6.0

SECONDARY outcome

Timeframe: From Day 1 to Week 14

Presence of neutralizing antibodies (nAb)

Outcome measures

Outcome measures
Measure	Emapalumab n=6 Participants Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo Saline i.v. infusion Saline: Saline single i.v infusion
Presence of Neutralizing Antibodies	NA Participants None of the subjects had positive anti-drug-antibody result, so this analyse was not done	—

Adverse Events

Emapalumab

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Emapalumab n=6 participants at risk Emapalumab i.v infusion NI-0501: Emapalumab single i.v infusion (1 mg/kg)	Placebo n=2 participants at risk Saline i.v. infusion Saline: Saline single i.v infusion
Investigations C-reactive protein increased	50.0% 3/6 • Number of events 3 • Adverse events were collected from start of dosing the IMP up to week 14	0.00% 0/2 • Adverse events were collected from start of dosing the IMP up to week 14
General disorders Feeling hot	33.3% 2/6 • Number of events 2 • Adverse events were collected from start of dosing the IMP up to week 14	0.00% 0/2 • Adverse events were collected from start of dosing the IMP up to week 14
General disorders Malaise	33.3% 2/6 • Number of events 2 • Adverse events were collected from start of dosing the IMP up to week 14	0.00% 0/2 • Adverse events were collected from start of dosing the IMP up to week 14
General disorders Pyrexia	16.7% 1/6 • Number of events 1 • Adverse events were collected from start of dosing the IMP up to week 14	0.00% 0/2 • Adverse events were collected from start of dosing the IMP up to week 14
Infections and infestations Nasopharyngitis	16.7% 1/6 • Number of events 1 • Adverse events were collected from start of dosing the IMP up to week 14	0.00% 0/2 • Adverse events were collected from start of dosing the IMP up to week 14
Injury, poisoning and procedural complications Contusion	0.00% 0/6 • Adverse events were collected from start of dosing the IMP up to week 14	50.0% 1/2 • Number of events 1 • Adverse events were collected from start of dosing the IMP up to week 14
Reproductive system and breast disorders Prostatitis	16.7% 1/6 • Number of events 1 • Adverse events were collected from start of dosing the IMP up to week 14	0.00% 0/2 • Adverse events were collected from start of dosing the IMP up to week 14

Additional Information

Veronica Asnaghi

Swedish Orphan Biovitrum AB

Phone: +41 61 508 72 13

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place