Trial Outcomes & Findings for Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT. (NCT NCT04731298)

Results Overview

Serum concentration of C-X-C Motif Chemokine Ligand 9 (CXCL9)

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

From start of treatment to EoS Visit, up to 34 weeks

Results posted on

2023-12-28

Participant Flow

One site was activated which screened a total of 3 subjects (2 enrolled; 1 screen failure). First and last subjects were enrolled 25May2021 and 07Oct2021 respectively.

Participant milestones

Participant milestones
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Allogeneic HSCT (Day 0) STARTED	0	2
Allogeneic HSCT (Day 0) COMPLETED	0	2
Allogeneic HSCT (Day 0) NOT COMPLETED	0	0
Monitoring - Primary GF (Day 1 to 42) STARTED	0	2
Monitoring - Primary GF (Day 1 to 42) COMPLETED	0	2
Monitoring - Primary GF (Day 1 to 42) NOT COMPLETED	0	0
Monitoring - Secondary GF (Up to Day 98) STARTED	0	2
Monitoring - Secondary GF (Up to Day 98) COMPLETED	0	2
Monitoring - Secondary GF (Up to Day 98) NOT COMPLETED	0	0
Follow-up (3 Years From HSCT) STARTED	0	2
Follow-up (3 Years From HSCT) COMPLETED	0	0
Follow-up (3 Years From HSCT) NOT COMPLETED	0	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Follow-up (3 Years From HSCT) Early termination of study.	0	2

Baseline Characteristics

Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)	Total n=2 Participants Total of all reporting groups
Age, Categorical <=18 years	—	2 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	—	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	—	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	—	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	—	2 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	—	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment Canada	—	2 participants n=7 Participants	2 participants n=5 Participants
Underlying disease: Aplastic anemia	—	2 Participants n=7 Participants	2 Participants n=5 Participants
Graft - Type of conditioning Non myeloablative regimen (NMA)	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Graft - Type of conditioning Reduced Intensity Conditioning (RIC)	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Graft - Source of stem cells Peripheral blood stem cell (PBSC)	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Graft - Source of stem cells Bone marrow (BM)	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Graft - Donor type Matched unrelated donor	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Graft - Donor type Mismatched unrelated donor	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Number of transplanted cells	—	290.9 million cells n=7 Participants	290.9 million cells n=5 Participants
ABO incompatibility Major	—	1 Participants n=7 Participants	1 Participants n=5 Participants
ABO incompatibility No incompatibility	—	1 Participants n=7 Participants	1 Participants n=5 Participants
Mean CXCL9 concentration (prior to HSCT)	—	203.50 pg/mL n=7 Participants	203.5 pg/mL n=5 Participants

PRIMARY outcome

Timeframe: From start of treatment to EoS Visit, up to 34 weeks

Population: No efficacy data was collected since no subject received study treatment.

Serum concentration of C-X-C Motif Chemokine Ligand 9 (CXCL9)

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: From Hematopoietic stem-cell transplantation (HSCT) [Day 0] up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Number of participants with primary graft failure (GF)

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Number of participants with secondary GF

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From start of treatment to EoS Visit, up to 34 weeks

Population: No efficacy data was collected since no subject received study treatment.

Serum concentration of free and total Interferon gamma (IFNγ)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From start of treatment to EoS, up to 34 weeks

Population: No patients enrolled in the emapalumab arm.

Peak emapalumab serum concentration

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From start of treatment to EoS, up to 34 weeks

Population: No patients enrolled in the emapalumab arm.

Concentration just before administration

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Ferritin - serum concentration

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Start of treatment until EoS, up to 34 weeks

Population: No patients enrolled in the emapalumab arm.

Number of participants developing antibodies against emapalumab (antidrug antibodies \[ADA\]) and Neutralizing antibodies (nAb)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No patients enrolled in the emapalumab arm.

Based on unselected leukocytes and based on sorted T cells

Outcome measures

Outcome measures
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Number of Participants With Mixed Donor Chimerism <10% and <20%	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No patients enrolled in the emapalumab arm.

defined as absence of GF or graft support

Outcome measures

Outcome measures
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Number of Participants With Event Free Engraftment	0 Participants	2 Participants

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No patients enrolled in the emapalumab arm.

(grade I to IV)

Outcome measures

Outcome measures
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Number of Participants With Acute and/or Chronic Mild to Severe Graft Versus Host Disease (GvHD)	0 Participants	1 Participants

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No patients enrolled in the emapalumab arm.

Number of participants with engraftment syndrome

Outcome measures

Outcome measures
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Engraftment Syndrome	0 Participants	1 Participants

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No patients enrolled in the emapalumab arm.

Number of patients alive at the end of study.

Outcome measures

Outcome measures
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Survival Rate	—	2 Participants

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in body temperature

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in heart rate

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline systolic and diastolic blood pressure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in body weight

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology: red blood cells (RBC)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology: hematocrit

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology: hemoglobin

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology: platelets

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology: white blood cells (WBC)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology differential: lymphocytes

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology differential: monocytes

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Hematology differential: neutrophils

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Ferritin

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Glucose

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: C-reactive protein

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Sodium

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Potassium

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Chloride

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Calcium

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Magnesium

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Phosphate

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Aspartate aminotransferase (AST)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: alanine aminotransferase (ALT)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: gamma-glutamyl transpeptidase (γGT)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: alkaline phosphatase (ALP)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: lactate dehydrogenase (LDH)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: bilirubin (total, direct and indirect)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: triglycerides

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: cholesterol (total and high-density lipoprotein \[HDL\])

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Albumin

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Creatinine

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Biochemistry: Urea

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Coagulation: activated partial thromboplastin (aPTT)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Coagulation: prothrombin time (PT)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: Glucose

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: Blood

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: Protein

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: Leucocytes

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: Ketones

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: pH

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in Urinalysis: specific gravity

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No patients enrolled in the emapalumab arm.

Outcome measures

Outcome measures
Measure	Emapalumab Treated Patients treated with emapalumab (i.e. patients who met the protocol requirements for initiating emapalumab treatment)	Non-emapalumab Treated n=2 Participants Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Number of Subjects With Change in Donor Chimerism Change < 100%	0 Participants	0 Participants
Number of Subjects With Change in Donor Chimerism Change = 100%	0 Participants	2 Participants

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Change from baseline in HLA antibodies against donor cells

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From HSCT (Day 0) up to study termination, approximately 46 weeks

Population: No efficacy data was collected since no subject received study treatment.

Only in patients presenting malignant disease

Outcome measures

Outcome data not reported

Adverse Events

Emapalumab Treated

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Non-emapalumab Treated

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Emmanuel Monnet

Sobi

Phone: +41225519156

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Emapalumab Treated Patients treated with emapalumab	Non-emapalumab Treated n=2 participants at risk Patients not treated with emapalumab (i.e.patients who did not meet the protocol requirements for initiating emapalumab treatment)
Respiratory, thoracic and mediastinal disorders Hypoxia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Vascular disorders Flushing	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Skin and subcutaneous tissue disorders Rash	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 3 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Gastrointestinal disorders Constipation	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 3 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Metabolism and nutrition disorders Hyperglycemia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Cardiac disorders Bradycardia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Vascular disorders Hypertension	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Investigations Hepatic enzyme increased	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
General disorders Chills	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
General disorders Pyrexia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 5 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Metabolism and nutrition disorders Hypomagnesaemia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Infections and infestations Sepsis	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Nervous system disorders Headache	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Gastrointestinal disorders Nausea	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 3 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Blood and lymphatic system disorders Febrile neutropenia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Gastrointestinal disorders Abdominal pain	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	100.0% 2/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Blood and lymphatic system disorders Thrombocytopenia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Renal and urinary disorders Nephropathy toxic	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Immune system disorders Graft versus host disease in skin	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Skin and subcutaneous tissue disorders Acne	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Respiratory, thoracic and mediastinal disorders Epistaxis	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 3 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Metabolism and nutrition disorders Hypophosphataemia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Metabolism and nutrition disorders Hypervolaemia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 3 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
General disorders Mucosal inflammation	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Psychiatric disorders Insomnia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Infections and infestations Perineal abscess	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Immune system disorders Engraftment syndrome	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Musculoskeletal and connective tissue disorders Myalgia	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 2 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Skin and subcutaneous tissue disorders Pityriasis rosea	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Investigations Salmonella test positive	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
General disorders Injection site pain	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Skin and subcutaneous tissue disorders Hypertrichosis	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Reproductive system and breast disorders Dysmenorrhoea	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Reproductive system and breast disorders Heavy menstrual bleeding	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Immune system disorders Allergy to immunoglobulin therapy	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Skin and subcutaneous tissue disorders Ingrown toe nail	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Investigations Chemokine increase	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Immune system disorders Drug hypersensitivity	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
General disorders Non-cardiac chest pain	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.
Gastrointestinal disorders Diarrhoea	— 0/0 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.	50.0% 1/2 • Number of events 1 • From Day 1 up to study termination, approximately 46 weeks No patients were enrolled in the emapalumab arm.