Trial Outcomes & Findings for A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH) (NCT NCT03311854)

NCT ID: NCT03311854

Last Updated: 2022-05-17

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 14 participants
• Primary outcome timeframe: Up to Week 8
• Results posted on: 2022-05-17

Participant Flow

Participant milestones

Measure	All Treated Population Of the 16 patients who were screened for the study, 14 were enrolled. All 14 patients completed the study; no patient was withdrawn after the start of treatment. All 14 patients received emapalumab and were included in the all treated population.
Overall Study STARTED	14
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)

Baseline characteristics by cohort

Measure	All Enrolled Participants n=14 Participants Baseline data are provided for all participants who were enrolled in the study.
Age, Categorical <=18 years	13 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	9.9 years STANDARD_DEVIATION 6.6 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	11 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	3 Participants n=5 Participants
Region of Enrollment Italy	7 Participants n=5 Participants
Region of Enrollment United Kingdom	2 Participants n=5 Participants
Region of Enrollment Spain	1 Participants n=5 Participants
Region of Enrollment France	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to Week 8

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a mild TEAE	13 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE related to emapalumab	4 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE	13 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a serious TEAE	6 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a severe TEAE	2 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a moderate TEAE	10 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE unrelated to emapalumab	13 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE with an outcome of recovered/resolved	13 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE with an outcome of not recovered/not resolved	3 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE with an outcome of recovering/resolving	1 Participants	—
Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) Number of participants with a TEAE with an outcome of unknown	1 Participants	—

PRIMARY outcome

Timeframe: Up to Week 8

Shifts from baseline in the following MAS-relevant laboratory parameters are reported:

• Leukocytes
• Platelets
• Lactate dehydrogenase (LDH)
• Alanine aminotransferase (ALT)
• Aspartate aminotransferase (AST)
• Ferritin
• C-reactive protein
• Activated partial thromboplastin time (aPTT)
• Prothrombin time
• D-dimer
• Fibrinogen

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Evolution of Laboratory Parameters Leukocytes: From a high value at baseline to a value within the reference range at Week 8	2 Participants	—
Evolution of Laboratory Parameters Leukocytes: From a low value at baseline to a value within the reference range at Week 8	4 Participants	—
Evolution of Laboratory Parameters Leukocytes: From a low value at baseline to a high value at Week 8	2 Participants	—
Evolution of Laboratory Parameters Platelets: From a high value at baseline to a value within the reference range at Week 8	1 Participants	—
Evolution of Laboratory Parameters Platelets: From a low value at baseline to a value within the reference range at Week 8	6 Participants	—
Evolution of Laboratory Parameters Platelets: From a low value at baseline to a high value at Week 8	3 Participants	—
Evolution of Laboratory Parameters LDH: From a high value at baseline to a value within the reference range at Week 8	6 Participants	—
Evolution of Laboratory Parameters ALT: From a high value at baseline to a value within the reference range at Week 8	10 Participants	—
Evolution of Laboratory Parameters ALT: From a high value at baseline to a low value at Week 8	1 Participants	—
Evolution of Laboratory Parameters AST: From a high value at baseline to a value within the reference range at Week 8	11 Participants	—
Evolution of Laboratory Parameters Ferritin: From a high value at baseline to a value within the reference range at Week 8	11 Participants	—
Evolution of Laboratory Parameters Ferritin: From a high value at baseline to a low value at Week 8	2 Participants	—
Evolution of Laboratory Parameters C-reactive protein: From a high value at baseline to a value within the reference range at Week 8	9 Participants	—
Evolution of Laboratory Parameters aPTT: From a high value at baseline to a value within the reference range at Week 8	1 Participants	—
Evolution of Laboratory Parameters aPTT: From a high value at baseline to a low value at Week 8	1 Participants	—
Evolution of Laboratory Parameters aPTT: From a value within the reference range at baseline to a low value at Week 8	3 Participants	—
Evolution of Laboratory Parameters aPTT: From a low value at baseline to a value within the reference range at Week 8	1 Participants	—
Evolution of Laboratory Parameters aPTT: From a low value at baseline to a high value at Week 8	1 Participants	—
Evolution of Laboratory Parameters Prothrombin time: From a high value at baseline to a value within the reference range at Week 8	5 Participants	—
Evolution of Laboratory Parameters Prothrombin time: From a high value at baseline to a low value at Week 8	1 Participants	—
Evolution of Laboratory Parameters Prothrombin time: From a value within the reference range at baseline to a low value at Week 8	3 Participants	—
Evolution of Laboratory Parameters Prothrombin time: From a low value at baseline to a value within the reference range at Week 8	1 Participants	—
Evolution of Laboratory Parameters D-dimer: From a high value at baseline to a value within the reference range at Week 8	8 Participants	—
Evolution of Laboratory Parameters Fibrinogen: From a high value at baseline to a value within the reference range at Week 8	1 Participants	—
Evolution of Laboratory Parameters Fibrinogen: From a value within the reference range at baseline to a high value at Week 8	1 Participants	—
Evolution of Laboratory Parameters Fibrinogen: From a low value at baseline to a value within the reference range at Week 8	5 Participants	—
Evolution of Laboratory Parameters Fibrinogen: From a low value at baseline to a high value at Week 8	1 Participants	—

PRIMARY outcome

Timeframe: Up to Week 8

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Number of Participants Withdrawn Due to Safety Reasons	0 Participants	—

PRIMARY outcome

Timeframe: Week 8

Remission from MAS was evaluated according to the following criteria:

Resolution of clinical signs and symptoms according to the investigator (MAS clinical signs and symptoms activity score ≤ 1)

and

Normalization of laboratory parameters relevant to MAS, as follows:

• WBC count and platelet count above the LLN.
• LDH below 1.5 × the ULN.
• ALT and AST both below 1.5 × the ULN.
• Fibrinogen higher than 100 mg/dL.
• Ferritin levels decreased by at least 80 % from values at screening or baseline (whichever was higher) or below 2000 ng/mL, whichever was lower.

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Number of Participants Achieving MAS Remission at Week 8 After Initiation of Emapalumab Treatment	11 Participants	—

PRIMARY outcome

Timeframe: Up to Week 8

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Time to First MAS Remission	25 days Interval 19.0 to 56.0	—

PRIMARY outcome

Timeframe: Up to Week 8

Permanently tapering by at least 50% of the equivalent dose of prednisone. This was defined as achieving reduction of 50% of the baseline dose [SD0] and maintaining the reduction until the end of study).

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Number of Participants for Whom Glucocorticoids Could be Permanently Tapered at Any Time During the Study	12 Participants	—

PRIMARY outcome

Timeframe: Up to Week 8

Time to achievement of permanent glucocorticoid tapering by at least 50% of the equivalent dose of prednisone administered at emapalumab treatment start.

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Time to Achievement of Permanent Glucocorticoids Tapering	14.5 days Interval 4.0 to 28.0	—

PRIMARY outcome

Timeframe: Up to Week 8

Number of participants alive at the end of the study

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Survival Time	14 Participants	—

PRIMARY outcome

Timeframe: Up to Week 8

Number of participants withdrawn from the study due to lack of efficacy

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Number of Participants Withdrawn From the Study Due to Lack of Efficacy	0 Participants	—

PRIMARY outcome

Timeframe: Data from the following time points are presented: SD0 (pre- and post-dose), Week 4 Visit 2 (pre- and post-dose), and 4-week follow-up visit/EoS.

Population: Emapalumab concentrations were below the detection limit for all patients at SD0, except for Patient 501-001, in whom the emapalumab concentration (pre-dose) was 62108.4 μg/L. It should be noted that on SD1, the measurement results of emapalumab were below the detection limit for Patient 501-001, so it could not be ruled out that the samples from SD0 and SD1 for this patient were interchanged.

On all infusion days, PK samples were collected before the infusion start and between 15 and 30 minutes after infusion completion. In addition, following the first emapalumab infusion, PK samples were collected approximately 24 and 48 hours post-infusion, and following the second emapalumab infusion, PK samples were collected approximately 48 hours post-infusion. Upon treatment completion, PK samples were collected on all non-infusion visits until the 4-week follow-up visit/EOS.

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Levels of Emapalumab Concentration Study Day 0 (pre-dose)	5455.444 μg/L Standard Deviation 19985.0660	—
Levels of Emapalumab Concentration Study Day 0 (post-dose)	104740.254 μg/L Standard Deviation 20994.1054	—
Levels of Emapalumab Concentration Week 4 Visit 2 (pre-dose)	115472.076 μg/L Standard Deviation 58676.7953	—
Levels of Emapalumab Concentration Week 4 Visit 2 (post-dose)	212900.657 μg/L Standard Deviation 87466.3031	—
Levels of Emapalumab Concentration 4-week follow-up Visit/EoS	46447.462 μg/L Standard Deviation 33657.0174	—

PRIMARY outcome

Timeframe: Up to Week 8

Levels of total INF-gamma, CXCL9 and CXCL10

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS n=14 Participants Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Pharmacodynamic Parameters CXCL9	21986.010 ng/L Standard Deviation 29405.8787	255.654 ng/L Standard Deviation 596.4149
Pharmacodynamic Parameters CXCL10	7935.418 ng/L Standard Deviation 9115.2857	639.102 ng/L Standard Deviation 1058.9589
Pharmacodynamic Parameters Total IFN-γ	425.528 ng/L Standard Deviation 1191.8391	2132.656 ng/L Standard Deviation 2967.4920

PRIMARY outcome

Timeframe: Up to Week 8

The presence of circulating antibodies against emapalumab was inferred by positive results for anti-drug antibodies (ADAs).

Outcome measures

Measure	All Treated Population n=14 Participants The all treated population included all patients who received any part of an infusion of emapalumab.	All Treated Population: 4-week Follow-up Visit/EoS Levels measured at the 4-week follow-up visit/EoS in participants in the all treated population which included all patients who received any part of an infusion of emapalumab.
Number of Participants Who Demonstrated a Presence of Circulating Antibodies Against Emapalumab to Determine Immunogenicity	0 Participants	—

Adverse Events

All Treated Population

Serious events: 6 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Measure	All Treated Population n=14 participants at risk The all treated population included all patients who received any part of an infusion of emapalumab.
Cardiac disorders Cardiopulmonary failure	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Intracardiac thrombus	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Juvenile myoclonic epilepsy	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Pericarditis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Gastrointestinal disorders Pneumatosis intestinalis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Rash	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Musculoskeletal and connective tissue disorders Still's disease	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Blood and lymphatic system disorders Thrombocytopenia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Infections and infestations Cytomegalovirus infection reactivation	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.

Other adverse events

Measure	All Treated Population n=14 participants at risk The all treated population included all patients who received any part of an infusion of emapalumab.
Skin and subcutaneous tissue disorders Rash	28.6% 4/14 • Number of events 4 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Acanthosis nigricans	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Dermatitis diaper	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Dry skin	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Rash erythematous	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Rash maculo-papular	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Rash pruritic	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Skin and subcutaneous tissue disorders Skin ulcer	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Gastrointestinal disorders Diarrhoea	21.4% 3/14 • Number of events 4 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Gastrointestinal disorders Abdominal pain	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Gastrointestinal disorders Abdominal pain upper	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Gastrointestinal disorders Nausea	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Gastrointestinal disorders Pneumatosis intestinalis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Infections and infestations Cytomegalovirus infection reactivation	21.4% 3/14 • Number of events 3 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Infections and infestations Cytomegalovirus infection	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Infections and infestations Nasopharyngitis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Infections and infestations Viral infection	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Headache	14.3% 2/14 • Number of events 4 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Axonal neuropathy	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Juvenile myoclonic epilepsy	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Lethargy	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Peripheral sensorimotor neuropathy	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Nervous system disorders Seizure	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Cardiopulmonary failure	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Intracardiac thrombus	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Pericarditis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Sinus bradycardia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Cardiac disorders Tachycardia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
General disorders Injection site reaction	14.3% 2/14 • Number of events 2 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
General disorders Catheter site thrombosis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
General disorders Chest pain	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
General disorders Condition aggravated	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
General disorders Pyrexia	7.1% 1/14 • Number of events 4 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
General disorders Secretion discharge	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Respiratory, thoracic and mediastinal disorders Cough	7.1% 1/14 • Number of events 2 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Respiratory, thoracic and mediastinal disorders Epistaxis	7.1% 1/14 • Number of events 3 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Respiratory, thoracic and mediastinal disorders Pharyngeal erythema	7.1% 1/14 • Number of events 2 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Respiratory, thoracic and mediastinal disorders Pleural effusion	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Respiratory, thoracic and mediastinal disorders Tachypnoea	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Vascular disorders Hypertension	21.4% 3/14 • Number of events 3 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Vascular disorders Thrombophlebitis superficial	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Injury, poisoning and procedural complications Allergic transfusion reaction	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Injury, poisoning and procedural complications Contusion	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Injury, poisoning and procedural complications Infusion related reaction	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Metabolism and nutrition disorders Hyperglycaemia	21.4% 3/14 • Number of events 3 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Metabolism and nutrition disorders Hyperkalaemia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Blood and lymphatic system disorders Leukopenia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Blood and lymphatic system disorders Neutropenia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Blood and lymphatic system disorders Thrombocytopenia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Eye disorders Eye oedema	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Eye disorders Eye pruritus	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Hepatobiliary disorders Cholelithiasis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Hepatobiliary disorders Hepatic steatosis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Investigations Adenovirus test positive	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Investigations BK polyomavirus test positive	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Investigations Body temperature increased	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Investigations Cytomegalovirus test positive	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Investigations Respirovirus test positive	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Musculoskeletal and connective tissue disorders Still's disease	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Musculoskeletal and connective tissue disorders Tenosynovitis	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Psychiatric disorders Anxiety	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Psychiatric disorders Confusional state	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Ear and labyrinth disorders Tympanic membrane hyperaemia	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.
Immune system disorders Drug hypersensitivity	7.1% 1/14 • Number of events 1 • Adverse events were recorded in patients from Study Day 0 (the day of the first emapalumab administration), during emapalumab treatment, and up to and including the 4-week follow-up visit/end of study (a minimum duration of 56 days). All adverse events were followed up for outcome at end of study; all serious adverse events were followed up until resolution. While all adverse events reported by the participants or their relatives or observed by the Investigator or their staff during the clinical study from the signature of the informed consent form up to and including the end-of-study visit were recorded, only treatment-emergent adverse events (those that occurred after the start of the first emapalumab administration) are reported here.

Additional Information

Veronica Asnaghi, MD

Sobi AG

Phone: +41 61 201 13 20

Email: veronica.asnaghi@sobi.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place