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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-001577-24 | EudraCT Number |
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The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic juvenile idiopathic arthritis and adult onset Still's disease) or with sHLH/MAS in systemic lupus erythematous, resenting an inadequate response to high dose glucocorticoid treatment.
Study NI-0501-14 is a two-cohort trial that enrolls subjects who are diagnosed with sHLH/MAS (MAS being a form of secondary HLH) and who are presenting an inadequate response to high doses of GCs. These subjects will be enrolled in 2 cohorts as per their background disease. The cohorts are defined as follows:
The study has the objectives to investigate the efficacy, safety and tolerability, for 8 weeks, and PK and PD, QoL and immunogenicity in these 2 cohorts for up to 1 year after last dose of of emapalumab.
Macrophage Activation Syndrome (MAS) Secondary Hemophagocytic Lymphohistiocytosis (sHLH) systemic Juvenile Idiopathic Arthritis (sJIA) Adult-onset Still's Disease (AOSD) Systemic Lupus Erythematosus (SLE)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 (sJIA and AOSD) and Cohort 2 (SLE) | Experimental | MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) or SLE |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emapalumab | Drug | Emapalumab iv infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | Resolution of clinical signs and symptoms present at baseline: The macrophage activation syndrome (MAS) clinical activity will be measured on a visual analog scale (VAS) 10 cm. Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm. Clinical signs will be considered as resolved if VAS is below or equal to 1/10. And Normalization of laboratory parameters relevant to MAS, as follows: WBC above LLN, platelet count above LLN, LDH below 1.5 ULN, ALT below 1.5 ULN, AST below 1.5 ULN, fibrinogen higher than 100 mg/dL, ferritin levels decreased by at least 80 % from values at screening or baseline (whichever is higher) or below 2000 ng/ml, whichever is lower | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients With Glucocorticoids (GC)s Tapering to a Dose Below 50% of Prednisolone (PDN) Equivalent at the Time of Emapalumab Start or to the Same (or Lower) Dose Being Administered Before the Occurrence of MAS Whichever Occurs First | GC tapering as per investigator discretion | At any time within the first 8 weeks from start of emapalumab treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events (AEs) (Serious and Non-serious). | number of patients that experienced at least one AE (serious and non-serious). | At any time in the study, up to 1 year |
| Study Interruption Due to Safety Reasons |
Inclusion criteria Run-in phase in all cohorts
Interventional phase in all cohorts
Informed consent provided by the subject or by the subject's legally authorized representative(s) with the assent of subjects who are legally capable of providing it, as as required by local law.
Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of active MAS.
Subjects who have shown an inadequate response to high dose intravenous (i.v.) GCs administered for at least 3 days according to local standard clinical practice, including but not limited to pulses of 30 mg/kg PDN on 3 consecutive days. High i.v. GCs dose is recommended not to be lower than 2 mg/kg/ day PDN equivalent (or at least 60 mg/day in pediatric subjects of 30 kg or more, and at least 1g/day in adult MAS subjects). In case of rapid worsening of the subject's condition and/or laboratory parameters, as per Investigator judgment, inclusion may occur within less than 3 days from starting high dose GCs.
Diagnosis of active MAS confirmed by the treating rheumatologist, having ascertained the followings:
a. Febrile subjects presenting with ferritin > 684 ng/mL. b. and any 2 of: i. Platelet count ≤ 181 x109/L ii. AST-level > 48 U/L iii. Triglycerides > 156 mg/dL iv. Fibrinogen level ≤ 360 mg/dL
Female subjects of child-bearing potential willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug.
Specific inclusion criteria to Cohort 1 and Cohort 2
Cohort 1:
Cohort 2:
Exclusion criteria
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| Name | Affiliation | Role |
|---|---|---|
| Brian Jamieson, MD | Swedish Orphan Biovitrum | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UAB Hospital | Birmingham | Alabama | 35233 | United States | ||
| UCLA Health |
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 (sJIA and AOSD) | MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) Emapalumab: Emapalumab iv infusion |
| FG001 | Cohort 2 (SLE) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 19, 2023 | Dec 9, 2025 |
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2 cohorts
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Open label
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| Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | GC tapering as per investigator discretion | At any time in the study, up to 1 year |
| Time to Achieve GCs Tapering | Median time to achieve GCs tapering to a dose < 50 % of PDN equivalent at the time of emapalumab start or to the same (or lower) dose being administered before the occurrence of MAS (in patients already treated for the underlying condition), or to ≤1mg/kg/Day of PDN Equivalent, whichever occurs first at any time during the study GC tapering as per investigator discretion | At any time in the study, up to 1 year |
| Time to First Complete Response (CR) | Time to CR until EOS Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity was measured on a visual analog scale (VAS) 10 cm. Definition of CR: Resolution of clinical signs and symptoms present at baseline: MAS clinical activity was measured on a 10-cm VAS. Clinical signs were considered resolved if VAS was below or equal to 1/10. and Normalization of laboratory parameters relevant to MAS as follows:
| At any time in the study, up to 1 year |
| Proportion of Subjects With Overall Response as Defined by CR or Partial Response (PR) | Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity was measured on a visual analog scale (VAS) 10 cm. CR: Resolution of clinical signs and symptoms present at baseline: MAS clinical activity was measured on a 10-cm VAS. Clinical signs were considered resolved if VAS was below or equal to 1/10. and Normalization of laboratory parameters relevant to MAS as follows:
PR: Resolution or improvement in clinical signs and symptoms measured by the MAS clinical activity on the VAS. The patient was classified as PR if he or she presented a VAS <4/10. and Normalization of at least 3 of the abnormal baseline laboratory parameters relevant to MAS, as defined above. | At week 8 |
| Time to First Overall Response as Defined by CR or PR | Time to first overall response CR: Resolution of clinical signs and symptoms present at baseline: MAS clinical activity was measured on a 10-cm VAS. Clinical signs were considered resolved if VAS was below or equal to 1/10. and Normalization of laboratory parameters relevant to MAS as follows:
PR: Resolution or improvement in clinical signs and symptoms measured by the MAS clinical activity on the VAS. The patient was classified as PR if he or she presented a VAS <4/10. and Normalization of at least 3 of the abnormal baseline laboratory parameters relevant to MAS, as defined above. | At any time in the study, up to 1 year |
| MAS Recurrence at Anytime After Achievement of CR | Number of patients with MAS recurrence at anytime after achievement of CR | At any time after CR, up to 1 year |
| Withdrawal From the Study Due to Lack of Response | Number of patients withdrawn from the study due to lack of response as per Investigator decision | At any time in the study, up to 1 year |
| Survival | Number of patients alive at the end of study | At end of study, 1 year |
Number of participants withdrawn from study treatment due to adverse event
| At any time in the study, up to 1 year |
| Laboratory Parameters | Number of patients with clinically meaningful changes in hematology and chemistry laboratory parameters from baseline | At any time in the study, up to 1 year |
| Patient Reported Outcomes : PedsQL™; | Pediatric Quality of Life Inventory (PedsQL™; Generic Core Scales and Infant Scales, Acute versions) The PedsQL is scored on a 5-point Likert scale from: 0 (Never) to 4 (Almost always) Then, for ease of interpretability, items are reversed scored and linearly transformed to a 0-100 scale, so that higher scores indicate better HRQOL (Health-Related Quality of Life). To reverse score, transform the 0-4 scale items to 0-100 as follows: 0=100, 1=75, 2=50, 3=25, 4=0 To create the Total Scale Score, the mean is computed as the sum of all the items over the number of items answered on all the Scales (this also accounts for missing data). If more than 50% of the items in the scale are missing, the Scale Scores should not be computed. If 50% or more items are completed: Impute the mean of the completed items in a scale | Baseline, week 8, 6 months, and EOS (1 year) |
| Patient Reported Outcomes: Clinician Global Impression of Severity | Health-related quality of life: Global Assessment: Clinician Global Impression of Severity Number of patients having an overall severity of the health status over the past week assessed by the clinician as None, Mild, Moderate, or Severe, at the specified timepoints. | Baseline, Week 8, month 6 and EOS (1 year) |
| Patient Reported Outcomes: Patient/Parent Global Impression of Severity | Health-related quality of life: Global Assessment: Patient/Parent Global Impression of Severity Number of patients having an overall severity of the health status over the past week assessed by the Patient/Parent as None, Mild, Moderate, or Severe, at the specified timepoints. | Screening, Week 8, month 6 and EOS (1 year) |
| PK Endpoints | Serum concentrations of emapalumab vs. time | Baseline (Day 1), week 8, Day 60, Day 100, 6 Months, EOS (1 year) |
| PD Endpoints Per Cohort: Chemokines | Levels of the main IFN-γ-induced chemokine (CXCL9). | Baseline, week 8, 6 months, EOS (1 year) |
| PD Endpoints Per Cohort: sCD25) | Levels of MAS markers; sCD25 (Soluble CD25 (i.e., soluble IL-2 receptor)) | Baseline, week 8, 6 months, EOS (1 year) |
| Immunogenicity Endpoints | Occurrence of ADAs to emapalumab until EOS (1 year after last dose of emapalumab) Baseline and overall incidence | At any time in the study, up to 1 year |
| Los Angeles |
| California |
| 90095 |
| United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | United States |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | 55455 | United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Nationwide Children's Hospital, Abigail Wexner Research Institute | Columbus | Ohio | 43205 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | United States |
| Universitair Ziekenhuis Leuven | Leuven | Belgium |
| Alberta Children's Hospital | Calgary | Canada |
| University of Calgary | Calgary | Canada |
| Centre Hospitalier de l'Université de Montréal | Montreal | Canada |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Canada |
| Hospital for sick children | Toronto | Canada |
| Beijing Children's Hospital | Beijing | China |
| Beijing Friendship Hospital | Beijing | China |
| Children's Hospital of Fudan University | Shanghai | China |
| Fakultní nemocnice Olomouc | Olomouc | Czechia |
| Vseobecna Fakultni Nemocnice v Praze | Prague | Czechia |
| Hôpital Claude Huriez | Lille | France |
| Hôpital De La Conception | Marseille | France |
| Hôpital Necker-Enfants Malades | Paris | France |
| Hôpital Universitaire Pitié Salpêtrière | Paris | France |
| Charité Universitätsmedizin | Berlin | Germany |
| Universitätsklinikum Heidelberg | Heidelberg | Germany |
| IRCCS G. Gaslini | Genova | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Ospedale Pediatrico Bambino Gesù | Roma | Italy |
| IRCCS - Materno-Infantile Burlo Garofolo | Trieste | Italy |
| St. Marianna University School of Medicine Hospital | Kawasaki | Japan |
| Osaka Medical and Pharmaceutical University Hospital | Takatsuki | Japan |
| Tokyo Medical and Dental University Hospital | Tokyo | Japan |
| Yokohama City University Hospital | Yokohama | Japan |
| UMC Utrecht | Utrecht | Netherlands |
| Szpital Specjalistyczny im. J. Dietla w Krakowie | Krakow | Poland |
| Wojewódzki Specjalistyczny Szpital Dziecięcy im. św. Ludwika w Krakowie | Krakow | Poland |
| Ortopedyczno - Rehabilitacyjny Szpital Kliniczny im. Wiktora Degi Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu | Poznan | Poland |
| Hospital Sant Joan de Déu | Barcelona | Spain |
| Hospital Universitario La Paz | La Paz | Spain |
| Hospital Universitario | La Paz | Spain |
| Hospital Universitari i Politècnic La Fe | Valencia | Spain |
| Great Ormond Street Hospital | London | United Kingdom |
| Imperial College Healthcare NHS Trust | London | United Kingdom |
MAS in the context of systemic lupus erythematosus (SLE)
Emapalumab: Emapalumab iv infusion
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 (sJIA and AOSD) | MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) Emapalumab: Emapalumab iv infusion |
| BG001 | Cohort 2 (SLE) | MAS in the context of systemic lupus erythematosus (SLE) Emapalumab: Emapalumab iv infusion |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Body weight | Mean | Standard Deviation | kg |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | Resolution of clinical signs and symptoms present at baseline: The macrophage activation syndrome (MAS) clinical activity will be measured on a visual analog scale (VAS) 10 cm. Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm. Clinical signs will be considered as resolved if VAS is below or equal to 1/10. And Normalization of laboratory parameters relevant to MAS, as follows: WBC above LLN, platelet count above LLN, LDH below 1.5 ULN, ALT below 1.5 ULN, AST below 1.5 ULN, fibrinogen higher than 100 mg/dL, ferritin levels decreased by at least 80 % from values at screening or baseline (whichever is higher) or below 2000 ng/ml, whichever is lower | All treated | Posted | Count of Participants | Participants | 8 weeks |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Number of Patients With Glucocorticoids (GC)s Tapering to a Dose Below 50% of Prednisolone (PDN) Equivalent at the Time of Emapalumab Start or to the Same (or Lower) Dose Being Administered Before the Occurrence of MAS Whichever Occurs First | GC tapering as per investigator discretion | Posted | Count of Participants | Participants | At any time within the first 8 weeks from start of emapalumab treatment |
|
| |||||||||||||||||||||||||||||||
| Secondary | Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | GC tapering as per investigator discretion | Posted | Count of Participants | Participants | At any time in the study, up to 1 year |
|
| |||||||||||||||||||||||||||||||
| Secondary | Time to Achieve GCs Tapering | Median time to achieve GCs tapering to a dose < 50 % of PDN equivalent at the time of emapalumab start or to the same (or lower) dose being administered before the occurrence of MAS (in patients already treated for the underlying condition), or to ≤1mg/kg/Day of PDN Equivalent, whichever occurs first at any time during the study GC tapering as per investigator discretion | Posted | Median | 95% Confidence Interval | weeks | At any time in the study, up to 1 year |
|
| ||||||||||||||||||||||||||||||
| Secondary | Time to First Complete Response (CR) | Time to CR until EOS Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity was measured on a visual analog scale (VAS) 10 cm. Definition of CR: Resolution of clinical signs and symptoms present at baseline: MAS clinical activity was measured on a 10-cm VAS. Clinical signs were considered resolved if VAS was below or equal to 1/10. and Normalization of laboratory parameters relevant to MAS as follows:
| Posted | Median | 95% Confidence Interval | weeks | At any time in the study, up to 1 year |
|
| ||||||||||||||||||||||||||||||
| Secondary | Proportion of Subjects With Overall Response as Defined by CR or Partial Response (PR) | Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity was measured on a visual analog scale (VAS) 10 cm. CR: Resolution of clinical signs and symptoms present at baseline: MAS clinical activity was measured on a 10-cm VAS. Clinical signs were considered resolved if VAS was below or equal to 1/10. and Normalization of laboratory parameters relevant to MAS as follows:
PR: Resolution or improvement in clinical signs and symptoms measured by the MAS clinical activity on the VAS. The patient was classified as PR if he or she presented a VAS <4/10. and Normalization of at least 3 of the abnormal baseline laboratory parameters relevant to MAS, as defined above. | Posted | Count of Participants | Participants | At week 8 |
| ||||||||||||||||||||||||||||||||
| Secondary | Time to First Overall Response as Defined by CR or PR | Time to first overall response CR: Resolution of clinical signs and symptoms present at baseline: MAS clinical activity was measured on a 10-cm VAS. Clinical signs were considered resolved if VAS was below or equal to 1/10. and Normalization of laboratory parameters relevant to MAS as follows:
PR: Resolution or improvement in clinical signs and symptoms measured by the MAS clinical activity on the VAS. The patient was classified as PR if he or she presented a VAS <4/10. and Normalization of at least 3 of the abnormal baseline laboratory parameters relevant to MAS, as defined above. | Posted | Median | 95% Confidence Interval | weeks | At any time in the study, up to 1 year |
| |||||||||||||||||||||||||||||||
| Secondary | MAS Recurrence at Anytime After Achievement of CR | Number of patients with MAS recurrence at anytime after achievement of CR | Posted | Count of Participants | Participants | At any time after CR, up to 1 year |
|
| |||||||||||||||||||||||||||||||
| Secondary | Withdrawal From the Study Due to Lack of Response | Number of patients withdrawn from the study due to lack of response as per Investigator decision | Posted | Count of Participants | Participants | At any time in the study, up to 1 year |
|
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| Secondary | Survival | Number of patients alive at the end of study | Posted | Count of Participants | Participants | At end of study, 1 year |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Adverse Events (AEs) (Serious and Non-serious). | number of patients that experienced at least one AE (serious and non-serious). | Posted | Count of Participants | Participants | At any time in the study, up to 1 year |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Study Interruption Due to Safety Reasons | Number of participants withdrawn from study treatment due to adverse event | Posted | Count of Participants | Participants | At any time in the study, up to 1 year |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Laboratory Parameters | Number of patients with clinically meaningful changes in hematology and chemistry laboratory parameters from baseline | Posted | Count of Participants | Participants | At any time in the study, up to 1 year |
|
| |||||||||||||||||||||||||||||||
| Other Pre-specified | Patient Reported Outcomes : PedsQL™; | Pediatric Quality of Life Inventory (PedsQL™; Generic Core Scales and Infant Scales, Acute versions) The PedsQL is scored on a 5-point Likert scale from: 0 (Never) to 4 (Almost always) Then, for ease of interpretability, items are reversed scored and linearly transformed to a 0-100 scale, so that higher scores indicate better HRQOL (Health-Related Quality of Life). To reverse score, transform the 0-4 scale items to 0-100 as follows: 0=100, 1=75, 2=50, 3=25, 4=0 To create the Total Scale Score, the mean is computed as the sum of all the items over the number of items answered on all the Scales (this also accounts for missing data). If more than 50% of the items in the scale are missing, the Scale Scores should not be computed. If 50% or more items are completed: Impute the mean of the completed items in a scale | PedsQL parent report (age from 2 years) over time for the Interventional Phase (All Treated Analysis Set) | Posted | Mean | Standard Deviation | score on a scale | Baseline, week 8, 6 months, and EOS (1 year) |
| ||||||||||||||||||||||||||||||
| Other Pre-specified | Patient Reported Outcomes: Clinician Global Impression of Severity | Health-related quality of life: Global Assessment: Clinician Global Impression of Severity Number of patients having an overall severity of the health status over the past week assessed by the clinician as None, Mild, Moderate, or Severe, at the specified timepoints. | Result not reported for each patient at each visit | Posted | Count of Participants | Participants | Baseline, Week 8, month 6 and EOS (1 year) |
|
| ||||||||||||||||||||||||||||||
| Other Pre-specified | Patient Reported Outcomes: Patient/Parent Global Impression of Severity | Health-related quality of life: Global Assessment: Patient/Parent Global Impression of Severity Number of patients having an overall severity of the health status over the past week assessed by the Patient/Parent as None, Mild, Moderate, or Severe, at the specified timepoints. | Result not reported for each patient at each visit | Posted | Count of Participants | Participants | Screening, Week 8, month 6 and EOS (1 year) |
|
| ||||||||||||||||||||||||||||||
| Other Pre-specified | PK Endpoints | Serum concentrations of emapalumab vs. time | Result not reported for each patient at each visit | Posted | Mean | Standard Deviation | ng/mL | Baseline (Day 1), week 8, Day 60, Day 100, 6 Months, EOS (1 year) |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | PD Endpoints Per Cohort: Chemokines | Levels of the main IFN-γ-induced chemokine (CXCL9). | Results not reported for each patient at each visit | Posted | Mean | Standard Deviation | ng/L | Baseline, week 8, 6 months, EOS (1 year) |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | PD Endpoints Per Cohort: sCD25) | Levels of MAS markers; sCD25 (Soluble CD25 (i.e., soluble IL-2 receptor)) | Results not reported for each patient at each visit | Posted | Mean | Standard Deviation | ng/L | Baseline, week 8, 6 months, EOS (1 year) |
|
| |||||||||||||||||||||||||||||
| Other Pre-specified | Immunogenicity Endpoints | Occurrence of ADAs to emapalumab until EOS (1 year after last dose of emapalumab) Baseline and overall incidence | All patients with at least one Immunogenicity sample | Posted | Count of Participants | Participants | At any time in the study, up to 1 year |
|
|
From signature of informed consent (i.e. screening) until EOS (1 year)
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 (sJIA and AOSD) | MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) Emapalumab: Emapalumab iv infusion | 2 | 25 | 18 | 25 | 24 | 25 |
| EG001 | Cohort 2 (SLE) | MAS in the context of systemic lupus erythematosus (SLE) Emapalumab: Emapalumab iv infusion | 0 | 8 | 4 | 8 | 8 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Still's disease | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Herpes Zoster | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Lupus nephritis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Pulmonary arterial hypertension | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Prothrombin level decreased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Multiple organ dysfunction syndrome | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Shock | Vascular disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Mycobacterium avium complex infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Fungal infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Latent tuberculosis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Catheter site haemorrhage | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Haemoperitoneum | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Intestinal haemorrhage | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Intestinal perforation | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Jejunal perforation | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Subileus | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Granulocytopenia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Graft versus host disease | Immune system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Oxygen saturation decreased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Pericarditis | Cardiac disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Spinal fracture | Injury, poisoning and procedural complications | MedDRA version 28.0 | Systematic Assessment |
| |
| Facial paralysis | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cytomegalovirus infection reactivation | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Viral pharyngitis | Infections and infestations | MedDRA version 28.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Thrombocytopenia 5 | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Still's disease | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Systemic lupus erythematosus | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Osteopenia | Musculoskeletal and connective tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Cytomegalovirus test positive | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Bone density decreased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Hepatic enzyme abnormal | Investigations | MedDRA version 28.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Lupus nephritis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Secondary immunodeficiency | Immune system disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Ocular hypertension | Eye disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Hyperparathyroidism secondary | Endocrine disorders | MedDRA version 28.0 | Systematic Assessment |
| |
| Steroid diabetes | Metabolism and nutrition disorders | MedDRA version 28.0 | Systematic Assessment |
|
Enrollment in Cohort 2 was discontinued by the Sponsor after 8 (of 16 planned) patients were enrolled in June 2024 due to enrollment challenges in this patient population.
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Info | Swedish Orphan Biovitrum (Sobi) | +1 774 548 5650 | medical.info@sobi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 10, 2025 | Dec 9, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D055501 | Macrophage Activation Syndrome |
| D001171 | Arthritis, Juvenile |
| D008180 | Lupus Erythematosus, Systemic |
| ID | Term |
|---|---|
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D001168 | Arthritis |
| D007592 | Joint Diseases |
| D009140 | Musculoskeletal Diseases |
| D012216 | Rheumatic Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000644327 | Emapalumab |
| D014944 | World Health Organization |
| ID | Term |
|---|---|
| D014480 | United Nations |
| D007390 | International Agencies |
| D009938 | Organizations |
| D004472 | Health Care Economics and Organizations |
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Czechia |
|
| Japan |
|
| United Kingdom |
|
| Spain |
|
| Canada |
|
| Netherlands |
|
| Belgium |
|
| China |
|
| Italy |
|
| France |
|
| Germany |
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Units | Counts |
|---|
| Participants |
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|
|
|
| mild |
|
| moderate |
|
| severe |
|
| mild |
|
| moderate |
|
| severe |
|
| mild |
|
| moderate |
|
| severe |
|
| mild |
|
| moderate |
|
| severe |
|
| mild |
|
| moderate |
|
| severe |
|
| mild |
|
| moderate |
|
| severe |
|