Macrophage Activation SyndromePhase 3EMERALDNCT05001737
Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
A Phase 3 study testing Emapalumab in people with Macrophage Activation Syndrome and Secondary Hemophagocytic Lymphohistiocytosis, run by Swedish Orphan Biovitrum at 44 sites in 13 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 6 months to 80 years
- Conditions the study is for
- Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease
- What is being tested?
- Emapalumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 44 sitesin 13 countries
- Study dates
- 15 Dec 2021to 4 Jun 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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 required by local law.
- Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of MAS.
- MAS defined as per the criteria defined below for each cohort and requiring treatment with GCs.
- 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.
Show all 16Show fewer
- 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:
- 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:
- Confirmed sJIA diagnosis. For subjects presenting with MAS in the context of the onset of sJIA, high presumption of sJIA will suffice for eligibility.
- Confirmed diagnosis of AOSD as per Yamaguchi criteria.
- Cohort 2:
- Confirmed diagnosis of SLE as per SLICC'12 criteria.
Who cannot join
- Primary HLH documented by either the presence of a known causative genetic mutation or abnormal perforin expression and CD107a degranulation assay as described with primary hemophagocytic lymphohistiocytosis or by the presence of family history.
- Confirmed malignancy. Note: subjects with a suspected malignancy should have mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable, to rule out malignancy.
- Treatment with canakinumab, JAK inhibitors, TNF inhibitors and tocilizumab at the time of emapalumab initiation.
- Ongoing treatment with anakinra at a dose above 4 mg/kg at time of emapalumab initiation.
- Subjects treated with etoposide for MAS in the last 1 month.
- Clinically active mycobacteria (typical and atypical), Histoplasma Capsulatum, or Salmonella infections.
Show all 12Show fewer
- Evidence of leishmania infections.
- Evidence of latent tuberculosis.
- History of hypersensitivity or allergy to any component of the study drug.
- Receipt of a Bacillus Calmette-Guerin (BCG) vaccine within 12 weeks prior to screening.
- Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to screening.
- Pregnancy or lactating female subjects.
Trial locations and contacts
44 sites in 13 countries.
44 sites in 13 countries
United States9 sites
Akron Children's Hospital
Akron, Ohio, 44308
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
UAB Hospital
Birmingham, Alabama, 35233
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital, Abigail Wexner Research Institute
Columbus, Ohio, 43205
University of Florida
Gainesville, Florida, 32610
UCLA Health
Los Angeles, California, 90095
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55455
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Canada5 sites
Alberta Children's Hospital
Calgary
University of Calgary
Calgary
Centre Hospitalier de l'Université de Montréal
Montreal
Centre Hospitalier Universitaire Sainte-Justine
Montreal
Hospital for sick children
Toronto
France4 sites
Hôpital Claude Huriez
Lille
Hôpital De La Conception
Marseille
Hôpital Necker-Enfants Malades
Paris
Hôpital Universitaire Pitié Salpêtrière
Paris
Italy4 sites
IRCCS G. Gaslini
Genova
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan
Ospedale Pediatrico Bambino Gesù
Roma
IRCCS - Materno-Infantile Burlo Garofolo
Trieste
Japan4 sites
St. Marianna University School of Medicine Hospital
Kawasaki
Osaka Medical and Pharmaceutical University Hospital
Takatsuki
Tokyo Medical and Dental University Hospital
Tokyo
Yokohama City University Hospital
Yokohama
Spain4 sites
Hospital Sant Joan de Déu
Barcelona
Hospital Universitario La Paz
La Paz
Hospital Universitario
La Paz
Hospital Universitari i Politècnic La Fe
Valencia
China3 sites
Beijing Children's Hospital
Beijing
Beijing Friendship Hospital
Beijing
Children's Hospital of Fudan University
Shanghai
Poland3 sites
Szpital Specjalistyczny im. J. Dietla w Krakowie
Krakow
Wojewódzki Specjalistyczny Szpital Dziecięcy im. św. Ludwika w Krakowie
Krakow
Ortopedyczno - Rehabilitacyjny Szpital Kliniczny im. Wiktora Degi Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
Poznan
Czechia2 sites
Fakultní nemocnice Olomouc
Olomouc
Vseobecna Fakultni Nemocnice v Praze
Prague
Germany2 sites
Charité Universitätsmedizin
Berlin
Universitätsklinikum Heidelberg
Heidelberg
United Kingdom2 sites
Great Ormond Street Hospital
London
Imperial College Healthcare NHS Trust
London
About this study
Official title: A Two-cohort, Open-label, Single Arm, Multicenter Study to Evaluate Efficacy, Safety and Tolerability, PK and PD, of Emapalumab in Children and Adults With MAS in Still's Disease or With MAS in Systemic Lupus Erythematous
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.
Read the detailed description
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:
- Cohort 1: MAS in the context of sJIA and AOSD.
- Cohort 2: MAS in the context of pediatric and adult SLE.
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)
- Conditions
- Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD
- Being tested
- Emapalumab
- Medical subject headings
- Macrophage Activation Syndrome, Arthritis, Juvenile, Lupus Erythematosus, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmapalumabDrug
Also called NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)
Emapalumab iv infusion
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Emapalumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 8 weeks
Secondary outcomes (9)
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
Measured: At any time within the first 8 weeks from start of emapalumab treatment
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
Measured: 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
Measured: 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:
- WBC >LLN.
- Platelet count >LLN.
- LDH <1.5 ULN.
- ALT <1.5 ULN.
- AST <1.5 ULN.
- Fibrinogen >100 mg/dL.
- Ferritin levels decreased by at least 80% from values at Screening or baseline (whichever was higher) or <2000 ng/mL, whichever was lower.
Measured: 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:
- WBC >LLN.
- Platelet count >LLN.
- LDH <1.5 ULN.
- ALT <1.5 ULN.
- AST <1.5 ULN.
- Fibrinogen >100 mg/dL.
- Ferritin levels decreased by at least 80% from values at Screening or baseline (whichever was higher) or <2000 ng/mL, whichever was lower.
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.
Measured: 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:
- WBC >LLN.
- Platelet count >LLN.
- LDH <1.5 ULN.
- ALT <1.5 ULN.
- AST <1.5 ULN.
- Fibrinogen >100 mg/dL.
- Ferritin levels decreased by at least 80% from values at Screening or baseline (whichever was higher) or <2000 ng/mL, whichever was lower.
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.
Measured: 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
Measured: 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
Measured: At any time in the study, up to 1 year
Survival
Number of patients alive at the end of study
Measured: At end of study, 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2021
- Study start15 Dec 2021
- Primary completion30 Jun 2024
- Study completion4 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Macrophage Activation SyndromeStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 2 cohorts
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05001737
- Sponsor's protocol ID
- NI-0501-14
- Other IDs
- 2021-001577-24
- Acronym
- EMERALD
- Status last verified
- Feb 2026
- First submitted
- 16 Jun 2021
- Last updated
- 26 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-cohort, Open-label, Single Arm, Multicenter Study to Evaluate Efficacy, Safety and Tolerability, PK and PD, of Emapalumab in Children and Adults With MAS in Still's Disease or With MAS in Systemic Lupus Erythematous
- ClinicalTrials.gov ID
- NCT05001737
- Acronym / sponsor ID
- EMERALD / NI-0501-14
- Conditions
- Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 15 Dec 2021 (actual)
- Primary completion
- 30 Jun 2024 (actual)
- Study completion
- 4 Jun 2025 (actual)
- First posted
- 12 Aug 2021 (actual)
- Results
- Posted 19 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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:
- Cohort 1: MAS in the context of sJIA and AOSD.
- Cohort 2: MAS in the context of pediatric and adult SLE.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 2 cohorts
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (sJIA and AOSD) and Cohort 2 (SLE) | Experimental | Drug: EmapalumabMAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) or SLE |
Who may qualify
- Ages
- 6 months to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- 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 required by local law.
- Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of MAS.
- MAS defined as per the criteria defined below for each cohort and requiring treatment with GCs.
- 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.
Key exclusion criteria (12 listed)
- Primary HLH documented by either the presence of a known causative genetic mutation or abnormal perforin expression and CD107a degranulation assay as described with primary hemophagocytic lymphohistiocytosis or by the presence of family history.
- Confirmed malignancy. Note: subjects with a suspected malignancy should have mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable, to rule out malignancy.
- Treatment with canakinumab, JAK inhibitors, TNF inhibitors and tocilizumab at the time of emapalumab initiation.
- Ongoing treatment with anakinra at a dose above 4 mg/kg at time of emapalumab initiation.
- Subjects treated with etoposide for MAS in the last 1 month.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EmapalumabAlso: NI-0501, emapalumab-lzsg, ATC code: L04AA39 (WHO)Emapalumab iv infusion | Drug | Cohort 1 (sJIA and AOSD) and Cohort 2 (SLE) |
Primary outcome
Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab
Time frame: 8 weeks
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
9 secondary outcomes
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
Time frame: At any time within the first 8 weeks from start of emapalumab treatment
Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS).
Time frame: At any time in the study, up to 1 year
Time to Achieve GCs Tapering
Time frame: At any time in the study, up to 1 year
Time to First Complete Response (CR)
Time frame: At any time in the study, up to 1 year
Proportion of Subjects With Overall Response as Defined by CR or Partial Response (PR)
Time frame: At week 8
Time to First Overall Response as Defined by CR or PR
Time frame: At any time in the study, up to 1 year
MAS Recurrence at Anytime After Achievement of CR
Time frame: At any time after CR, up to 1 year
Withdrawal From the Study Due to Lack of Response
Time frame: At any time in the study, up to 1 year
Survival
Time frame: At end of study, 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director, MD study director
Swedish Orphan Biovitrum
44 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Emapalumab in people with Macrophage Activation Syndrome and Secondary Hemophagocytic Lymphohistiocytosis, run by Swedish Orphan Biovitrum at 44 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 44 sites in United States, Canada, France, Italy, Japan and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026; AzTrials last refreshed its copy on 8 Nov 2026.