Trial · NCT03311854
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)
NCT03311854, Phase 2, Completed, Swedish Orphan Biovitrum; results first posted 2022-01-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. 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). Trials, record NCT03311854. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03311854
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
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 number
- NCT03311854
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 14 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic, Arthritis, Juvenile, Adult Onset Still Disease
- Interventions
- Emapalumab
- First posted
- 2017-10-17
- Last updated
- 2022-05-17
- Results first posted
- 2022-01-26
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | Up to Week 8 | |
| Primary | Evolution of Laboratory Parameters | 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 |
| Primary | Number of Participants Withdrawn Due to Safety Reasons | Up to Week 8 | |
| Primary | Number of Participants Achieving MAS Remission at Week 8 After Initiation of Emapalumab Treatment | 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. |
| Primary | Time to First MAS Remission | Up to Week 8 | |
| Primary | Number of Participants for Whom Glucocorticoids Could be Permanently Tapered at Any Time During the Study | 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). |
| Primary | Time to Achievement of Permanent Glucocorticoids Tapering | 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. |
| Primary | Survival Time | Up to Week 8 | Number of participants alive at the end of the study |
| Primary | Number of Participants Withdrawn From the Study Due to Lack of Efficacy | Up to Week 8 | Number of participants withdrawn from the study due to lack of efficacy |
| Primary | Levels of Emapalumab Concentration | 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. | 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. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | Up to Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Evolution of Laboratory Parameters | Up to Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number of Participants Withdrawn Due to Safety Reasons | Up to Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number of Participants Achieving MAS Remission at Week 8 After Initiation of Emapalumab Treatment | Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Time to First MAS Remission | Up to Week 8 | days | MEDIAN | |
| PRIMARY | Number of Participants for Whom Glucocorticoids Could be Permanently Tapered at Any Time During the Study | Up to Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Time to Achievement of Permanent Glucocorticoids Tapering | Up to Week 8 | days | MEDIAN | |
| PRIMARY | Survival Time | Up to Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number of Participants Withdrawn From the Study Due to Lack of Efficacy | Up to Week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Levels of Emapalumab Concentration | 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. | 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. | μg/L | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group57
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a TEAE | 13 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a serious TEAE | 6 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a severe TEAE | 2 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a moderate TEAE | 10 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a mild TEAE | 13 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a TEAE related to emapalumab | 4 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a TEAE unrelated to emapalumab | 13 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | Number of participants with a TEAE with an outcome of recovered/resolved | 13 | Participants | |
| Incidence, Severity, Causality, and Outcomes of AEs (Serious and Nonserious) | All Treated Population | 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) | All Treated Population | Number of participants with a TEAE with an outcome of recovering/resolving | 1 | Participants |
1 to 10 of 57Source ClinicalTrials.gov
See all 57 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | All Treated Population | 14 | |
| Overall Study | COMPLETED | All Treated Population | 14 | |
| Overall Study | NOT COMPLETED | All Treated Population | 0 |
Source ClinicalTrials.gov
Adverse events74
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Rash | Skin and subcutaneous tissue disorders | All Treated Population | 4 | 14 | 4 |
| other | Acanthosis nigricans | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Dermatitis diaper | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Dry skin | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Rash erythematous | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Rash maculo-papular | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Rash pruritic | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Skin ulcer | Skin and subcutaneous tissue disorders | All Treated Population | 1 | 14 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | All Treated Population | 3 | 14 | 4 |
| other | Abdominal pain | Gastrointestinal disorders | All Treated Population | 1 | 14 | 1 |
1 to 10 of 74Source ClinicalTrials.gov
See all 74 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | All Treated Population | 0 | 14 |
| Total, other adverse events | All Treated Population | 13 | 14 |
| Total, serious adverse events | All Treated Population | 6 | 14 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03311854
GET https://conductscience.org/api/trials/v1/NCT03311854/results?page=2