Trial · NCT05001737
Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
NCT05001737, Phase 3, Completed, Swedish Orphan Biovitrum; results first posted 2026-02-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE. Trials, record NCT05001737. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05001737
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
- NCT number
- NCT05001737
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 33 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS
- Interventions
- Emapalumab
- First posted
- 2021-08-12
- Last updated
- 2026-08-26
- Other registries' IDs
- eudract:2021-001577-24
- Results first posted
- 2026-02-19
- Limitations and caveats
- 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.
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | 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 |
| 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 | At any time within the first 8 weeks from start of emapalumab treatment | GC tapering as per investigator discretion |
| Secondary | Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | At any time in the study, up to 1 year | GC tapering as per investigator discretion |
| Secondary | Time to Achieve GCs Tapering | At any time in the study, up to 1 year | 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 |
| Secondary | Time to First Complete Response (CR) | At any time in the study, up to 1 year | 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. |
| Secondary | Proportion of Subjects With Overall Response as Defined by CR or Partial Response (PR) | At week 8 | 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. |
| Secondary | Time to First Overall Response as Defined by CR or PR | At any time in the study, up to 1 year | 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. |
| Secondary | MAS Recurrence at Anytime After Achievement of CR | At any time after CR, up to 1 year | Number of patients with MAS recurrence at anytime after achievement of CR |
| Secondary | Withdrawal From the Study Due to Lack of Response | At any time in the study, up to 1 year | Number of patients withdrawn from the study due to lack of response as per Investigator decision |
| Secondary | Survival | At end of study, 1 year | Number of patients alive at the end of study |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | 8 weeks | All treated | Participants | COUNT_OF_PARTICIPANTS |
| 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 | At any time within the first 8 weeks from start of emapalumab treatment | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | At any time in the study, up to 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Time to Achieve GCs Tapering | At any time in the study, up to 1 year | weeks | MEDIAN | |
| SECONDARY | Time to First Complete Response (CR) | At any time in the study, up to 1 year | weeks | MEDIAN | |
| SECONDARY | Proportion of Subjects With Overall Response as Defined by CR or Partial Response (PR) | At week 8 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Time to First Overall Response as Defined by CR or PR | At any time in the study, up to 1 year | weeks | MEDIAN | |
| SECONDARY | MAS Recurrence at Anytime After Achievement of CR | At any time after CR, up to 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Withdrawal From the Study Due to Lack of Response | At any time in the study, up to 1 year | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Survival | At end of study, 1 year | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group130
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | Cohort 1 (sJIA and AOSD) | 11 | Participants | ||
| Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | Cohort 2 (SLE) | 4 | Participants | ||
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 | Cohort 1 (sJIA and AOSD) | 15 | Participants | ||
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 | Cohort 2 (SLE) | 7 | Participants | ||
| Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | Cohort 1 (sJIA and AOSD) | 19 | Participants | ||
| Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | Cohort 2 (SLE) | 6 | Participants | ||
| Time to Achieve GCs Tapering | Cohort 1 (sJIA and AOSD) | 5 | 95% Confidence Interval 2.0 to 10.0 | weeks | |
| Time to Achieve GCs Tapering | Cohort 2 (SLE) | 1 | 95% Confidence Interval 1.0 to 5.0 | weeks | |
| Time to First Complete Response (CR) | Cohort 1 (sJIA and AOSD) | 8.4 | 95% Confidence Interval 5.0 to 20.9 | weeks | |
| Time to First Complete Response (CR) | Cohort 2 (SLE) | 5.9 | 95% Confidence Interval 3.4 to NA | weeks |
1 to 10 of 130Source ClinicalTrials.gov
See all 130 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 (sJIA and AOSD) | 25 | |
| Overall Study | STARTED | Cohort 2 (SLE) | 8 | |
| Overall Study | COMPLETED | Cohort 1 (sJIA and AOSD) | 23 | |
| Overall Study | COMPLETED | Cohort 2 (SLE) | 7 | |
| Overall Study | NOT COMPLETED | Cohort 1 (sJIA and AOSD) | 2 | |
| Overall Study | NOT COMPLETED | Cohort 2 (SLE) | 1 |
Source ClinicalTrials.gov
Adverse events216
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 | Cytomegalovirus infection reactivation | Infections and infestations | Cohort 1 (sJIA and AOSD) | 5 | 25 | 6 |
| other | Cytomegalovirus infection reactivation | Infections and infestations | Cohort 2 (SLE) | 0 | 8 | 0 |
| other | Cytomegalovirus infection | Infections and infestations | Cohort 1 (sJIA and AOSD) | 3 | 25 | 3 |
| other | Cytomegalovirus infection | Infections and infestations | Cohort 2 (SLE) | 2 | 8 | 2 |
| other | COVID-19 | Infections and infestations | Cohort 1 (sJIA and AOSD) | 3 | 25 | 4 |
| other | COVID-19 | Infections and infestations | Cohort 2 (SLE) | 1 | 8 | 1 |
| other | Sinusitis | Infections and infestations | Cohort 1 (sJIA and AOSD) | 2 | 25 | 2 |
| other | Sinusitis | Infections and infestations | Cohort 2 (SLE) | 2 | 8 | 2 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 1 (sJIA and AOSD) | 1 | 25 | 1 |
| other | Upper respiratory tract infection | Infections and infestations | Cohort 2 (SLE) | 3 | 8 | 3 |
1 to 10 of 216Source ClinicalTrials.gov
See all 216 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 (sJIA and AOSD) | 2 | 25 |
| Total, all-cause mortality | Cohort 2 (SLE) | 0 | 8 |
| Total, other adverse events | Cohort 1 (sJIA and AOSD) | 24 | 25 |
| Total, other adverse events | Cohort 2 (SLE) | 8 | 8 |
| Total, serious adverse events | Cohort 1 (sJIA and AOSD) | 18 | 25 |
| Total, serious adverse events | Cohort 2 (SLE) | 4 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons12
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Laboratory Parameters | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Study Interruption Due to Safety Reasons | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Withdrawal From the Study Due to Lack of Response | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Adverse Events (AEs) (Serious and Non-serious). | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Survival | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): MAS Recurrence at Anytime After Achievement of CR | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Proportion of Subjects With Overall Response as Defined by CR or Partial Response (PR) | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Proportion of Subjects With Complete Response (CR) at Week 8 After First Administration of Emapalumab | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): Number of Patients With GCs Tapering to ≤1mg/kg/Day of PDN Equivalent at Any Time Until End of Study (EOS). | Trial comparisons |
| Cohort 1 (sJIA and AOSD) vs Cohort 2 (SLE): deaths from any cause | Trial comparisons |
1 to 10 of 12
See all 12 trial comparisonsTests0
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/NCT05001737
GET https://conductscience.org/api/trials/v1/NCT05001737/results?page=2