Trial · NCT03985423
A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH
NCT03985423, Phase 2, Phase 3, Terminated, Swedish Orphan Biovitrum; results first posted 2023-10-06
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH. Trials, record NCT03985423. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03985423
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH
- NCT number
- NCT03985423
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 7 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Hemophagocytic Lymphohistiocytoses
- Interventions
- Emapalumab-Lzsg
- First posted
- 2019-06-13
- Last updated
- 2023-10-06
- Results first posted
- 2023-10-06
- Limitations and caveats
Since the study was prematurely terminated, and considering the low number of patients enrolled, no analysis related to primary and secondary efficacy endpoints and no Pharmacokinetic and Pharmacodynamic summary statistics were performed. Accordingly, no conclusion on the efficacy of emapalumab on this patient population could be withdrawn from the study.
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response | Week 4 | Achievement of either a Complete or Partial Response Complete Response is adjudicated if the following are observed: * No fever = body temperature \<37.5°C * Normal spleen size * No cytopenia = Absolute Neutrophil Counts \>=1.0x10\^9/L and platelet count \>=100x10\^9/L \[absence of G-CSF and transfusion support must be documented for at least 4 days to report no cytopenia\] * No hyperferritinemia = serum level is \<2000 µg/L * No evidence of coagulopathy, i.e., normal D-Dimer and/or normal (\>150 mg/dL) fibrinogen levels * No neurological and CSF abnormalities attributed to HLH * No sustained worsening of sCD25 (as indicated by at least two consecutive measurements that are \>2-fold higher than baseline) Partial Response is adjudicated if there is an improvement (\>50% change from baseline or normalization) of at least 3 HLH clinical and laboratory criteria (including Central Nervous System abnormalities). |
| Secondary | Best Response on Treatment | Week 4; End of Treatment Visit (on average of 12 weeks) | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Overall Response | End of Treatment Visit (on average of 12 weeks) | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Overall Survival | End of Treatment Visit (on average of 12 weeks) | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Time to Complete Response or Partial Response | Week 4; End of Treatment visit (on average of 12 weeks) | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Duration of Response | Up to 1 year after last emapalumab administration | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Hemophagocytic Lymphohistiocytosis Relapse | Up to 1 year after last emapalumab administration | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Incidence, Severity, Causality and Outcomes of Serious Adverse Events and Non-serious Adverse Events | Up to 1 year after last emapalumab administration | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Serum Concentrations of Emapalumab | Up to 1 year after last emapalumab administration | As no data are reported for this outcome measure, additional method is not applicable in the outcome measure description. |
| Secondary | Serum Biomarker Levels | Up to 1 year after last emapalumab administration | Levels of interferon-gamma, C-X-C chemokine ligand 9, soluble CD25, interleukin-6. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response | Week 4 | Since the study was prematurely terminated, no data was collected for the primary efficacy endpoint. | ||
| SECONDARY | Best Response on Treatment | Week 4; End of Treatment Visit (on average of 12 weeks) | Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints. | ||
| SECONDARY | Overall Response | End of Treatment Visit (on average of 12 weeks) | Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints. | ||
| SECONDARY | Overall Survival | End of Treatment Visit (on average of 12 weeks) | Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints. | ||
| SECONDARY | Time to Complete Response or Partial Response | Week 4; End of Treatment visit (on average of 12 weeks) | Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints. | ||
| SECONDARY | Duration of Response | Up to 1 year after last emapalumab administration | Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints. | ||
| SECONDARY | Hemophagocytic Lymphohistiocytosis Relapse | Up to 1 year after last emapalumab administration | Since the study was prematurely terminated, no data was collected for the secondary efficacy endpoints. | ||
| SECONDARY | Incidence, Severity, Causality and Outcomes of Serious Adverse Events and Non-serious Adverse Events | Up to 1 year after last emapalumab administration | Since the study was prematurely terminated, no data was collected for the secondary safety endpoints. | ||
| SECONDARY | Serum Concentrations of Emapalumab | Up to 1 year after last emapalumab administration | Since the study was prematurely terminated, no data was collected for the secondary Pharmacokinetic endpoints. | ||
| SECONDARY | Serum Biomarker Levels | Up to 1 year after last emapalumab administration | Since the study was prematurely terminated, no data was collected for the secondary Pharmacodynamic endpoints. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Emapalumab | 7 | |
| Overall Study | COMPLETED | Emapalumab | 0 | |
| Overall Study | NOT COMPLETED | Emapalumab | 7 |
Source ClinicalTrials.gov
Adverse events77
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 | Hypercoagulation | Blood and lymphatic system disorders | Emapalumab | 1 | 7 | 1 |
| other | Splenomegaly | Blood and lymphatic system disorders | Emapalumab | 1 | 7 | 1 |
| other | Sinus tachycardia | Cardiac disorders | Emapalumab | 2 | 7 | 2 |
| other | Tachycardia | Cardiac disorders | Emapalumab | 1 | 7 | 1 |
| other | Constipation | Gastrointestinal disorders | Emapalumab | 1 | 7 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Emapalumab | 1 | 7 | 1 |
| other | Diverticulum intestinal | Gastrointestinal disorders | Emapalumab | 1 | 7 | 1 |
| other | Haematochezia | Gastrointestinal disorders | Emapalumab | 1 | 7 | 1 |
| other | Haemorrhoids | Gastrointestinal disorders | Emapalumab | 1 | 7 | 1 |
| other | Lip disorder | Gastrointestinal disorders | Emapalumab | 1 | 7 | 1 |
1 to 10 of 77Source ClinicalTrials.gov
See all 77 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Emapalumab | 4 | 7 |
| Total, other adverse events | Emapalumab | 7 | 7 |
| Total, serious adverse events | Emapalumab | 5 | 7 |
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 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/NCT03985423
GET https://conductscience.org/api/trials/v1/NCT03985423/events?page=2