Trial · NCT03312751
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
NCT03312751, Phase 3, Completed, Swedish Orphan Biovitrum; results first posted 2024-03-12
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis. Trials, record NCT03312751. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03312751
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
- NCT number
- NCT03312751
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 35 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Primary Hemophagocytic Lymphohistiocytosis
- Interventions
- Emapalumab
- First posted
- 2017-10-18
- Last updated
- 2024-03-12
- Results first posted
- 2024-03-12
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response at Week 8 or End of Treatment (if Earlier) | Up to Week 8 | The overall response rate (ORR) of patients achieving either Complete or Partial Response or HLH Improvement, at Week 8 or EOT (whichever occurs earlier). |
| Secondary | Overall Survival at End of Study | Up to 18 months | Number of patients surviving to the end of the study. |
| Secondary | Overall Survival to HSCT | From start of treatment to HSCT or from start of treatment until 1 year after EOT for patient who did not undergo HSCT | Number of patients surviving to HSCT. |
| Secondary | Overall Survival for Patients Receiving HSCT | Up to 1 year post HSCT | Number of patients surviving post HSCT. |
| Secondary | Event-free Survival | Up to 1 year post HSCT | Number of patients experiencing event-free survival, the duration of which was defined as time from HSCT to date of (whichever occurs first): death from any cause, graft failure, or HLH reactivation. |
| Secondary | Overall Response at Start of Conditioning | Up to 6 months | Number of patients achieving either a Complete or Partial Response or HLH Improvement, at start of conditioning (or at last emapalumab infusion if HSCT is not performed). |
| Secondary | Duration of Response | Up to 18 months | Duration of response, i.e., maintenance of the response achieved at any time during the study (with censoring time at start of conditioning for patients with no event) calculated only for patients showing confirmed overall response. Summarized by number of patients experiencing each category of response duration. |
| Secondary | Time to Response | Up to 18 months | Time to first response at any time during the study. |
| Secondary | Number of Patients Reducing Glucocorticoids by 50% or More of the Baseline Dose During Emapalumab Treatment | Up to 6 months | Number of patients able to reduce glucocorticoids by 50% or more of the baseline dose during emapalumab treatment. |
| Secondary | Number of Patients Proceeding to HSCT | Up to 18 months | Number of patients able to proceed to HSCT when deemed indicated. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response at Week 8 or End of Treatment (if Earlier) | Up to Week 8 | Percentage of participants | NUMBER | |
| SECONDARY | Overall Survival at End of Study | Up to 18 months | 'All-treated analysis set - treatment experienced' includes a censored observation for 1 patient who discontinued the study early. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Overall Survival to HSCT | From start of treatment to HSCT or from start of treatment until 1 year after EOT for patient who did not undergo HSCT | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Overall Survival for Patients Receiving HSCT | Up to 1 year post HSCT | All-treated analysis includes total number or patients who received HSCT. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Event-free Survival | Up to 1 year post HSCT | Analysis population based on the number of patients with HSCT. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Overall Response at Start of Conditioning | Up to 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Duration of Response | Up to 18 months | Only patients showing at least one maintained response were included. Duration of response is defined as maintenance of response for at least 4 days, achieved at any time during the study until EOT. Patients who do not achieve at least HLH improvement between the date of first dose and EOT are excluded from the analysis. For both first response and loss of response, persistence for at least 4 days is required, otherwise the next response/loss of response period will be considered. | days | MEDIAN |
| SECONDARY | Time to Response | Up to 18 months | Only patients with a response were included. | days | MEDIAN |
| SECONDARY | Number of Patients Reducing Glucocorticoids by 50% or More of the Baseline Dose During Emapalumab Treatment | Up to 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients Proceeding to HSCT | Up to 18 months | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group464
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Response at Week 8 or End of Treatment (if Earlier) | All-treated Analysis Set | 62.9 | 95% Confidence Interval 44.9 to 78.5 | Percentage of participants | |
| Overall Response at Week 8 or End of Treatment (if Earlier) | All-treated Analysis Set - Treatment naïve | 50.0 | 95% Confidence Interval 24.7 to 75.3 | Percentage of participants | |
| Overall Response at Week 8 or End of Treatment (if Earlier) | All-treated Analysis Set - Treatment Experienced | 73.7 | 95% Confidence Interval 48.8 to 90.9 | Percentage of participants | |
| Overall Survival at End of Study | All-treated Analysis Set | Alive at end of study | 19 | Participants | |
| Overall Survival at End of Study | All-treated Analysis Set - Treatment naïve | Alive at end of study | 9 | Participants | |
| Overall Survival at End of Study | All-treated Analysis Set - Treatment Experienced | Alive at end of study | 10 | Participants | |
| Overall Survival at End of Study | All-treated Analysis Set | Early discontinuation | 1 | Participants | |
| Overall Survival at End of Study | All-treated Analysis Set - Treatment naïve | Early discontinuation | 0 | Participants | |
| Overall Survival at End of Study | All-treated Analysis Set - Treatment Experienced | Early discontinuation | 1 | Participants | |
| Overall Survival to HSCT | All-treated Analysis Set | 25 | Participants |
1 to 10 of 464Source ClinicalTrials.gov
See all 464 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | All-treated Analysis Set - Treatment naïve | 16 | |
| Overall Study | STARTED | All-treated Analysis Set - Treatment Experienced | 19 | |
| Overall Study | COMPLETED | All-treated Analysis Set - Treatment naïve | 9 | |
| Overall Study | COMPLETED | All-treated Analysis Set - Treatment Experienced | 9 | |
| Overall Study | NOT COMPLETED | All-treated Analysis Set - Treatment naïve | 7 | |
| Overall Study | NOT COMPLETED | All-treated Analysis Set - Treatment Experienced | 10 |
Source ClinicalTrials.gov
Adverse events1,011
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 | Vomiting | Gastrointestinal disorders | All-treated Analysis Set - Treatment naïve | 7 | 16 | 10 |
| other | Vomiting | Gastrointestinal disorders | All-treated Analysis Set - Treatment Experienced | 10 | 19 | 15 |
| other | Vomiting | Gastrointestinal disorders | All-treated Analysis Set | 17 | 35 | 25 |
| other | Diarrhoea | Gastrointestinal disorders | All-treated Analysis Set - Treatment naïve | 5 | 16 | 7 |
| other | Diarrhoea | Gastrointestinal disorders | All-treated Analysis Set - Treatment Experienced | 10 | 19 | 16 |
| other | Diarrhoea | Gastrointestinal disorders | All-treated Analysis Set | 15 | 35 | 23 |
| other | Abdominal pain | Gastrointestinal disorders | All-treated Analysis Set - Treatment naïve | 2 | 16 | 5 |
| other | Abdominal pain | Gastrointestinal disorders | All-treated Analysis Set - Treatment Experienced | 5 | 19 | 6 |
| other | Abdominal pain | Gastrointestinal disorders | All-treated Analysis Set | 7 | 35 | 11 |
| other | Stomatitis | Gastrointestinal disorders | All-treated Analysis Set - Treatment naïve | 2 | 16 | 2 |
1 to 10 of 1,011Source ClinicalTrials.gov
See all 1,011 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | All-treated Analysis Set - Treatment naïve | 7 | 16 |
| Total, all-cause mortality | All-treated Analysis Set - Treatment Experienced | 9 | 19 |
| Total, all-cause mortality | All-treated Analysis Set | 16 | 35 |
| Total, other adverse events | All-treated Analysis Set - Treatment naïve | 14 | 16 |
| Total, other adverse events | All-treated Analysis Set - Treatment Experienced | 19 | 19 |
| Total, other adverse events | All-treated Analysis Set | 35 | 35 |
| Total, serious adverse events | All-treated Analysis Set - Treatment naïve | 16 | 16 |
| Total, serious adverse events | All-treated Analysis Set - Treatment Experienced | 16 | 19 |
| Total, serious adverse events | All-treated Analysis Set | 32 | 35 |
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/NCT03312751
GET https://conductscience.org/api/trials/v1/NCT03312751/results?page=2