Trial · NCT05744063
A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
NCT05744063, Phase 4, Completed, Swedish Orphan Biovitrum; results first posted 2026-03-13
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients. Trials, record NCT05744063. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05744063
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
- NCT number
- NCT05744063
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 13 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Primary Hemophagocytic Lymphohistiocytosis
- Interventions
- Emapalumab-Lzsg 5 MG/ML [Gamifant]
- First posted
- 2023-02-24
- Last updated
- 2026-08-26
- Results first posted
- 2026-03-13
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event | Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose | Number of participants permanently discontinuation of study drug due to emapalumab-related adverse event as judged by Investigator, until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose |
| Secondary | Overall Response | End of treatment or week 8 (whichever occurs earlier) | Number of participants with an overall response i.e., achievement of either Complete Response or Partial Response or HLH Improvement, at end of treatment or week 8 (whichever occurs earlier). |
| Secondary | Time to First Overall Response | End of treatment, likely within 6 months from first dose | Time to first overall response from first dose of study drug to the first achievement of response (Complete Response, Partial Response, or HLH Improvement) |
| Secondary | Cumulative Duration of Response | End of treatment, likely within 6 months from first dose | Cumulative duration of response is defined as total time in response from the first achievement of an Overall Response until EOT. For patients who achieve a response, lost that response, and then achieve it subsequently, the total time in response is calculated by adding together these separate periods in response. |
| Secondary | Ability to Reduce Glucocorticoids by 50% or More | End of treatment, likely within 6 months from first dose | Number of participants able to reduce glucocorticoids by 50% or more of the baseline dose at any time point of the treatment period |
| Secondary | Investigator Assessed Response | End of treatment | Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response |
| Secondary | Survival | End of study (1 year) | Number of participants surviving to start of HSCT conditioning and Number of participants that underwent HSCT surviving after HSCT to end of study |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event | Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Overall Response | End of treatment or week 8 (whichever occurs earlier) | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Time to First Overall Response | End of treatment, likely within 6 months from first dose | Two participants with no response are censored at the date of EOT visit or last assessment.~The analysis is conducted in all participants treated. | Days | MEDIAN |
| SECONDARY | Cumulative Duration of Response | End of treatment, likely within 6 months from first dose | The analysis is conducted in all participants treated excluding those (2 participants) who do not achieve response at least once between the date of first dose and EOT. | Days | MEAN |
| SECONDARY | Ability to Reduce Glucocorticoids by 50% or More | End of treatment, likely within 6 months from first dose | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Investigator Assessed Response | End of treatment | Two participants were not assessed at EOT or last assessment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Survival | End of study (1 year) | All participants are included in the Survival to start of HSCT conditioning. For survival after HSCT only participants that underwent HSCT are included in the measure, i.e. 9 participants | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event | Emapalumab | 0 | Participants | ||
| Overall Response | Emapalumab | Overall response | 10 | Participants | |
| Overall Response | Emapalumab | No Response | 2 | Participants | |
| Overall Response | Emapalumab | Missing | 1 | Participants | |
| Time to First Overall Response | Emapalumab | 4 | 95% Confidence Interval 3.00 to 5.00 | Days | |
| Cumulative Duration of Response | Emapalumab | 55.0 | Standard Deviation 33.36 | Days | |
| Ability to Reduce Glucocorticoids by 50% or More | Emapalumab | 6 | Participants | ||
| Investigator Assessed Response | Emapalumab | Complete Response | 2 | Participants | |
| Investigator Assessed Response | Emapalumab | Partial Response | 6 | Participants | |
| Investigator Assessed Response | Emapalumab | No response | 3 | Participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Emapalumab | 13 | |
| Overall Study | COMPLETED | Emapalumab | 9 | |
| Overall Study | NOT COMPLETED | Emapalumab | 4 |
Source ClinicalTrials.gov
Adverse events121
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 | Myelosuppression | Blood and lymphatic system disorders | Emapalumab | 10 | 13 | 10 |
| other | Anaemia | Blood and lymphatic system disorders | Emapalumab | 3 | 13 | 3 |
| other | Hypofibrinogenaemia | Blood and lymphatic system disorders | Emapalumab | 2 | 13 | 2 |
| other | Coagulopathy | Blood and lymphatic system disorders | Emapalumab | 1 | 13 | 1 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Emapalumab | 1 | 13 | 1 |
| other | Lymphopenia | Blood and lymphatic system disorders | Emapalumab | 1 | 13 | 1 |
| other | Splenomegaly | Blood and lymphatic system disorders | Emapalumab | 1 | 13 | 1 |
| other | Hepatic function abnormal | Hepatobiliary disorders | Emapalumab | 10 | 13 | 11 |
| other | Hyperbilirubinaemia | Hepatobiliary disorders | Emapalumab | 2 | 13 | 3 |
| other | Liver injury | Hepatobiliary disorders | Emapalumab | 1 | 13 | 2 |
1 to 10 of 121Source ClinicalTrials.gov
See all 121 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Emapalumab | 2 | 13 |
| Total, other adverse events | Emapalumab | 13 | 13 |
| Total, serious adverse events | Emapalumab | 7 | 13 |
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/NCT05744063
GET https://conductscience.org/api/trials/v1/NCT05744063/events?page=2