Trial · NCT02069899
A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
NCT02069899, Phase 2, Phase 3, Completed, Swedish Orphan Biovitrum; results first posted 2022-06-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody. Trials, record NCT02069899. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02069899
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
- NCT number
- NCT02069899
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 58 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Hemophagocytic Lymphohistiocytosis
- Interventions
- Emapalumab
- First posted
- 2014-02-24
- Last updated
- 2022-06-28
- Results first posted
- 2022-06-28
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Event (AE) | From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days) | Adverse events were defined as any undesirable experience occurring in a participant during the study, whether or not considered related to emapalumab. |
| Secondary | Cumulative Duration of Response (Enrolled-04 Cohort) | From 1st achievement of overall response until HSCT or last treatment date if participant did not undergo HSCT (maximum 250 days) | Cumulative duration of response: total number of days in response from 1st achievement of overall response until HSCT or last treatment date if the participant did not undergo HSCT. Overall response: achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI). CR: no fever, normal spleen size, no cytopenia (absolute neutrophil count \[ANC\] ≥1.0 x 10\^9/L and platelet count ≥ 100 x 10\^9/L), no hyperferritinemia (serum ferritin \<2000 μg/L), no coagulopathy (normal D-dimer and/or fibrinogen \>150 mg/dL), no neurological and cerebrospinal fluid \[CSF\] abnormalities attributed to HLH, no sustained worsening of soluble cluster of differentiation (CD) 25. PR: at least 3 HLH clinical and laboratory criteria (including central nervous system \[CNS\] abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology. HI: improvement (\>50% change from baseline) of at least 3 HLH clinical and laboratory abnormalities (including CNS involvement). |
| Secondary | Duration of First Response (Enrolled-06 Cohort) | From first date of response and first date of loss of response or death (maximum 416 days) | Duration of first response was defined as the number of days between first date of response and first date of loss of response or death. Response was defined as macrophage activation syndrome (MAS) remission, which was resolution of clinical signs and symptoms according to the Investigator (MAS clinical signs and symptoms score ≤ 1) and normalization of laboratory parameters relevant to MAS as follows: white blood cells (WBC) and platelet count above the upper limit of normal (LLN), Lactate dehydrogenase \< 1.5 × lower limit of normal (ULN), aspartate aminotransferase/alanine aminotransferase \<1.5 × ULN, fibrinogen \> 100 mg/dL, ferritin level decreased by at least 80% from values at screening or baseline (whichever was higher) or \< 2000 ng/mL, whichever was lower. |
| Secondary | Overall Survival (Enrolled-04 Cohort) | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) | Overall survival was defined as time from the date of the last emapalumab dose to the date of death. Participants without an event were censored at the time of last contact or 12 months after last dose (whichever came first). As some participants had their last emapalumab dose in the parent study (NI-0501-04), data from both NI-0501-05 and NI-0501-04 studies were considered for the assessment of overall survival. Kaplan-Meier methodology was used for estimation. |
| Secondary | Overall Survival (Enrolled-06 Cohort) | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) | Overall survival was defined as time from the date of last emapalumab dose to the date of death. Participants without an event were censored at the time of last contact or 12 months after last dose (whichever came first). As participants in the Enrolled-06 Cohort did not receive emapalumab in the current study, data from both NI-0501-05 and NI-0501-06 studies were considered for the assessment of overall survival. Kaplan-Meier methodology was used for estimation. |
| Secondary | Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort) | From HSCT up to 12 months | For participants who underwent HSCT either in parent study (NI-0501-04) or current study (NI-0501-05), engraftment rate was based on the number of participants experiencing primary or secondary graft failure (blood stem cell transplant failure, engraft failure, or transplant dysfunction), as reported as an adverse event. |
| Secondary | Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort) | From HSCT to 12 months | For participants who underwent HSCT, achievement of donor chimerism was considered based on donor chimerism in peripheral blood completed, that is, donor cells \>95%. |
| Secondary | Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort) | From HSCT to 12 months | Occurrence of graft-versus-host-disease, reported in Study NI-0501-05 as an AE. |
| Secondary | MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort) | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study | MAS activity was monitored using a visual analogue scale ranging from 0 to 10 with a higher score indicted higher disease activity. |
| Secondary | Circulating Emapalumab Level (Enrolled-04 Cohort) | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, last infusion day (infusion Day 188), 12 months post-transplant | Circulating Emapalumab level in Enrolled-04 Cohort who continued to receive treatment with emapalumab in the current study (NI-0501-05). |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Event (AE) | From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days) | Participants who previously received treatment in a parent study (NI-0501-04 or NI-0501-06) or under compassionate use and who provided informed consent for participation in Study NI-0501-05. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Cumulative Duration of Response (Enrolled-04 Cohort) | From 1st achievement of overall response until HSCT or last treatment date if participant did not undergo HSCT (maximum 250 days) | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who had at least 1 response. | days | MEAN |
| SECONDARY | Duration of First Response (Enrolled-06 Cohort) | From first date of response and first date of loss of response or death (maximum 416 days) | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05 and who had at least 1 response. | days | MEDIAN |
| SECONDARY | Overall Survival (Enrolled-04 Cohort) | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05. | days | MEDIAN |
| SECONDARY | Overall Survival (Enrolled-06 Cohort) | From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days) | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05. | days | MEDIAN |
| SECONDARY | Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort) | From HSCT up to 12 months | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who underwent HSCT. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort) | From HSCT to 12 months | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who underwent HSCT. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort) | From HSCT to 12 months | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who underwent HSCT. | percentage of participants | NUMBER |
| SECONDARY | MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort) | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study | Enrolled-06 population included participants previously enrolled and treated in Study NI-0501-06 (participants with MAS in Still's disease) who provided informed consent for participation in Study NI-0501-05 and had available data for MAS activity. | score on a scale | MEAN |
| SECONDARY | Circulating Emapalumab Level (Enrolled-04 Cohort) | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, last infusion day (infusion Day 188), 12 months post-transplant | Enrolled-04 population included participants previously enrolled and treated in Study NI-0501-04 (participants with primary HLH) who provided informed consent for participation in Study NI-0501-05 and who had data available for specified timepoints. Circulating emapalumab level post-transplant included participants who had HSCT and data available at 12 months post-HSCT (Samples were not to be taken once it had been determined that emapalumab was below measurable level of 62.5 µg/L). | µg/L | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group28
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Event (AE) | Enrolled-04 Cohort | 37 | Participants | ||
| Number of Participants With Adverse Event (AE) | Enrolled-06 Cohort | 12 | Participants | ||
| Number of Participants With Adverse Event (AE) | Enrolled-CU Cohort | 7 | Participants | ||
| Cumulative Duration of Response (Enrolled-04 Cohort) | Enrolled-04 Cohort | 70.7 | Standard Deviation 56.35 | days | |
| Duration of First Response (Enrolled-06 Cohort) | Enrolled-06 Cohort | 61.0 | Inter-Quartile Range 9.0 to 358.0 | days | |
| Overall Survival (Enrolled-04 Cohort) | Enrolled-04 Cohort | NA | Inter-Quartile Range NA to NA | days | |
| Overall Survival (Enrolled-06 Cohort) | Enrolled-06 Cohort | NA | Inter-Quartile Range NA to NA | days | |
| Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort) | Enrolled-04 Cohort | 20.7 | 95% Confidence Interval 9.8 to 38.4 | percentage of participants | |
| Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort) | Enrolled-04 Cohort | 72.4 | 95% Confidence Interval 54.3 to 85.3 | percentage of participants | |
| Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort) | Enrolled-04 Cohort | 24.1 | 95% Confidence Interval 12.2 to 42.1 | percentage of participants |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Enrolled-04 Cohort | 37 | |
| Overall Study | STARTED | Enrolled-06 Cohort | 14 | |
| Overall Study | STARTED | Enrolled-CU Cohort | 7 | |
| Overall Study | Treated in Current Study | Enrolled-04 Cohort | 22 | |
| Overall Study | Treated in Current Study | Enrolled-06 Cohort | 0 | |
| Overall Study | Treated in Current Study | Enrolled-CU Cohort | 5 | |
| Overall Study | COMPLETED | Enrolled-04 Cohort | 24 | |
| Overall Study | COMPLETED | Enrolled-06 Cohort | 13 | |
| Overall Study | COMPLETED | Enrolled-CU Cohort | 4 | |
| Overall Study | NOT COMPLETED | Enrolled-04 Cohort | 13 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events1,056
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 | Iron deficiency anaemia | Blood and lymphatic system disorders | Enrolled-04 Cohort | 2 | 37 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Enrolled-06 Cohort | 0 | 14 | |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Enrolled-CU Cohort | 0 | 7 | |
| other | Thrombotic microangiopathy | Blood and lymphatic system disorders | Enrolled-04 Cohort | 2 | 37 | |
| other | Thrombotic microangiopathy | Blood and lymphatic system disorders | Enrolled-06 Cohort | 0 | 14 | |
| other | Thrombotic microangiopathy | Blood and lymphatic system disorders | Enrolled-CU Cohort | 0 | 7 | |
| other | Anaemia | Blood and lymphatic system disorders | Enrolled-04 Cohort | 1 | 37 | |
| other | Anaemia | Blood and lymphatic system disorders | Enrolled-06 Cohort | 0 | 14 | |
| other | Anaemia | Blood and lymphatic system disorders | Enrolled-CU Cohort | 0 | 7 | |
| other | Haemolytic anaemia | Blood and lymphatic system disorders | Enrolled-04 Cohort | 1 | 37 |
1 to 10 of 1,056Source ClinicalTrials.gov
See all 1,056 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Enrolled-04 Cohort | 9 | 37 |
| Total, all-cause mortality | Enrolled-06 Cohort | 0 | 14 |
| Total, all-cause mortality | Enrolled-CU Cohort | 2 | 7 |
| Total, other adverse events | Enrolled-04 Cohort | 37 | 37 |
| Total, other adverse events | Enrolled-06 Cohort | 11 | 14 |
| Total, other adverse events | Enrolled-CU Cohort | 7 | 7 |
| Total, serious adverse events | Enrolled-04 Cohort | 28 | 37 |
| Total, serious adverse events | Enrolled-06 Cohort | 3 | 14 |
| Total, serious adverse events | Enrolled-CU Cohort | 6 | 7 |
Source ClinicalTrials.gov
Papers3
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/NCT02069899
GET https://conductscience.org/api/trials/v1/NCT02069899/results?page=2