Trial · NCT02069899
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 14 of 14
Download this page as JSON| 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). |
| Secondary | Circulating Emapalumab Level (Enrolled-06 Cohort) | Baseline (first NI-0501-05 visit), Day 100, Month 6 | |
| Secondary | Total Human Interferon Gamma Levels (Enrolled-04 Cohort) | First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, Day 100 post-transplant, 12 months post-transplant | |
| Secondary | Total Human Interferon Gamma Levels (Enrolled-06 Cohort) | Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study | |
| Secondary | Number of Participants With Anti-drug Antibody | From enrolment up to 12 months post-transplant or last emapalumab infusion (maximum 639 days) |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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.
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