Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
Trial · NCT03312751
Outcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 18 of 18
Download this page as JSON| 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 | |
| SECONDARY | Quality of Life Assessed Through PedsQL™, Pediatric Quality of Life Inventory™ | Up to 6 months | Results from the PedsQL were not available for all patients; in addition, due to the age and treatment history of patients, not all age categories were represented in each of the analysis sets. | scale on a score | MEAN |
| SECONDARY | Quality of Life Assessed Through Behavioral, Affective and Somatic Experiences Scales (BASES) | Up to 6 months | Assessment completed at the end of treatment for those who proceeded/were planned to proceed to HSCT. | scale on a score | MEAN |
| SECONDARY | Incidence, Severity, Causality and Outcomes of AEs (Serious and Non-serious) | Up to 18 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Evolution of Laboratory Parameters Change From Baseline to EOT/Week 8 | To Week 8 or End of treatment if before Week 8. | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Patients Who Discontinued Emapalumab Treatment | Up to 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| OTHER_PRE_SPECIFIED | Serum Concentrations of Emapalumab | Up to Week 8, with data presented at Baseline and EOT/W8 | The number of participants analyzed at each timepoint reflects the number of participants who provided samples. | ng/mL | MEAN |
| OTHER_PRE_SPECIFIED | Change in Pharmacodynamic Parameters | Up to 18 months with data presented at Baseline and EOT/W8 | The number of participants analyzed at each timepoint reflects the number of participants who provided samples. | ng/L | MEAN |
| OTHER_PRE_SPECIFIED | Number of Patients Who Demonstrated a Presence of Circulating Antibodies Against Emapalumab to Determine Immunogenicity | Up to 1 year follow up post end of treatment with assessments at first dose of emapalumab, Week 4, Week 8, EOT and following treatment at day 100 and at the 1 year follow up visit, with data presented at study day 21 and EOT/Week 8. | Number of patients who demonstrated a presence of circulating antibodies shown only for visits where a positive result was recorded. | Participants | COUNT_OF_PARTICIPANTS |
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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