Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
Trial · NCT03312751
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 18 of 18
Download this page as JSON| 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. |
| Secondary | Quality of Life Assessed Through PedsQL™, Pediatric Quality of Life Inventory™ | Up to 6 months | Assessment of the quality of life using the PedsQL "Pediatric Quality of Life Inventory". The PedsQL uses a 100-point scale ranging from 0 to 100 with higher values indicating better quality of life. |
| Secondary | Quality of Life Assessed Through Behavioral, Affective and Somatic Experiences Scales (BASES) | Up to 6 months | Assessment of the quality of life using the BASES questionnaire, which is a validated 38-item questionnaire; a reduced nonvalidated 22-item version of the questionnaire is used in an exploratory nature for the secondary endpoint. Subscale scores were calculated using a 5-point Likert scale from 1 to 5 for all questions. Scores for all questions in each subscale were added up and divided by the number of patients in the analysis population to reach the mean score. Subscale scores were calculated for the following domains: * Physical Discomfort (5 questions, '1' = best response, total range 5-25) * Cooperation/Compliance (5 questions, '1' = best response, total range 5-25) * Mood/Behavior (7 questions, '5' = best response, total range 7-35) * Quality of Interactions (3 questions, '1' = best response, total range 3-15) * Activity/Sleep (2 questions, '5' = best response for patient's activity level and '1' = best response for patient's sleeping, total range 2-10) |
| Secondary | Incidence, Severity, Causality and Outcomes of AEs (Serious and Non-serious) | Up to 18 months | Incidence of adverse events. SAE = serious adverse event; TEAE = treatment-emergent adverse event |
| Secondary | Evolution of Laboratory Parameters Change From Baseline to EOT/Week 8 | To Week 8 or End of treatment if before Week 8. | Number of patients experiencing shifts from baseline in the following relevant laboratory parameters are reported: * Biochemistry: glucose ferritin, C-reactive protein (CRP), liver function (alkaline phosphatase \[ALP\], alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], gamma glutamyl transferase \[γGT\], lactate dehydrogenase \[LDH\], bilirubin, renal function (albumin, creatinine, urea, urea nitrogen), triglycerides * Complete blood count: basophils, basophils/leukocytes, eosinophils, eosinophils/leukocytes, hematocrit, hemoglobin, large unstained cells, lymphocytes, lymphocytes/leukocytes, monocytes, monocytes/leukocytes, neutrophils band form, neutrophils band form/leukocytes, platelets, erythrocytes, leukocytes * Coagulation tests (activated partial thromboplastin time \[aPTT\], aPTT ratio, prothrombin time, prothrombin international normalized ratio \[INR\]), D-dimer, fibrinogen |
| Secondary | Number of Patients Who Discontinued Emapalumab Treatment | Up to 6 months | Number of patients who discontinued emapalumab treatment for safety reasons. |
| Other | Serum Concentrations of Emapalumab | Up to Week 8, with data presented at Baseline and EOT/W8 | The serum concentration of emapalumab will be measured as a function of time to determine the emapalumab PK profile. |
| Other | Change in Pharmacodynamic Parameters | Up to 18 months with data presented at Baseline and EOT/W8 | Levels (in ng/L) of total IFNγ (interferon gamma), markers of its neutralization (CXCL9 and CXCL10), and sCD25. |
| Other | 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. | The presence of circulating antibodies against emapalumab was inferred by positive results for anti-drug antibodies (ADAs). |
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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