Phase 3
Completed N=45
Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
Primary Haemophagocytic Lymphohistiocytosis
Source: ClinicalTrials.gov NCT01818492 ↗
Enrolled (actual)
45
Serious AEs
64.0%
Results posted
Sep 2020
Primary outcomePrimary: Overall Response Rate (ORR) Second Line — 63.0 percentage of participants — p=0.0134
◆ Published Evidence
Highly cited
511citations · ~85 / year
Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis.
Summary
The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.
Linked Publications (2)
-
Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis.
-
Population Pharmacokinetics of the Anti-Interferon-Gamma Monoclonal Antibody Emapalumab: An Updated Analysis.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Response Rate (ORR) Second Line |
63.0 | 0.0134 sig |
| PRIMARY Overall Response Rate (ORR) All Treated |
64.7 | 0.0031 sig |
| PRIMARY Overall Response Rate (ORR) Follow-on Analysis Set: |
58.8 | 0.0205 sig |
| PRIMARY Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated |
60.0 | 0.0053 sig |
| SECONDARY Time to Response |
7.0 | — |
| SECONDARY Durability of First Response |
58.0 | — |
| SECONDARY Overall Survival |
42 | — |
| SECONDARY Number of Patients Able to Reduce Glucocorticoids |
46.7; 11.1 | — |
| SECONDARY Cumulative Duration of Response |
78.6 | — |
| SECONDARY Survival Pre-HSCT |
0.83; 0.73 | — |
| SECONDARY Survival Post-HSCT |
0.82; 0.82 | — |
Eligibility Criteria
Inclusion Criteria
- Gender: male and female
- Age: up to and including 18 years at diagnosis of Haemophagocytic Lymphohistiocytosis
- Primary HLH patients
- Patient (if ≥ 18 years old), or patient's legal representative(s) must have signed informed consent
Exclusion Criteria
- Diagnosis of secondary Haemophagocytic Lymphohistiocytosis consequent to a proven rheumatic or neoplastic disease.
- Body weight < 3 kg.
- Patients treated with biologics within a specific timeframe
- Active Mycobacteria, Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections.
- Presence of malignancy.
- Concomitant disease or malformation severely affecting the cardiovascular, pulmonary, liver or renal function
Data sourced from ClinicalTrials.gov (NCT01818492) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.