Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
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|ClinicalTrials.gov Identifier: NCT03312751|
Recruitment Status : Active, not recruiting
First Posted : October 18, 2017
Last Update Posted : November 5, 2021
The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, with special focus on long-term outcome and quality of life assessments. Emapalumab 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 available standard of care.
Emapalumab is to be administered until the start of conditioning, with an anticipated duration ranging from a minimum of 4 weeks to approximately 12 weeks and not exceeding 6 months.
After treatment completion, patients will continue in the study for long-term follow-up until 1 year after either HSCT or last emapalumab infusion (if HSCT is not performed).
|Condition or disease||Intervention/treatment||Phase|
|Primary Hemophagocytic Lymphohistiocytosis||Drug: Emapalumab||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||34 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFNγ) Monoclonal Antibody, and to Assess Its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients With Primary Hemophagocytic Lymphohistiocytosis|
|Actual Study Start Date :||February 5, 2019|
|Estimated Primary Completion Date :||October 2022|
|Estimated Study Completion Date :||October 2022|
Emapalumab will be administered by intravenous infusion, twice weekly.
- Overall Response at Week 8 or End of Treatment (if earlier) [ Time Frame: Up to Week 8 ]Achievement of either Complete or Partial Response or HLH Improvement, at Week 8 or EOT (whichever occurs earlier)
- Overall Survival [ Time Frame: Up to 18 months ]Including survival to HSCT and post-HSCT
- Number of patients proceeding to HSCT [ Time Frame: Up to 18 months ]Number of patients proceeding to HSCT
- Change in PedsQL Score [ Time Frame: Up to 18 months ]Assessment of the quality of life using the PedsQL "Pediatric Quality of Life Inventory"
- Incidence, severity, causality and outcomes of AEs (serious and non-serious) [ Time Frame: Up to 18 months ]Incidence of adverse events
- Immunogenicity [ Time Frame: Up to 18 months ]Incidence of circulating antibodies against emapalumab
- Overall Response at start of conditioning [ Time Frame: Up to 6 months ]Achievement of either Complete or Partial Response or HLH Improvement, at start of conditioning
- Serum concentrations of emapalumab [ Time Frame: Up to 18 months ]Serum concentrations of emapalumab
- Pharmacodynamic parameters [ Time Frame: Up to 18 months ]Levels of total IFNγ, markers of its neutralization (CXCL9 and CXCL10) and other relevant disease biomarkers e.g. sCD25
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03312751