The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH.
This is an open-label, multi center, single arm, post-authorization study aiming to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with confirmed or suspected primary hemophagocytic lymphohistiocytosis (pHLH). The main objectives of the study are to collect safety and efficacy data on emapalumab in treatment experienced Chinese pHLH patients
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Swedish Orphan Biovitrum Research site
Shanghai, Fudan, China
Swedish Orphan Biovitrum Research site
Beijing, Xicheng, China
Swedish Orphan Biovitrum Research site
Beijing, China
Swedish Orphan Biovitrum Research site
Chongqing, China
Permanent discontinuation of study drug due to emapalumab-related adverse event
as judged by Investigator
Time frame: until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Overall response
measured as complete response, partial response, or HLH improvement
Time frame: end of treatment or week 8 (whichever occurs earlier)
Time to first overall response
from first dose of study drug to first response
Time frame: end of treatment, likely within 6 months from first dose
Cumulative duration of response
total time in response
Time frame: end of treatment, likely within 6 months from first dose
Ability to reduce glucocorticoids by 50% or more
reduction from baseline dose during emapalumab treatment
Time frame: end of treatment, likely within 6 months from first dose
Investigator assessed response
Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response
Time frame: end of treatment or week 8 (whichever occurs earlier)
Survival
To start of HSCT conditioning and after time from HSCT to death
Time frame: end of study
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Swedish Orphan Biovitrum Research site
Guangzhou, China
Swedish Orphan Biovitrum Research site
Nanjing, China
Swedish Orphan Biovitrum Research site
Zhengzhou, China