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
Number of participants permanently discontinuation of study drug due to emapalumab-related adverse event as judged by Investigator, until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Time frame: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Overall Response
Number of participants with an overall response i.e., achievement of either Complete Response or Partial Response or HLH Improvement, at end of treatment or week 8 (whichever occurs earlier).
Time frame: End of treatment or week 8 (whichever occurs earlier)
Time to First Overall Response
Time to first overall response from first dose of study drug to the first achievement of response (Complete Response, Partial Response, or HLH Improvement)
Time frame: End of treatment, likely within 6 months from first dose
Cumulative Duration of Response
Cumulative duration of response is defined as total time in response from the first achievement of an Overall Response until EOT. For patients who achieve a response, lost that response, and then achieve it subsequently, the total time in response is calculated by adding together these separate periods in response.
Time frame: End of treatment, likely within 6 months from first dose
Ability to Reduce Glucocorticoids by 50% or More
Number of participants able to reduce glucocorticoids by 50% or more of the baseline dose at any time point of the treatment period
Time frame: End of treatment, likely within 6 months from first dose
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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
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
Survival
Number of participants surviving to start of HSCT conditioning and Number of participants that underwent HSCT surviving after HSCT to end of study
Time frame: End of study (1 year)