CompletedPhase 4

A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients

China13 participantsStarted 2023-02-03

Plain-language summary

The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Male and female HLH patients of any age.
. Patients diagnosed with confirmed or suspected pHLH, based on; a molecular diagnosis or familial history consistent with pHLH or fulfilment of HLH-2004 diagnostic criteria, i.e., five out of eight of the criteria below:
. Presence of active HLH disease as assessed by the investigator.
. Patients must fulfil one of the following criteria as assessed by the investigator:
. Expectation of survival beyond 1 week as judged by the investigator.
. Patient has expectation of proceeding to HSCT
. Informed consent signed by the patient (as required by local law), or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable.
. Willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug, if female and of childbearing potential.

Exclusion criteria

. Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event

Timeframe: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose

Trial details

NCT IDNCT05744063

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-02-21

Results submitted2026-02-04

View on ClinicalTrials.gov More Primary Hemophagocytic Lymphohistiocytosis trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Male and female HLH patients of any age.
. Patients diagnosed with confirmed or suspected pHLH, based on; a molecular diagnosis or familial history consistent with pHLH or fulfilment of HLH-2004 diagnostic criteria, i.e., five out of eight of the criteria below:
. Presence of active HLH disease as assessed by the investigator.
. Patients must fulfil one of the following criteria as assessed by the investigator:
. Expectation of survival beyond 1 week as judged by the investigator.
. Patient has expectation of proceeding to HSCT
. Informed consent signed by the patient (as required by local law), or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable.
. Willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug, if female and of childbearing potential.

Exclusion criteria

. Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.