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.

SexALL

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

✓. 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.

✕. Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.
✕. Active mycobacteria, Histoplasma capsulatum, Salmonella, or Leishmania infections.
✕. Evidence of latent tuberculosis.
✕. Presence of malignancy.
✕. Existence of any severe co-morbidity or any other medical condition which, in the opinion of the investigator, makes the patient unsuitable for the treatment
✕. History of hypersensitivity or allergy to any component of the study regimen (e.g., polysorbate).
✕. Receipt of a Bacillus Calmette-Guérin (BCG) vaccine within 12 weeks prior to Screening.
✕. Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to Screening.

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

NCT IDNCT05744063

SponsorSwedish Orphan Biovitrum

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-02-21

Results submitted2026-02-04

Who can participate

SexALL

See this in plain English?

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

✓. 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.

✕. Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.
✕. Active mycobacteria, Histoplasma capsulatum, Salmonella, or Leishmania infections.
✕. Evidence of latent tuberculosis.
✕. Presence of malignancy.
✕. Existence of any severe co-morbidity or any other medical condition which, in the opinion of the investigator, makes the patient unsuitable for the treatment
✕. History of hypersensitivity or allergy to any component of the study regimen (e.g., polysorbate).
✕. Receipt of a Bacillus Calmette-Guérin (BCG) vaccine within 12 weeks prior to Screening.
✕. Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to Screening.