A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
Hemophagocytic Lymphohistiocytosis
About this trial
This is an interventional other trial for Hemophagocytic Lymphohistiocytosis focused on measuring Hemophagocytic lymphohistiocytosis (HLH) previously treated with NI-0501, Emapalumab
Eligibility Criteria
Inclusion Criteria:
- Having received at least one dose of emapalumab.
- Having signed the Informed Consent by the participant or the participant's legal representative(s), as applicable, with the assent of participant who are legally capable of providing it.
Exclusion Criteria:
- None
Sites / Locations
- Spectrum Health Helen Devos Children's Hospital
- University of North Carolina at Chapel Hill
- Cincinnati Children's Hospital - Division of Immunobiology
- Cincinnati Children's Hospital
- Texas Children's Cancer Center
- Hôpital Necker-Enfants Malades
- Fondazione MBBM c/o Ospedale San Gerardo Clinica Pediatrica
- Azienda Ospedaliera Padova
- Ospedale Pediatrico Bambino Gesu - UO Reumatologia
- Ospedale Pediatrico Bambino Gesu
- Ospedale della Donna e del Bambino - U.O.C. Oncoematologia Pediatrica
- Hospital Universitario Vall d'Hebron Servei de Hematologia i Oncologia
- Sant Joan de Déu Hospital - Pediatric Rheumatology Department
- Hospital Universitario Niño Jesús Servicio de Hemato-Oncología Pediátrica
- UCL Institute of Child Health Great Ormond Street Hospital
- Great Ormond Street Hospital - Department of Haematology
Arms of the Study
Arm 1
Arm 2
Arm 3
Other
No Intervention
Other
Enrolled-04 Cohort
Enrolled-06 Cohort
Enrolled-CU Cohort
Participants enrolled in Study NI-0501-04 (NCT01818492) will be invited to participate for long-term follow-up for 1 year either after haematopoietic stem cell transplantation (HSCT) or after the last administration of emapalumab. In Study NI-0501-04, participants received emapalumab for 4 to 8 weeks. After the treatment period, participants could have undergone HSCT. Participants for whom an appropriate donor was not identified by Week 8, or in a case where HSCT will be delayed for reasons unrelated to the administration of emapalumab, can continue receiving treatment with emapalumab beyond the foreseen 8 weeks in the current study (NI-0501-05, NCT02069899) at the request of the investigator, providing a favorable benefit/risk assessment of treatment is established. The dose and timing was either carried forward from the last administered emapalumab dose as part of the parent protocol or an adjusted dose was administered, if necessary.
All participants who received at least 1 dose of emapalumab and were monitored for at least 4 weeks after the last drug administration in Study NI-0501-06 (NCT03311854) will be invited to participate for long-term follow-up for 1 year after the last administration of emapalumab. Participants will not receive emapalumab in the current study (NI-0501-05, NCT02069899).
In exceptional cases, at the spontaneous request of a treating physician, CU treatment will be granted to the participants who had exhausted all possible treatment options and who could not be enrolled in a clinical study. All participants who receive at least 1 dose of emapalumab under CU will be invited to participate for long-term follow-up for 1 year either after HSCT or after the last administration of emapalumab. Participants can continue treatment in the context of the current Study (NI-0501-05, NCT02069899) while stem cell donor search is ongoing, or if the investigator assesses that continuation of treatment is beneficial.