Clinical trial NCT02069899: 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
Official title: A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hemophagocytic Lymphohistiocytosis
Keywords: Hemophagocytic lymphohistiocytosis (HLH) previously treated with NI-0501
Emapalumab
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
Collaborators: name: Seventh Framework Programme
First posted: 2014-02-24
Enrollment: 58
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under compassionate use (CU).

Detailed description: The aim of this study is to monitor the long-term safety profile of emapalumab in participants who have previously received at least one dose of emapalumab, including survival time after the administration of emapalumab.

Moreover, the elimination profile of emapalumab and the immunogenicity will also be assessed.

Furthermore, safety, tolerability, efficacy, and pharmacokinetic (PK) profile of emapalumab will be closely monitored in the event that some participants, upon request of the treating physician, will receive emapalumab treatment in the follow-up study.

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

Primary outcomes:
measure: Number of Participants With Adverse Event (AE)
timeFrame: From the date of enrollment in this study up to 1 year either after HSCT or after the last administration of emapalumab (maximum duration: 639 days)
Secondary outcomes:
measure: Cumulative Duration of Response (Enrolled-04 Cohort)
timeFrame: From 1st achievement of overall response until HSCT or last treatment date if participant did not undergo HSCT (maximum 250 days)
measure: Duration of First Response (Enrolled-06 Cohort)
timeFrame: From first date of response and first date of loss of response or death (maximum 416 days)
measure: Overall Survival (Enrolled-04 Cohort)
timeFrame: From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days)
measure: Overall Survival (Enrolled-06 Cohort)
timeFrame: From the date of last of emapalumab dose to the date of death or last contact or 12 months after last dose, whichever came first (maximum 366 days)
measure: Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort)
timeFrame: From HSCT up to 12 months
measure: Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort)
timeFrame: From HSCT to 12 months
measure: Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort)
timeFrame: From HSCT to 12 months
measure: MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort)
timeFrame: Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study
measure: Circulating Emapalumab Level (Enrolled-04 Cohort)
timeFrame: First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, last infusion day (infusion Day 188), 12 months post-transplant
measure: Circulating Emapalumab Level (Enrolled-06 Cohort)
timeFrame: Baseline (first NI-0501-05 visit), Day 100, Month 6
measure: Total Human Interferon Gamma Levels (Enrolled-04 Cohort)
timeFrame: First infusion day (infusion duration: 1-2 hours) in Study NI-0501-05, Day 100 post-transplant, 12 months post-transplant
measure: Total Human Interferon Gamma Levels (Enrolled-06 Cohort)
timeFrame: Baseline (first NI-0501-05 visit), Day 100, Month 12/End of Study
measure: Number of Participants With Anti-drug Antibody
timeFrame: From enrolment up to 12 months post-transplant or last emapalumab infusion (maximum 639 days)
Other outcomes:
Locations:
city: Grand Rapids
country: United States
facility: Spectrum Health Helen Devos Children's Hospital
state: Michigan
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital - Division of Immunobiology
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Houston
country: United States
facility: Texas Children's Cancer Center
state: Texas
city: Paris
country: France
facility: Hôpital Necker-Enfants Malades
city: Monza
country: Italy
facility: Fondazione MBBM c/o Ospedale San Gerardo Clinica Pediatrica
city: Padova
country: Italy
facility: Azienda Ospedaliera Padova
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu - UO Reumatologia
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Verona
country: Italy
facility: Ospedale della Donna e del Bambino - U.O.C. Oncoematologia Pediatrica
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron Servei de Hematologia i Oncologia
city: Barcelona
country: Spain
facility: Sant Joan de Déu Hospital - Pediatric Rheumatology Department
city: Madrid
country: Spain
facility: Hospital Universitario Niño Jesús Servicio de Hemato-Oncología Pediátrica
city: London
country: United Kingdom
facility: UCL Institute of Child Health Great Ormond Street Hospital
city: London
country: United Kingdom
facility: Great Ormond Street Hospital - Department of Haematology
Study officials:
References:
citation: Jordan MB, Locatelli F, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington T, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, Grom A, De Benedetti F, de Min C. Post-Transplant Outcomes of Children with Primary Hemophagocytic Lymphohistiocytosis Treated with Emapalumab.Transplant Cell Ther. 2021; 27(Supplement 3): S118.
citation: Laveille C, Jacqmin P, de Graaf K, de Min C. Population Pharmacokinetic Analysis of Emapalumab, a Fully Human, Anti-Interferon Gamma Monoclonal Antibody, in Children with Primary Hemophagocytic Lymphohistiocytosis. Blood 2020; 136 (Supplement 1): 20
citation: Locatelli F, Jordan MB, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington TP, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, De Benedetti F, Grom A, Lapeyre G, Jacqmin P, Ballabio M, de Min C. Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326.