Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE
- Conditions
- Macrophage Activation SyndromeSJIAMASSecondary Hemophagocytic LymphohistiocytosisStill DiseaseSystemic Lupus ErythematosusAOSD
- Interventions
- Registration Number
- NCT05001737
- Lead Sponsor
- Swedish Orphan Biovitrum
- Brief Summary
The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic juvenile idiopathic arthritis and adult onset Still's disease) or with sHLH/MAS in systemic lupus erythematous, resenting an inadequate response to high dose glucocorticoid treatment.
- Detailed Description
Study NI-0501-14 is a two-cohort trial that enrolls subjects who are diagnosed with sHLH/MAS (MAS being a form of secondary HLH) and who are presenting an inadequate response to high doses of GCs. These subjects will be enrolled in 2 cohorts as per their background disease. The cohorts are defined as follows:
* Cohort 1: MAS in the context of sJIA and AOSD.
* Cohort 2: MAS in the context of pediatric and adult SLE.
The study has the objectives to investigate the efficacy, safety and tolerability, for 8 weeks, and PK and PD, QoL and immunogenicity in these 2 cohorts for up to 1 year after last dose of of emapalumab.
Macrophage Activation Syndrome (MAS) Secondary Hemophagocytic Lymphohistiocytosis (sHLH) systemic Juvenile Idiopathic Arthritis (sJIA) Adult-onset Still's Disease (AOSD) Systemic Lupus Erythematosus (SLE)
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 41
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Cohort 1 (sJIA and AOSD) and Cohort 2 (SLE) Emapalumab MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) or SLE
- Primary Outcome Measures
Name Time Method Proportion of subjects with complete response (CR) at Week 8 after first administration of emapalumab 8 weeks Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm.
Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm. Clinical signs will be considered as resolved if VAS is below or equal to 1/10.
And
Normalization of laboratory parameters relevant to MAS, as follows:
WBC above LLN platelet count above LLN LDH below 1.5 ULN ALT below 1.5 ULN AST below 1.5 ULN fibrinogen higher than 100 mg/dL ferritin levels decreased by at least 80 % from values at screening or baseline (whichever is higher) or below 2000 ng/ml, whichever is low
- Secondary Outcome Measures
Name Time Method Time to achieve GCs tapering as defined above. At any time in the study, up to 1 year GC tapering as per investigator discretion
Time to first Complete Remission At any time in the study, up to 1 year Time to CR
GCs tapering to a dose below 50% of prednisolone (PDN) equivalent at the time of emapalumab start or to the same (or lower) dose being administered before the occurrence of MAS whichever occurs first At any time in the study, up to 1 year GC tapering as per investigator discretion
Proportion of subjects with overall response as defined by CR or PR At any time in the study, up to 1 year Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm.
Survival time At any time in the study, up to 1 year Time to Survival
GCs tapering to ≤1mg/kg/day of PDN equivalent at any time during the study. At any time in the study, up to 1 year GC tapering as per investigator discretion
MAS recurrence at anytime after achievement of CR At any time after CR, up to 1 year Time to MAS recurrence after CR
Time to first overall response as defined by CR or PR At any time in the study, up to 1 year CR defined as below:
Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm.Withdrawal from the study due to lack of response as per Investigator decision At any time in the study, up to 1 year Time to withdrawal
Trial Locations
- Locations (46)
Universitair Ziekenhuis Leuven
🇧🇪Leuven, Belgium
Children's Hospital of Fudan University
🇨🇳Shanghai, China
UMC Utrecht
🇳🇱Utrecht, Netherlands
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Nationwide Children's Hospital, Abigail Wexner Research Institute
🇺🇸Columbus, Ohio, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
UAB Hospital
🇺🇸Birmingham, Alabama, United States
UCLA Health
🇺🇸Los Angeles, California, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
University of Minnesota Masonic Children's Hospital
🇺🇸Minneapolis, Minnesota, United States
University of Calgary
🇨🇦Calgary, Canada
Alberta Children's Hospital
🇨🇦Calgary, Canada
Hospital for sick children
🇨🇦Toronto, Canada
Centre Hospitalier de l'Université de Montréal
🇨🇦Montréal, Canada
Centre Hospitalier Universitaire Sainte-Justine
🇨🇦Montréal, Canada
Beijing Children's Hospital
🇨🇳Beijing, China
Beijing Friendship Hospital
🇨🇳Beijing, China
Hôpital De La Conception
🇫🇷Marseille, France
Vseobecna Fakultni Nemocnice v Praze
🇨🇿Praha, Czechia
Hôpital Claude Huriez
🇫🇷Lille, France
Fakultní nemocnice Olomouc
🇨🇿Olomouc, Czechia
Hôpital Necker-Enfants Malades
🇫🇷Paris, France
Charité Universitätsmedizin
🇩🇪Berlin, Germany
Hôpital Universitaire Pitié Salpêtrière
🇫🇷Paris, France
Universitätsklinikum Heidelberg
🇩🇪Heidelberg, Germany
IRCCS G. Gaslini
🇮🇹Genova, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
St. Marianna University School of Medicine Hospital
🇯🇵Kawasaki, Japan
IRCCS - Materno-Infantile Burlo Garofolo
🇮🇹Trieste, Italy
Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, Italy
Osaka Medical and Pharmaceutical University Hospital
🇯🇵Takatsuki, Japan
Tokyo Medical and Dental University Hospital
🇯🇵Tokyo, Japan
Yokohama City University Hospital
🇯🇵Yokohama, Japan
Szpital Specjalistyczny im. J. Dietla w Krakowie
🇵🇱Kraków, Poland
Wojewódzki Specjalistyczny Szpital Dziecięcy im. św. Ludwika w Krakowie
🇵🇱Kraków, Poland
Ortopedyczno - Rehabilitacyjny Szpital Kliniczny im. Wiktora Degi Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu
🇵🇱Poznań, Poland
Hospital Sant Joan de Déu
🇪🇸Barcelona, Spain
Hospital Universitario La Paz
🇪🇸La Paz, Spain
Hospital Universitario
🇪🇸La Paz, Spain
Hospital Universitari i Politècnic La Fe
🇪🇸Valencia, Spain
Karolinska Universitetssjukhuset Solna
🇸🇪Stockholm, Sweden
Karolinska Universitetssjukhuset Solna (Ped. Rheum.)
🇸🇪Stockholm, Sweden
Great Ormond Street Hospital
🇬🇧London, United Kingdom
Imperial College Healthcare NHS Trust
🇬🇧London, United Kingdom
University of Florida
🇺🇸Gainesville, Florida, United States