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A study to evaluate efficacy, safety and tolerability, pharmacokinetics and pharmacodynamics of emapalumab in children and adults with macrophage activation syndrome (MAS)
EU CTIS ID: 2024-516153-52-00
What this study is testing
To demonstrate efficacy of emapalumab in the treatment of patients in: - Cohort 1: Macrophage Activation Syndrome (MAS) in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (AOSD). - Cohort 2: MAS in the context of pediatric and adult systemic lupus erythematosus (SLE)
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Informed consent provided by the patient or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as required by local law. (Run-in phase in all cohorts)
- Cohort 2 (Specific inclusion criteria for Cohort 1 and Cohort 2): a. Confirmed diagnosis of SLE as per Systemic Lupus International Collaborating Clinics (SLICC) 2012 criteria.
- Sexually active female patients of childbearing potential are expected to use highly effective methods of contraception during dosing and for 6 months after last dose of study drug. Highly effective contraception methods include: • Combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation: o Oral. o Intravaginal. o Transdermal. • Progestogen-only hormonal contraception associated with inhibition of ovulation: o Oral. o Injectable. o Implantable. • Intra-uterine device. • Intra-uterine hormone-releasing system. • Bilateral tubal occlusion. • Vasectomised partner. • Sexual abstinence.
- Male and female patients aged between 6 months and 80 years of age at the time of diagnosis of active MAS. (Run-in phase in all cohorts)
- MAS defined as per the criteria defined below for each cohort and requiring treatment with GCs as per standard of care. (Run-in phase in all cohorts)
- Informed consent provided by the patient or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as as required by local law. (Interventional phase in all cohorts)
You likely can't join if
- Primary hemophagocytic lymphohistiocytosis (p-HLH) documented by either the presence of a known causative genetic mutation or abnormal perforin expression or CD107a degranulation assay as described with pHLH or by the presence of family history.
- Evidence of latent tuberculosis.
- History of hypersensitivity or allergy to any component of the study drug.
- Receipt of a Bacillus Calmette-Guerin (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.
- Pregnancy or lactating female patients.
See the full eligibility criteria
Who can join
- Informed consent provided by the patient or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as required by local law. (Run-in phase in all cohorts)
- Cohort 2 (Specific inclusion criteria for Cohort 1 and Cohort 2): a. Confirmed diagnosis of SLE as per Systemic Lupus International Collaborating Clinics (SLICC) 2012 criteria.
- Sexually active female patients of childbearing potential are expected to use highly effective methods of contraception during dosing and for 6 months after last dose of study drug. Highly effective contraception methods include: • Combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation: o Oral. o Intravaginal. o Transdermal. • Progestogen-only hormonal contraception associated with inhibition of ovulation: o Oral. o Injectable. o Implantable. • Intra-uterine device. • Intra-uterine hormone-releasing system. • Bilateral tubal occlusion. • Vasectomised partner. • Sexual abstinence.
- Male and female patients aged between 6 months and 80 years of age at the time of diagnosis of active MAS. (Run-in phase in all cohorts)
- MAS defined as per the criteria defined below for each cohort and requiring treatment with GCs as per standard of care. (Run-in phase in all cohorts)
- Informed consent provided by the patient or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as as required by local law. (Interventional phase in all cohorts)
- Male and female patients aged between 6 months and 80 years of age at the time of diagnosis of active MAS. (Interventional phase in all cohorts)
- Patients who have shown an inadequate response to high dose intravenous (i.v.) GCs administered for at least 3 days according to local standard clinical practice, including but not limited to pulses of 30 mg/kg methylprednisolone (mPDN) on 3 consecutive days. High i.v. GCs dose is recommended not to be lower than 2 mg/kg/ day PDN equivalent (or at least 60 mg/day in pediatric patients of 30 kg or more, and at least 1 g/day in adult MAS patients). In case of rapid worsening of the patient's condition and/or laboratory parameters, as per Investigator judgment, inclusion may occur within less than 3 days from starting high dose GCs. (Interventional phase in all cohorts)
- Diagnosis of active MAS confirmed by the treating rheumatologist, having ascertained the followings (Interventional phase in all cohorts): a. Febrile patients presenting with ferritin > 684 ng/mL. b. and any 2 of: i. Platelet count ≤ 181 x109/L ii. Aspartate aminotransferase (AST)-level > 48 U/L iii. Triglycerides > 156 mg/dL iv. Fibrinogen level ≤ 360 mg/dL
- Female patients of child-bearing potential (sexually or non-sexually active). Female patients who are sexually active must be willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug. (Interventional phase in all cohorts)
- Cohort 1 (Specific inclusion criteria for Cohort 1 and Cohort 2): a. Confirmed sJIA diagnosis. For patients presenting with MAS in the context of the onset of sJIA, high presumption of sJIA will suffice for eligibility. b. Confirmed diagnosis of AOSD as per Yamaguchi criteria (Yamaguchi et al. 1992)
What rules you out
- Primary hemophagocytic lymphohistiocytosis (p-HLH) documented by either the presence of a known causative genetic mutation or abnormal perforin expression or CD107a degranulation assay as described with pHLH or by the presence of family history.
- Evidence of latent tuberculosis.
- History of hypersensitivity or allergy to any component of the study drug.
- Receipt of a Bacillus Calmette-Guerin (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.
- Pregnancy or lactating female patients.
- Confirmed malignancy. Note: patients with a suspected malignancy should have mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable, to rule out malignancy.
- Treatment with canakinumab, Janus kinase (JAK) inhibitors, Tumour necorsis factor (TNF) inhibitors and tocilizumab at the time of emapalumab initiation.
- Ongoing treatment with anakinra at a dose above 4 mg/kg/ day at time of emapalumab initiation.
- Patients treated with etoposide for MAS in the last 1 month.
- Presence of any medical or psychological condition or laboratory result that in the opinion of the Investigator can interfere with the patient's ability to comply with the protocol requirements or makes the patient not appropriate for inclusion to the study and treatment with emapalumab.
- Foreseeable inability to cooperate with given instructions or study procedures.
- Clinically active mycobacteria (typical and atypical), Histoplasma Capsulatum, or Salmonella infections.
- Evidence of leishmania infections.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United States
- China
- Japan
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; United States; China; Japan; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.