A pilot, open-label, single arm, multicenter study to evaluate safety, tolerability, pharmacokinetics and efficacy of intravenous administrations of emapalumab, an anti-interferon gamma (anti-IFNγ) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still*s Disease (AOSD) developing Macrophage Activation Syndrome/ secondary HLH (MAS/sHLH)
- Conditions
- complication of Juvenile Idiopathic Arthritis [sJIA] or Adult-onset Stills Disease (AOSD)Macrophage Activation Syndrome1000381610023213
- Registration Number
- NL-OMON50760
- Lead Sponsor
- Swedish Orphan Biovitrum AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 3
1. Patients of both genders 2. For sJIA patients: confirmed sJIA diagnosis. For
patients presenting with MAS in the context of the onset of sJIA high
presumption of sJIA (as per Appendix A) will suffice for eligibility.AOSD
patients: confirmed AOSD diagnosis as per Yamaguchi criteria (Appendix E) 3.
Diagnosis of active MAS confirmed by the treating rheumatologist, having
ascertained the followings:
Febrile patient presenting with:
- Ferritin > 684 ng/mL
and any two of:
- Platelet count <= 181 x10^9/L
- AST levels > 48 U/L
- Triglycerides > 156 mg/dL
- Fibrinogen levels <= 360 mg/dL.
(see Appendix B), 4 Patient presenting an inadequate response to high dose i.v.
glucocorticoid treatment administered for at least 3 days as per local standard
of care (including but not limited to pulses of 30 mg/kg methylprednisolone
(mPDN) on 3 consecutive days).
High i.v. glucocorticoid dose should not be lower than 2 mg/kg/ day of PDN
equivalent in 2 divided doses, (or at least 60 mg/day in patients of 30 kg or
more) In case of rapid worsening of the patient*s condition and/or lab
parameters, inclusion may occur within less than 3 days from starting high dose
i.v. glucocorticoids. 5.Tocilizumab, TNF inhibitors and canakinumab, if
administered, have to be discontinued before emapalumab initiation. 6. Informed
consent provided 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. 7. Having received guidance on
contraception for both male and female patients sexually active and having
reached puberty:
Females of child-bearing potential require use of highly effective
contraceptive measures (failure rate of less than 1% per year) from screening
until 6 months after receiving last dose of the study drug.
Highly effective contraceptive measures include:
o Sexual abstinence
o Hormonal contraceptives: combination or progesterone only
o Intrauterine methods: intrauterine devices or systems
o Bilateral tubal occlusion
o Vasectomised partner
Males with partners(s) of child-bearing potential must agree to take
appropriate precautions (such as sexual abstinence, barrier contraception,
vasectomy) to avoid fathering a child from screening until 6 months after
receiving last dose of the study drug.
1. Diagnosis of suspected or confirmed primary HLH or HLH consequent to a
neoplastic disease. 2.Active mycobacteria (typical and atypical), Histoplasma
Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections., 3.
Clinical suspicion of latent tuberculosis., 4. Positive serology for HIV
antibodies., 5. Presence of malignancy., 6. Patients who have another
concomitant disease or malformation severely affecting the cardiovascular,
pulmonary, CNS, liver or renal function that in the opinion of the Investigator
may significantly affect likelihood to respond to treatment and/or assessment
of emapalumab safety., 7. History of hypersensitivity or allergy to any
component of the study drug., 8. Receipt of a BCG vaccine within 12 weeks prior
to screening., 9. Receipt of live or attenuated live vaccines (other than BCG)
within 6 weeks prior to screening., 10. Pregnant or lactating female patients.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method