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An open-label, single-arm, active-treatment study to evaluate efficacy and safety of canakinumab (ACZ885) administered for at least 48 weeks in Japanese patients with Adult Onset Still's Disease (AOSD)

Study of Efficacy and Safety of canakinumab (ACZ885) in Patients With Adult Onset Still's Disease (AOSD)

Yamada Hiroyuki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku@novartis.com

Yamada Hiroyuki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku@novartis.com

Not Recruiting

Feb. 26, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. Signed informed consent must be obtained prior to participation in the study. Parent's or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for participants < 20 years of age
2. Japanese male and female participants aged >= 16 years
3. Confirmed diagnosis of AOSD as per Yamaguchi criteria (Yamaguchi M, 1992) with an onset of disease >= 16 years of age. Yamaguchi criteria requires at least five criteria, including two major criteria and no exclusion criteria:
Major criteria
- Fever >= 39 degree lasting 1 week or more
- Arthralgia lasting 2 weeks or more
- Typical skin rash: maculopapular, nonpruritic, salmon-pink rash with concomitant fever spikes
- Leukocytosis >=10,000/mm3 with neutrophil polymorphonuclear proportion >= 80%
Minor criteria
- Pharyngitis or sore throat
- Lymphadenopathy and/or splenomegaly
- Liver enzyme abnormalities
- Negative for RF or antinuclear antibodies
Exclusion criteria
- Infection, especially sepsis and Epstein-Barr viral infection
- Malignant diseases, especially malignant lymphomas
- Inflammatory disease, especially polyarteritis nodosa
4. Active disease at the time of baseline defined as follows
- Fever (body temperature > 38 degree) due to AOSD for at least 1 day within 1 week before baseline
- At least 2 active joints (tender or swollen)
- CRP >= 10 mg/L
5. Participants who have history of inadequate response to more than 2 weeks of corticosteroids equivalent to at least 0.4 mg/kg/day of prednisolone

1. History/evidence of active MAS or disseminated intravascular coagulation (DIC) prior to 6 months of enrollment.
2. With underlying metabolic, renal, hepatic, infectious or gastrointestinal conditions which in the opinion of the investigator compromises the participant and/ or places the participant at unacceptable risk for participation in an immunomodulatory therapy.
3. Clinical evidence of liver disease or liver injury as indicated by abnormal liver function tests at screening such as Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), Gamma-glutamyl transferase (GGT), alkaline phosphatase (ALP)(must not exceed 3 times the upper limit value of the normal range for age), or serum bilirubin (must not exceed twice the upper limit value of the normal range for age).
4. With active or recurrent bacterial, fungal or viral infection at the time of enrollment, including participants with evidence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B and Hepatitis C infection.

Both

Adult Onset Still's Disease (AOSD)

canakinumab administered subcutaneously every 4 weeks

Proportion of participants who achieve adapted ACR 30 response at Week 8

+81-11-706-7061

Dec. 23, 2020

No

none