A STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SATRALIZUMAB IN PATIENTS WITH NMDAR OR LGI1ENCEPHALITIS

JRCT ID: jRCT2011220016

Registered date:26/08/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Autoimmune Encephalitis
• Date of first enrollment: 27/09/2022
• Target sample size: 152
• Countries of recruitment: USA,Japan,Argentina,Japan,Austria,Japan,Netherlands,Japan,Italy,Japan,Czech Republic,Japan,South Korea,Japan,China,Japan,France,Japan,Poland,Japan,Brazil,Japan,Denmark,Japan,Ghana,Japan,Taiwan,Japan
• Study type: Interventional
• Intervention(s): Satralizumab: SC injection at protocol-specified dose every 4 weeks (Q4W)

Outcome(s)

Primary Outcome

Safety, Efficacy
Part1 (Primary Treatment Period) : To evaluate the efficacy of satralizumab compared with placebo
- Proportion of participants with mRS score improvement >= 1 from baseline and no use of rescue
therapy at Week 24

Part 2 (Extension Period) : To evaluate the long-term safety and tolerability of satralizumab
- Incidence, seriousness, and severity of adverse events
- Change from satralizumab baseline in targeted vital signs, clinical laboratory test results, ECG results, weight, height (<18 years only), and C-SSRS

Secondary Outcome

Safety, Efficacy, Phamacokinetics, Phamacodynamics, Other
To evaluate the efficacy of satralizumab compared with placebo
- Time to mRS score improvement >=1 from baseline without use of rescue therapy
- Time to rescue therapy
- Time to seizure freedom or cessation of status epilepticus without use of rescue therapy
- Change in CASE score from baseline at Week 24
- MOCA total score, RAVLT score (LGI1 AIE cohort) and mRS score (NMDAR AIE cohort) at Week 24

To evaluate the safety of satralizumab compared with placebo

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

For all participants
1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the Informed Consent Form and in this protocol
2. Reasonable exclusion of tumor or malignancy before baseline visit (randomization)
3. Onset of AIE symptoms <= 9 months before randomization
4. Meet the definition of "New Onset" or "Incomplete Responder" AIE
5. Has a stable (for at least 24 hours) Modified Rankin Scale (mRS) score >= 2, measured at baseline
6. For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab or placebo

For the NMDAR AIE Cohort
1. Age>=12 years at the time of signing Informed Consent Form
2. Diagnosis of probable or definite NMDAR encephalitis

For the LGI1 AIE Cohort
1. Age>=18 years at the time of signing Informed Consent Form
2. Diagnosis of LGI1 encephalitis

Exclude criteria

1. Any untreated teratoma or thymoma at baseline visit (randomization)
2. History of carcinoma or malignancy, unless deemed cured by adequate treatment with no evidence of recurrence for >=5 years before screening
3. For patients with NMDAR AIE, history of negative anti-NMDAR antibody in cerebrospinal fluid (CSF) using a cell-based assay within 9 months of symptom onset
4. Historically known positivity to an intracellular antigen with high cancer association or GAD-65
5. Historically known positivity to any cell surface neuronal antibodies other than NMDAR and LGI1
6. Confirmed paraneoplastic encephalitis
7. Confirmed central or peripheral nervous system demyelinating disease
8. Alternative causes of associated symptoms
9. History of herpes simplex virus encephalitis in the previous 24 weeks

Related Information

• Primary Sponsor: James Overell
• Secondary ID(s): NCT05503264,2021-002395-39