Clinical pharmacology study of satralizumab in patients with anti-AQP4 antibody-positive neuromyelitis optica spectrum disorder (AQP4-IgG + NMOSD)

JRCT ID: jRCT2051230006

Registered date:10/04/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Neuromyelitis optica spectrum disorders(NMOSD)
• Date of first enrollment: 09/02/2024
• Target sample size: 3
• Study type: Interventional
• Intervention(s): satralizumab: single intravenous administration at protocol-specified dose

Outcome(s)

Primary Outcome

Phamacodynamics
Calculate summary statistics for IL-6 and soluble Interleukin-6 Receptor (sIL-6R) in serum and CSF
The calculated CSF/serum ratios and summary statistics (mean, standard deviation, coefficient of variation, median, minimum, and maximum) for IL-6, sIL-6R, and albumin concentrations are summarized in the table

Secondary Outcome

Safety
It will be performed on all subjects who received the study drug. Individual data for adverse events, serious adverse events, AESI, laboratory tests (hematology, blood biochemistry, coagulation, urinalysis, immunoserology), 12-lead ECG, vital signs (temperature, systolic blood pressure, diastolic blood pressure, heart rate), and C-SSRS will be listed. Summarize (descriptive statistics) if necessary.
Phamacokinetics
It will be performed on subjects who received the study drug and had at least one measurement of drug concentration in serum or CSF. The individual and mean serum and CSF satralizumab concentrations over time will be summarized in tables and graphs
Phamacodynamics
It will be performed on subjects who received the study drug and had at least one pharmacodynamic meaasurement taken.Summary statistics of anti-AQP4 antibody concentrations in serum and CSF will be calculated.
Other
It will be performed on subjects who have undergone at least one ADA evaluation before and after treatment with the study drug. List individual serum anti-satralizumab antibody data. Summary (descriptive statistics) if necessary.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 74age old
• Gender: Both

Include criteria

At least 18 years old and less than 75 years old at the time of informed consent
Ability to understand and willingness to sign a written informed consent document and the study protocol
Confirmed diagnosis of developed new neurological symptoms due to NMOSD attacks
Meet the following diagnostic criteria for AQP4-IgG+NMOSD
1- At least one core clinical symptom*
2- Positive test for Anti-AQP4 antibody (in the case of recurrent patients, typical clinical symptoms that meet the diagnostic criteria and MRI findings during previous attacks allow the determination of anti-AQP4 antibody positivity based on previous anti-AQP4 antibody positivity)
3- Exclusion of alternative diagnoses
*Main Clinical Symptoms
- Optic neuritis
- Acute myelitis
- Area postrema syndrome : episode of otherwise unexplained hiccups or neusea and vomiting
- Acute brainstem syndrome
- Symptomatic narcolepsy or acute diencephalic clinical syndrome with NMOSD-typical diencephalic MRI lesions
- Symptomatic cerebral syndrome with NMOSD-typical brain MRI lesions

Exclude criteria

Patients previously treated with IL-6 inhibitors, alemtuzumab, total-body radiation, or bone marrow transplant
Patients treated with CD20 inhibitors within 6 months prior to screening and B-cell fraction < 2.9%
Patients treated with eculizumab, belimumab, interferon (IFN), natalizumab, glatiramer acetate, fingolimod, teriflunomide, or dimethyl fumarate within 6 months before screening
Patients treated with CD4 inhibitors, cladribine, or mitoxantrone within 2 years prior to screening
Pregnant or breastfeeding women
Surgical procedures (excluding minor surgery) within 4 weeks prior to screening
Clinical evidence of other demyelinating diseases or progressive multifocal leukoencephalopathy
Patients with uncontrolled serious comorbidities that may preclude participation in the study
Active infection within 4 weeks prior to screening
Developed an infection requiring hospitalization or intravenous (IV) treatment within 4 weeks prior to screening
Active chronic hepatitis B or C
Bladder and rectal problems that are associated with urinary retention or urinary incontinence
History of diverticulitis, which, in the judgment of the physician, may increase the risk of complications such as perforation of the lower gastrointestinal tract
Patients with evidence of active tuberculosis (excluding patients receiving chemoprophylaxis for latent tuberculosis infection)
Patients with active interstitial lung disease
Receipt of live or live attenuated vaccine within 6 weeks prior to screening

Related Information

• Primary Sponsor: Okuno Tatsusada