Clinical Trials List
Protocol NumberWN42349
NCT Number(ClinicalTrials.gov Identfier)NCT04660539
Completed
2020-01-01 - 2025-12-31
Phase III
Recruiting4
A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)
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Trial Applicant
Chugai Pharma Taiwan Ltd.
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Sponsor
Hoffmann-La Roche
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Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
Co-Principal Investigator
- Yi-Chu Liao Division of Neurology
- Yi-Chun Lee Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Chung-Hsiang Liu Division of Neurology
- Yu-Wan Yang Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Thy-Sheng Lin Division of Neurology
- Han-Wei Huang Division of Neurology
- Yuan-Ting Sun Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- WEN-CHIN WENG Division of Neurology
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Objectives
This study will evaluate the long-term safety, efficacy, and pharmacokinetics/
pharmacodynamics of satralizumab and will provide continued treatment with
satralizumab to patients with NMOSD who completed the OLE periods of the Phase III
Studies BN40898 and BN40900. Specific objectives and corresponding endpoints for
the study are outlined below.
2.1 SAFETY OBJECTIVE
The primary safety objective for this study is:
To evaluate the long-term safety of satralizumab in patients with NMOSD
The secondary safety objective for this study is:
To further evaluate the risks of serious infections and hepatotoxicity in patients with
NMOSD who are treated with satralizumab
Safety outcome measures:
Incidence and severity of adverse events (AE), adverse events of special interest
(AESI), serious AEs (SAE), and selected AEs
Vital signs (temperature, blood pressure, and pulse rate), clinical laboratory tests
(hematology, chemistry, and urinalysis), 12-lead electrocardiograms (ECG), and
suicidality (Columbia-Suicide Severity Rating Scale [C-SSRS])
2.2 EFFICACY OBJECTIVES
The efficacy objective for this study is to evaluate long-term efficacy of satralizumab on
the basis of the following endpoints:
Time to first relapse (TFR) and proportion of patients who are relapse-free
Annualized relapse rate (ARR)
Change in Expanded Disability Status Scale (EDSS) score
Time to EDSS worsening and proportion of patients without EDSS worsening
Change in visual acuity
Further information on the efficacy endpoints is provided in Section 6.5.
2.3 PHARMACODYNAMIC OBJECTIVE
The pharmacodynamics (PD) objective for this study is to further characterize the target
engagement in response to satralizumab treatment on the basis of the assessment of
IL-6, soluble IL-6R (sIL-6R) and C-reactive protein (CRP).
2.4 PHARMACOKINETIC OBJECTIVES
The pharmacokinetic (PK) objective for this study is to further characterize the
satralizumab PK profile on the basis of the following endpoint:
Serum concentration of satralizumab at specified timepoints
The exploratory PK objectives for this study are as follows:
To evaluate potential relationships between drug exposure and the efficacy and
safety of satralizumab on the basis of the following endpoints:
– Relationship between serum concentration or PK parameters for satralizumab
and efficacy endpoints
– Relationship between serum concentration or PK parameters for satralizumab
and safety endpoints
To evaluate potential relationships between selected covariates and exposure to
satralizumab on the basis of the following endpoint:
– Relationship between selected covariates and serum concentration or PK
parameters for satralizumab
2.5 IMMUNOGENICITY OBJECTIVES
The immunogenicity objective for this study is to evaluate the immune response to
satralizumab on the basis of the following endpoint:
Incidence of anti-drug antibodies (ADAs) from the first dose of satralizumab in
Studies BN40898 or BN40900 (parent studies)
The exploratory immunogenicity objective for this study is to evaluate potential effects of
ADAs on the basis of the following endpoint:
Relationship between ADA status and efficacy, safety, and PK endpoints
2.6 BIOMARKER OBJECTIVE
The exploratory biomarker objective for this study is to identify biomarkers that can
provide evidence of satralizumab activity (i.e., pharmacodynamic biomarkers), or can
increase the knowledge and understanding of disease biology and/or related pathways,
on the basis of the following endpoint:
Relationship between biomarkers in blood (listed in Section 4.5) and efficacy, safety,
PK, immunogenicity, or other biomarker endpoints
2.7 HEALTH STATUS UTILITY OBJECTIVE
The exploratory health status utility objective for this study is to evaluate health status
utility scores of patients treated with satralizumab on the basis of the following endpoint:
Change in EuroQol 5-Dimension Questionnaire (3-level version; EQ-5D-3L) indexbased and Visual Analogue Scale (VAS) scores
Test Drug
ENSPRYNG 120mg for SC Injection
Active Ingredient
Satralizumab
Dosage Form
Prefilled syringe
Dosage
120mg/ml
Endpoints
Primary Outcome Measures :
Percentage of Participants with Adverse Events (AE) AEs of Special Interest (AESI), Serious AEs (SAE), and Selected AEs [ Time Frame: Up to 39 Months ]
Columbia-Suicide Severity Rating Scale (C-SSRS) Scores [ Time Frame: Up to 39 Months ]
Secondary Outcome Measures :
Percentage of Participants with Serious Infections and Hepatotoxicity [ Time Frame: Up to 39 Months ]
Participants with NMOSD who are treated with satralizumab
Time to First Relapse (TFR) [ Time Frame: Up to 39 Months ]
Percentage of Relapse-Free Participants [ Time Frame: Up to 39 Months ]
Annualized Relapse Rate (ARR) [ Time Frame: Up to 39 Months ]
Change in Expanded Disability Status Scale (EDSS) Score [ Time Frame: Up to 39 Months ]
Time to EDSS Worsening [ Time Frame: Up to 39 Months ]
Percentage of Participants without EDSS Worsening [ Time Frame: Up to 39 Months ]
Change in Visual Acuity Assessed by a Snellen 20-Foot Wall Chart [ Time Frame: Up to 39 Months ]
The test will be performed monocularly and participants may use their habitual distance glasses or contact lenses
Concentrations of Interleukin-6 (IL-6) and Soluble IL-6 Receptor (sIL-6R) in Blood [ Time Frame: Up to 39 Months ]
Measured in picogram per milliliter (pg/mL)
Concentration of C-Reactive Protein (CRP) in Blood [ Time Frame: Up to 39 Months ]
Measured in milligram per liter (mg/L)
Serum Concentration of Satralizumab at Specified Timepoints [ Time Frame: Up to 39 Months ]
Percentage of Participants with Anti-Drug Antibodies (ADAs) from the First Dose of Satralizumab in Studies BN40898 or BN40900 (parent studies) [ Time Frame: Up to 39 Months ]
Percentage of Participants with ADA to Satralizumab [ Time Frame: Up to 39 Months ]
Percentage of Participants with Adverse Events (AE) AEs of Special Interest (AESI), Serious AEs (SAE), and Selected AEs [ Time Frame: Up to 39 Months ]
Columbia-Suicide Severity Rating Scale (C-SSRS) Scores [ Time Frame: Up to 39 Months ]
Secondary Outcome Measures :
Percentage of Participants with Serious Infections and Hepatotoxicity [ Time Frame: Up to 39 Months ]
Participants with NMOSD who are treated with satralizumab
Time to First Relapse (TFR) [ Time Frame: Up to 39 Months ]
Percentage of Relapse-Free Participants [ Time Frame: Up to 39 Months ]
Annualized Relapse Rate (ARR) [ Time Frame: Up to 39 Months ]
Change in Expanded Disability Status Scale (EDSS) Score [ Time Frame: Up to 39 Months ]
Time to EDSS Worsening [ Time Frame: Up to 39 Months ]
Percentage of Participants without EDSS Worsening [ Time Frame: Up to 39 Months ]
Change in Visual Acuity Assessed by a Snellen 20-Foot Wall Chart [ Time Frame: Up to 39 Months ]
The test will be performed monocularly and participants may use their habitual distance glasses or contact lenses
Concentrations of Interleukin-6 (IL-6) and Soluble IL-6 Receptor (sIL-6R) in Blood [ Time Frame: Up to 39 Months ]
Measured in picogram per milliliter (pg/mL)
Concentration of C-Reactive Protein (CRP) in Blood [ Time Frame: Up to 39 Months ]
Measured in milligram per liter (mg/L)
Serum Concentration of Satralizumab at Specified Timepoints [ Time Frame: Up to 39 Months ]
Percentage of Participants with Anti-Drug Antibodies (ADAs) from the First Dose of Satralizumab in Studies BN40898 or BN40900 (parent studies) [ Time Frame: Up to 39 Months ]
Percentage of Participants with ADA to Satralizumab [ Time Frame: Up to 39 Months ]
Inclution Criteria
Participated in Study BN40898 or Study BN40900 with satralizumab in NMOSD, are
on ongoing satralizumab treatment and were AQP4-IgG seropositive at screening in
these studies. Patients with NMOSD who were AQP4-IgG seronegative at
screening in Study BN40898 or Study BN40900 can be enrolled if the investigator
considers the continued treatment with satralizumab to be beneficial for the patient.
Signed Informed Consent Form (ICF)
Ability to comply with the study protocol
For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use adequate contraception during the treatment
period and for 3 months after the final dose of satralizumab.
A woman is considered to be of childbearing potential if she is postmenarchal,
has not reached a postmenopausal state (12 continuous months of
amenorrhea with no identified cause other than menopause), and is not
permanently infertile due to surgery (i.e., removal of ovaries, fallopian tubes,
and/or uterus) or another cause as determined by the investigator
(e.g., Müllerian agenesis). The definition of childbearing potential may be
adapted for alignment with local guidelines or regulations.
The following are examples of adequate contraceptive methods: bilateral tubal
ligation; male sterilization; hormonal contraceptives; hormone-releasing
intrauterine devices; copper intrauterine devices; male or female condom with
or without spermicide; and cap, diaphragm, or sponge with spermicide.
The reliability of sexual abstinence should be evaluated in relation to the
duration of the clinical trial and the preferred and usual lifestyle of the patient.
Periodic abstinence (e.g., calendar, ovulation, symptothermal, or postovulation
methods) and withdrawal are not adequate methods of contraception.
If required per local guidelines or regulations, locally recognized adequate methods of contraception and information about the reliability of abstinence will
be described in the local ICF.
on ongoing satralizumab treatment and were AQP4-IgG seropositive at screening in
these studies. Patients with NMOSD who were AQP4-IgG seronegative at
screening in Study BN40898 or Study BN40900 can be enrolled if the investigator
considers the continued treatment with satralizumab to be beneficial for the patient.
Signed Informed Consent Form (ICF)
Ability to comply with the study protocol
For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use adequate contraception during the treatment
period and for 3 months after the final dose of satralizumab.
A woman is considered to be of childbearing potential if she is postmenarchal,
has not reached a postmenopausal state (12 continuous months of
amenorrhea with no identified cause other than menopause), and is not
permanently infertile due to surgery (i.e., removal of ovaries, fallopian tubes,
and/or uterus) or another cause as determined by the investigator
(e.g., Müllerian agenesis). The definition of childbearing potential may be
adapted for alignment with local guidelines or regulations.
The following are examples of adequate contraceptive methods: bilateral tubal
ligation; male sterilization; hormonal contraceptives; hormone-releasing
intrauterine devices; copper intrauterine devices; male or female condom with
or without spermicide; and cap, diaphragm, or sponge with spermicide.
The reliability of sexual abstinence should be evaluated in relation to the
duration of the clinical trial and the preferred and usual lifestyle of the patient.
Periodic abstinence (e.g., calendar, ovulation, symptothermal, or postovulation
methods) and withdrawal are not adequate methods of contraception.
If required per local guidelines or regulations, locally recognized adequate methods of contraception and information about the reliability of abstinence will
be described in the local ICF.
Exclusion Criteria
Use of prohibited medication (see Section 4.4.2).
Pregnant or breastfeeding, or intending to become pregnant during the study or
within 3 months after the final dose of study drug. Women of childbearing potential
must have a negative urine pregnancy test result on the baseline visit prior to
initiation of study drug.
Evidence of any serious uncontrolled concomitant diseases that may preclude
patient participation, such as:
other nervous system disease, cardiovascular disease,
hematologic/hematopoiesis disease, respiratory disease, muscular disease,
endocrine disease, renal/urologic disease, digestive system disease, congenital
or acquired severe immunodeficiency.
Known active infection that requires delaying the next satralizumab dose at the time
of enrollment
In case of an active infection, the patient should remain in the parent study, as
governed by that protocol, and may enroll in this study once the active infection is
controlled.
NMOSD relapse at the time of enrollmentb
In case of a relapse, the patient should remain in the parent study, as governed
by that protocol, and may enroll in this study once the patient is stable.
Laboratory abnormalities at the last assessment in Study BN40898 or
Study BN40900 that preclude re-treatment with satralizumab (see Section 5.1.1)
If a patient does not meet the criteria to restart treatment with satralizumab
based on laboratory assessments, the patient should remain in the parent study and
the baseline visit should be delayed. The last assessment before enrollment must
meet the re-treatment criteria.
Pregnant or breastfeeding, or intending to become pregnant during the study or
within 3 months after the final dose of study drug. Women of childbearing potential
must have a negative urine pregnancy test result on the baseline visit prior to
initiation of study drug.
Evidence of any serious uncontrolled concomitant diseases that may preclude
patient participation, such as:
other nervous system disease, cardiovascular disease,
hematologic/hematopoiesis disease, respiratory disease, muscular disease,
endocrine disease, renal/urologic disease, digestive system disease, congenital
or acquired severe immunodeficiency.
Known active infection that requires delaying the next satralizumab dose at the time
of enrollment
In case of an active infection, the patient should remain in the parent study, as
governed by that protocol, and may enroll in this study once the active infection is
controlled.
NMOSD relapse at the time of enrollmentb
In case of a relapse, the patient should remain in the parent study, as governed
by that protocol, and may enroll in this study once the patient is stable.
Laboratory abnormalities at the last assessment in Study BN40898 or
Study BN40900 that preclude re-treatment with satralizumab (see Section 5.1.1)
If a patient does not meet the criteria to restart treatment with satralizumab
based on laboratory assessments, the patient should remain in the parent study and
the baseline visit should be delayed. The last assessment before enrollment must
meet the re-treatment criteria.
The Estimated Number of Participants
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Taiwan
13 participants
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Global
127 participants
