Clinical Trials List
Protocol NumberSA-307JG
2014-02-01 - 2021-12-31
Phase III
Terminated5
ICD-10G36.0
Neuromyelitis optica [Devic]
ICD-9341.0
Neuromyelitis optica
A MULTICENTER, RANDOMIZED, ADDITION TO BASELINE TREATMENT, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SA237 IN PATIENTS WITH NEUROMYELITIS OPTICA (NMO) AND NMO SPECTRUM DISORDER (NMOSD)
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Trial Applicant
Chugai Pharma Taiwan Ltd.
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Sponsor
Chugai Pharmaceutical Co., Ltd.
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- 蔡清標 Division of Neurology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- Wei-Chi WU Division of Ophthalmology
- 王超然 Division of Radiology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- Chou-Ching Lin Division of Neurology
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- Yu-Wan Yang Division of Neurology
- Chung-Hsiang Liu Division of Neurology
The Actual Total Number of Participants Enrolled
5 Terminated
Condition/Disease
NEUROMYELITIS OPTICA (NMO) AND NMO SPECTRUM DISORDER (NMOSD)
Objectives
Efficacy Objective
The efficacy objective for this study is as follows:
To evaluate the efficacy of SA237 compared with placebo in patients with neuromyelitis
optica (NMO) and NMO spectrum disorder (NMOSD).
Safety Objective
The safety objective for this study is as follows:
To evaluate the safety of SA237 compared with placebo in patients with NMO and
NMOSD.
Exploratory Objectives
The exploratory objectives for this study are as follows:
To examine the pharmacokinetics (PK), pharmacodynamics (PD) and immunogenicity of
SA237.
Test Drug
SA237
Active Ingredient
SA237
Dosage Form
solution for injection (subcutaneous)
Dosage
120mg
Endpoints
Primary Endpoint
1. Time to first protocol-defined relapse (TFR) in the double-blind period.
Secondary Endpoints
1. Change in Visual Analogue Scale (VAS) for pain
2. Change in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue
3. Change in Short Form generic health survey (SF-36)
4. Change in EQ-5D
5. The proportion of relapse-free patients
6. ARR
7. Change in modified Rankin Scale (mRS)
8. Change in Zarit Burden Interview (ZBI)
9. Change in EDSS
10.Change in visual acuity (Snellen chart).
1. Time to first protocol-defined relapse (TFR) in the double-blind period.
Secondary Endpoints
1. Change in Visual Analogue Scale (VAS) for pain
2. Change in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue
3. Change in Short Form generic health survey (SF-36)
4. Change in EQ-5D
5. The proportion of relapse-free patients
6. ARR
7. Change in modified Rankin Scale (mRS)
8. Change in Zarit Burden Interview (ZBI)
9. Change in EDSS
10.Change in visual acuity (Snellen chart).
Inclution Criteria
1. Patients must be diagnosed as having either:
a. NMO as defined by 2006 criteria*, or
b. NMOSD as defined by 2007 criteria** who do not meet above NMO criteria
and have anti-aquaporin 4 antibody (AQP4Ab) seropositive status at
screening.
2. Clinical evidence of at least 2 documented relapses (including first attack) in the
last 2 years prior to screening, at least one of which has occurred in the 12 months
prior to screening.
3. Expanded Disability Status Scale (EDSS) score from 0 to 6.5 inclusive at
screening.
4. Age 18 to 74 years, inclusive at the time of informed consent.
5. One of the following baseline treatments for relapse prevention must be at stable
dose as a monotherapy for 8 weeks prior to baseline:
a. Azathioprine.
b. Mycophenolate mofetil.
c. Oral corticosteroids.
6. Ability and willingness to provide written informed consent and to comply with
the requirements of the protocol.
*According to Wingerchuk et al. 2006, a diagnosis of NMO includes:
I. Optic neuritis
II. Acute myelitis
III. At least two of three supportive criteria:
• Contiguous spinal cord lesion identified on a magnetic resonance image (MRI)
scan extending over 3 vertebral segments
• Brain MRI not meeting diagnostic criteria for multiple sclerosis (MS)
• NMO-immunoglobulin G (IgG) seropositive status
**According to Wingerchuk et al. 2007, a diagnosis of NMOSD includes:
I. Neuromyelitis optica
II. Limited forms of NMO:
• Idiopathic single or recurrent events of longitudinally extensive myelitis
(≥3 vertebral segment spinal cord MRI lesion)
• Optic neuritis: recurrent or simultaneous bilateral
III. Asian optic-spinal MS
IV. Optic neuritis or longitudinally extensive myelitis associated with systemic
autoimmune disease
V. Optic neuritis or myelitis associated with NMO-typical brain lesions
(hypothalamic, corpus callosal, periventricular, brain stem).
a. NMO as defined by 2006 criteria*, or
b. NMOSD as defined by 2007 criteria** who do not meet above NMO criteria
and have anti-aquaporin 4 antibody (AQP4Ab) seropositive status at
screening.
2. Clinical evidence of at least 2 documented relapses (including first attack) in the
last 2 years prior to screening, at least one of which has occurred in the 12 months
prior to screening.
3. Expanded Disability Status Scale (EDSS) score from 0 to 6.5 inclusive at
screening.
4. Age 18 to 74 years, inclusive at the time of informed consent.
5. One of the following baseline treatments for relapse prevention must be at stable
dose as a monotherapy for 8 weeks prior to baseline:
a. Azathioprine.
b. Mycophenolate mofetil.
c. Oral corticosteroids.
6. Ability and willingness to provide written informed consent and to comply with
the requirements of the protocol.
*According to Wingerchuk et al. 2006, a diagnosis of NMO includes:
I. Optic neuritis
II. Acute myelitis
III. At least two of three supportive criteria:
• Contiguous spinal cord lesion identified on a magnetic resonance image (MRI)
scan extending over 3 vertebral segments
• Brain MRI not meeting diagnostic criteria for multiple sclerosis (MS)
• NMO-immunoglobulin G (IgG) seropositive status
**According to Wingerchuk et al. 2007, a diagnosis of NMOSD includes:
I. Neuromyelitis optica
II. Limited forms of NMO:
• Idiopathic single or recurrent events of longitudinally extensive myelitis
(≥3 vertebral segment spinal cord MRI lesion)
• Optic neuritis: recurrent or simultaneous bilateral
III. Asian optic-spinal MS
IV. Optic neuritis or longitudinally extensive myelitis associated with systemic
autoimmune disease
V. Optic neuritis or myelitis associated with NMO-typical brain lesions
(hypothalamic, corpus callosal, periventricular, brain stem).
Exclusion Criteria
Exclusion criteria related to previous or concomitant therapy:
1. Previous treatment with interleukin 6 (IL-6) inhibitory therapy (e.g. tocilizumab).
2. Any previous treatment with anti-cluster of differentiation 20 (anti-CD20) treatment,
eculizumab, belimumab, interferon, natalizumab, glatiramer acetate, fingolimod,
teriflunomide, dimethyl fumarate within 6 months prior to baseline.
3. Any previous treatment with alemtuzumab, anti-CD4, cladribine, total body
irradiation, bone marrow transplantation within 2 years prior to baseline.
4. Treatment with any investigational agent within 3 months prior to baseline.
Exclusions for general safety:
5. Pregnancy or lactation.
6. For patients of reproductive potential, a positive result from a serum pregnancy test
at screening, or not willing to use reliable means of contraception.
7. Any surgical procedure (except for minor surgeries) within 4 weeks prior to baseline.
8. Evidence of other demyelinating disease or progressive multifocal
leukoencephalopathy (PML).
9. Evidence of 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.
10. Known active infection (excluding fungal infections of nail beds or caries dentium)
within 4 weeks prior to baseline.
11. Evidence of chronic active hepatitis B or C.
12. History of drug or alcohol abuse.
13. History of diverticulitis that, in the Investigator’s opinion, may preclude patient
participation.
14. Evidence of active tuberculosis (excluding patients receiving chemoprophylaxis for
latent tuberculosis infection).
15. Evidence of active interstitial lung disease.
16. Receipt of any live or live attenuated vaccine within 6 weeks prior to baseline.
17. History of malignancy within the last 5 years, including solid tumors, hematologic
malignancies and in situ carcinoma (except basal cell and squamous cell carcinomas
of the skin, or in situ carcinoma of the cervix uteri that have been completely excised
and cured).
18. History of severe allergic reaction to a biologic agent (e.g. shock, anaphylactic
reactions).
Laboratory exclusion criteria (at screening):
19. Following laboratory abnormalities at screening.
a. White blood cells (WBC) <4.0 x103 /μL
b. Absolute neutrophil count (ANC) <2.0 x 103
/μL
c. Absolute lymphocyte count <0.5 x 103 /μL
d. Platelet count <10 x 104
/μL
e. Aspartate aminotransferase (AST) or alanine aminotranferase (ALT) >1.5 times
the upper limit of normal (ULN).
1. Previous treatment with interleukin 6 (IL-6) inhibitory therapy (e.g. tocilizumab).
2. Any previous treatment with anti-cluster of differentiation 20 (anti-CD20) treatment,
eculizumab, belimumab, interferon, natalizumab, glatiramer acetate, fingolimod,
teriflunomide, dimethyl fumarate within 6 months prior to baseline.
3. Any previous treatment with alemtuzumab, anti-CD4, cladribine, total body
irradiation, bone marrow transplantation within 2 years prior to baseline.
4. Treatment with any investigational agent within 3 months prior to baseline.
Exclusions for general safety:
5. Pregnancy or lactation.
6. For patients of reproductive potential, a positive result from a serum pregnancy test
at screening, or not willing to use reliable means of contraception.
7. Any surgical procedure (except for minor surgeries) within 4 weeks prior to baseline.
8. Evidence of other demyelinating disease or progressive multifocal
leukoencephalopathy (PML).
9. Evidence of 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.
10. Known active infection (excluding fungal infections of nail beds or caries dentium)
within 4 weeks prior to baseline.
11. Evidence of chronic active hepatitis B or C.
12. History of drug or alcohol abuse.
13. History of diverticulitis that, in the Investigator’s opinion, may preclude patient
participation.
14. Evidence of active tuberculosis (excluding patients receiving chemoprophylaxis for
latent tuberculosis infection).
15. Evidence of active interstitial lung disease.
16. Receipt of any live or live attenuated vaccine within 6 weeks prior to baseline.
17. History of malignancy within the last 5 years, including solid tumors, hematologic
malignancies and in situ carcinoma (except basal cell and squamous cell carcinomas
of the skin, or in situ carcinoma of the cervix uteri that have been completely excised
and cured).
18. History of severe allergic reaction to a biologic agent (e.g. shock, anaphylactic
reactions).
Laboratory exclusion criteria (at screening):
19. Following laboratory abnormalities at screening.
a. White blood cells (WBC) <4.0 x103 /μL
b. Absolute neutrophil count (ANC) <2.0 x 103
/μL
c. Absolute lymphocyte count <0.5 x 103 /μL
d. Platelet count <10 x 104
/μL
e. Aspartate aminotransferase (AST) or alanine aminotranferase (ALT) >1.5 times
the upper limit of normal (ULN).
The Estimated Number of Participants
-
Taiwan
20 participants
-
Global
70 participants
