Clinical Trials List
Protocol NumberMOG001
NCT Number(ClinicalTrials.gov Identfier)NCT05063162
Active
2023-03-01 - 2025-09-09
Phase III
Not yet recruiting6
ICD-10G36.0
Neuromyelitis optica [Devic]
ICD-9341.0
Neuromyelitis optica
A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 3, Pivotal Study With an Open-Label Extension Period to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-Associated Disease (MOG-AD)
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Trial Applicant
PAREXEL INTERNATIONAL CO., LTD.
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Sponsor
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Trial scale
Multi-Regional Multi-Center
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Update
2026/02/01
Investigators and Locations
Co-Principal Investigator
- 邵儀菁 Division of Neurology
- Ching-Hua Lu Lu Division of Neurology
- Fu-Yu Lin Division of Neurology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 張宏旭 Division of Neurology
- 陳與潔 Division of Neurology
- 朱俊哲 Division of Neurology
- 孫銘輝 Division of Neurology
- Long-Sun Ro Division of Neurology
- Chin-Chang Huang Division of Neurology
- Hong-Chou Kuo Division of Neurology
- 杜振豐 Division of Neurology
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Condition/Disease
Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease (MOG-AD)
Objectives
MOG-AD is an inflammatory demyelinating disease caused by serum MOG antibodies. MOG-AD most commonly presents as optic neuritis (ON) (occurring in 54% to 61% of patients), followed by
Caused by myelitis, acute disseminated encephalomyelitis (ADEM), or ADEM-like conditions (eg, brainstem attacks) (Wynford-Thomas et al., 2019). Already 44% to 83%
MOG-AD patients report a relapsing course (Cobo-Calvo et al., 2018) and increasingly often affects the optic nerve (Jurynczyk et al., 2017). Rozanolixizumab is a human
Anti-FcRn monoclonal antibody, specifically designed to inhibit the binding of IgG to FcRn, but does not inhibit the binding of albumin to FcRn. MOG-AD contains pathogenic IgG antibodies, and removal of IgG antibodies by blocking FcRn may have clinical benefits in reducing autoimmune attack and relapse. The current trial will evaluate the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of rozanolixizumab versus placebo in participants with MOG-AD.
Double-blind trial period (Part A)
main target
Assessing the efficacy of rozanolixizumab in participants with MOG-AD Secondary objectives
To assess the safety and tolerability of rozanolixizumab in participants with MOG-AD
Other goals
Evaluating the PK Properties of rozanolixizumab
To assess the incidence and occurrence of rozanolixizumab anti-drug antibodies (ADA)
Evaluating the PD effects of rozanolixizumab
To evaluate the effect of rozanolixizumab on total protein, albumin, alpha- and beta-globulins, IgM, IgA, IgE, serum and plasma complement concentrations
Evaluating the effects of rozanolixizumab on exploratory biomarkers
Assessing the impact of rozanolixizumab treatment on vaccine potency
Evaluating the impact of rozanolixizumab on vaccine response in trial participants who have received a COVID-19 vaccine
Open-label extension (OLE) trial (Part B)
main target
To further evaluate the safety and tolerability of rozanolixizumab in participants with MOG-AD
secondary goals
Provide additional efficacy information on ARR during the OLE treatment period
Provide additional narrative, non-comparative efficacy information during the OLE treatment period to supplement the double-blind treatment period
Test Drug
Rozanolixizumab
Active Ingredient
Rozanolixizumab
Dosage Form
solution for injection
Dosage
140mg/mL
Endpoints
Main test purpose
Main test indicators (efficacy):
‧ Time from randomization to first independently centrally judged relapse (TTFR)
Main test indicators (efficacy):
‧ Time from randomization to first independently centrally judged relapse (TTFR)
Inclution Criteria
Participants are eligible to be included in the study only if all of the following criteria apply:
Age
1. Participant must be ?18 to ?89 years of age, at the time of signing the informed consent. For Japan-specific requirements, see Appendix 9, Section 10.9.
Type of Participant and Disease Characteristics
2b. Confirmed diagnosis of MOG-AD consistent with published diagnostic criteria for MOG-AD (Banwell 2023).
3. Removed.
4a. Participant has history of relapsing MOG-AD with at least 1 documented relapse over the last 12 months and a documented positive serum MOG Ab test using a cell-based assay (CBA) within 6 months prior to randomization.
5. Participant must be clinically stable at the time of the Screening Visit and during the Screening Period
Weight
6. Participant weighs at least 35kg (for males and females) at Screening.
Sex
7a. Male or female
? Removed.
? A female participant is eligible to participate if she is not pregnant (see Appendix 4 [(Section 10.4)]), not breastfeeding, and at least one of the following conditions applies:
? Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 (Section 10.4)
OR
A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 (Section 10.4) during the treatment period and for at least 90 days after the last dose of study treatment.
Informed Consent
8. Participant is capable of giving signed informed consent as described in Appendix 1 (Section 10.1.3) which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. For France-specific requirements, see Appendix 9 (Section 10.9).
Other
9. Participant is considered reliable and capable of adhering to the protocol visit schedule or medication intake according to the judgment of the investigator.Participants are excluded from the study if any of the following criteria apply (criteria are applicable for the Screening Visit unless otherwise stated):
Medical Conditions
1. Participant has any medical or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the participant’s ability to participate in this study.
2. Participant has been diagnosed with a neurological autoimmune disease (including MS and aquaporin-4 positive NMOSD), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant.
3. Participant has a history of alcohol use disorder or other substance use disorder (as per Diagnostic and Statistical Manual of Mental Disorders-5) within the previous 12 months.
4. Participant has a known hypersensitivity to any components of the study medication or any other anti-FcRn medications. This includes a known history of hyperprolinemia, since Lproline is a constituent of the rozanolixizumab formulation.
5a. Participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator.
6. Participant has 12-lead ECG with findings considered to be clinically significant upon medical review. The clinical significance of the findings needs to be assessed by the investigator to determine eligibility.
7. Participant has a history of chronic ongoing infections (eg, Hepatitis B or C, human immune deficiency virus [HIV], active or latent tuberculosis [TB]) or who tests positive for HIV, Hepatitis B or C at Screening.
8. Participant has active neoplastic disease or history of neoplastic disease within 5 years of study entry (screening) (except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the uterine cervix that have been definitively treated with standard of care approaches).
9. Participant has renal impairment, defined as a glomerular filtration rate less than 45mL/min/1.73m2 at the Screening Visit.
10. Participant has current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: with exception of stable chronic liver disease (including Gilbert’s syndrome, asymptomatic gallstones).
12a. Participant meets any of the following TB exclusion criteria:
‧ Known active TB disease
‧ History of active TB involving any organ system unless adequately treated according to World Health Organization (WHO)/Centers for Disease Control and Prevention (CDC) therapeutic guidance and proven to be fully recovered upon consult with an appropriate specialist
‧ Latent TB infection (LTBI) (unless appropriate treatment is initiated at least 1 week prior to IMP dosing and will be continued to completion). Tuberculosis preventive therapy should be in accordance with applicable clinical guidelines and appropriate specialist judgment.
‧ High risk of exposure to TB infection, as assessed by the investigator
For further information relating to definitions of known active TB, past history of TB, LTBI, high risk of acquiring TB infection and NTM infection, see Appendix 12, Section 10.12.
13. Participant has a current or medical history of primary immunodeficiency.
14. Removed.
15. Participant has a history of a solid organ transplant or hematopoietic stem cell/marrow transplant.
16. Removed.
17. Participant has undergone a splenectomy.
Prior/Concomitant therapy
18. Participant has been previously treated with rozanolixizumab in a clinical study.
19a.Participant has received a live vaccination within 4 weeks prior to Visit 1A (1 year for Bacillus Calmette–Guerin [BCG]), or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of IMP
20b.Participant has been treated with prohibited immunosuppressants, immunomodulators, biologics, and other therapies within timeframe shorter than that listed in the following table (refer to protocol)
Prior/Concurrent Clinical Study Experience
21. Participant has previously been randomized to treatment in this study.
22. Participant has participated in another study of an IMP (and/or an investigational device) within the previous 3 months or 5 half-lives prior to Baseline (whichever is longer) or is currently participating in another study of an IMP (and/or an investigational device).
Diagnostic Assessments
23. Participant tests positive for aquaporin-4 antibodies at Screening.
24. MRI findings at screening highly suggestive of MS as assessed by the central reader, such as presence of a lesion adjacent to the lateral ventricle that is ovoid/round or associated with an inferior temporal lobe lesion, or Dawson’s finger-type lesions, or brain MRI activity over time with silent increase in lesion burden between relapses.
25. Participant has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the participant′s ability to participate in this study.
26. Removed.
27. Participant has a serum total IgG level ?5.5g/L.
28. Participant has absolute neutrophil count ＜1500 cells/mm3 .
29a.Alanine transaminase (ALT), aspartate aminotransferase (AST) or alkaline phosphatase (ALP) are ＞3.0x upper limit of normal (ULN).
For randomized participants with a Baseline result ＞ULN for ALT, AST, ALP, or total bilirubin but ＜1.5xULN, a Baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in the Case Report form (CRF).
If participant has ＞ULN ALT, AST, or ALP that does not meet the exclusion limit at screening, the tests should be repeated, if possible, prior to dosing to ensure there was no further ongoing clinically relevant increase. In case of a clinically relevant increase as per investigator’s judgment, inclusion of the participant must be discussed with the Medical Monitor.
Study participant has bilirubin ＞1.5xULN at Screening (Visit 1) (unless confirmed Gilbert’s syndrome). If participant has elevations only in total bilirubin, the fractionate bilirubin needs to be checked, to identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin ＜35%).
Other Exclusions
30. Participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation with at least some intent to act in the past 6 months as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) at screening.
31. Participant is unable to undergo an MRI scan (eg, hypersensitivity to MRI contrast agents, implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans).
For Japan- and France-specific requirements, see Appendix 9 (Section 10.9).
Age
1. Participant must be ?18 to ?89 years of age, at the time of signing the informed consent. For Japan-specific requirements, see Appendix 9, Section 10.9.
Type of Participant and Disease Characteristics
2b. Confirmed diagnosis of MOG-AD consistent with published diagnostic criteria for MOG-AD (Banwell 2023).
3. Removed.
4a. Participant has history of relapsing MOG-AD with at least 1 documented relapse over the last 12 months and a documented positive serum MOG Ab test using a cell-based assay (CBA) within 6 months prior to randomization.
5. Participant must be clinically stable at the time of the Screening Visit and during the Screening Period
Weight
6. Participant weighs at least 35kg (for males and females) at Screening.
Sex
7a. Male or female
? Removed.
? A female participant is eligible to participate if she is not pregnant (see Appendix 4 [(Section 10.4)]), not breastfeeding, and at least one of the following conditions applies:
? Not a woman of childbearing potential (WOCBP) as defined in Appendix 4 (Section 10.4)
OR
A WOCBP who agrees to follow the contraceptive guidance in Appendix 4 (Section 10.4) during the treatment period and for at least 90 days after the last dose of study treatment.
Informed Consent
8. Participant is capable of giving signed informed consent as described in Appendix 1 (Section 10.1.3) which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. For France-specific requirements, see Appendix 9 (Section 10.9).
Other
9. Participant is considered reliable and capable of adhering to the protocol visit schedule or medication intake according to the judgment of the investigator.Participants are excluded from the study if any of the following criteria apply (criteria are applicable for the Screening Visit unless otherwise stated):
Medical Conditions
1. Participant has any medical or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the participant’s ability to participate in this study.
2. Participant has been diagnosed with a neurological autoimmune disease (including MS and aquaporin-4 positive NMOSD), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant.
3. Participant has a history of alcohol use disorder or other substance use disorder (as per Diagnostic and Statistical Manual of Mental Disorders-5) within the previous 12 months.
4. Participant has a known hypersensitivity to any components of the study medication or any other anti-FcRn medications. This includes a known history of hyperprolinemia, since Lproline is a constituent of the rozanolixizumab formulation.
5a. Participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator.
6. Participant has 12-lead ECG with findings considered to be clinically significant upon medical review. The clinical significance of the findings needs to be assessed by the investigator to determine eligibility.
7. Participant has a history of chronic ongoing infections (eg, Hepatitis B or C, human immune deficiency virus [HIV], active or latent tuberculosis [TB]) or who tests positive for HIV, Hepatitis B or C at Screening.
8. Participant has active neoplastic disease or history of neoplastic disease within 5 years of study entry (screening) (except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the uterine cervix that have been definitively treated with standard of care approaches).
9. Participant has renal impairment, defined as a glomerular filtration rate less than 45mL/min/1.73m2 at the Screening Visit.
10. Participant has current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: with exception of stable chronic liver disease (including Gilbert’s syndrome, asymptomatic gallstones).
12a. Participant meets any of the following TB exclusion criteria:
‧ Known active TB disease
‧ History of active TB involving any organ system unless adequately treated according to World Health Organization (WHO)/Centers for Disease Control and Prevention (CDC) therapeutic guidance and proven to be fully recovered upon consult with an appropriate specialist
‧ Latent TB infection (LTBI) (unless appropriate treatment is initiated at least 1 week prior to IMP dosing and will be continued to completion). Tuberculosis preventive therapy should be in accordance with applicable clinical guidelines and appropriate specialist judgment.
‧ High risk of exposure to TB infection, as assessed by the investigator
For further information relating to definitions of known active TB, past history of TB, LTBI, high risk of acquiring TB infection and NTM infection, see Appendix 12, Section 10.12.
13. Participant has a current or medical history of primary immunodeficiency.
14. Removed.
15. Participant has a history of a solid organ transplant or hematopoietic stem cell/marrow transplant.
16. Removed.
17. Participant has undergone a splenectomy.
Prior/Concomitant therapy
18. Participant has been previously treated with rozanolixizumab in a clinical study.
19a.Participant has received a live vaccination within 4 weeks prior to Visit 1A (1 year for Bacillus Calmette–Guerin [BCG]), or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of IMP
20b.Participant has been treated with prohibited immunosuppressants, immunomodulators, biologics, and other therapies within timeframe shorter than that listed in the following table (refer to protocol)
Prior/Concurrent Clinical Study Experience
21. Participant has previously been randomized to treatment in this study.
22. Participant has participated in another study of an IMP (and/or an investigational device) within the previous 3 months or 5 half-lives prior to Baseline (whichever is longer) or is currently participating in another study of an IMP (and/or an investigational device).
Diagnostic Assessments
23. Participant tests positive for aquaporin-4 antibodies at Screening.
24. MRI findings at screening highly suggestive of MS as assessed by the central reader, such as presence of a lesion adjacent to the lateral ventricle that is ovoid/round or associated with an inferior temporal lobe lesion, or Dawson’s finger-type lesions, or brain MRI activity over time with silent increase in lesion burden between relapses.
25. Participant has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the participant′s ability to participate in this study.
26. Removed.
27. Participant has a serum total IgG level ?5.5g/L.
28. Participant has absolute neutrophil count ＜1500 cells/mm3 .
29a.Alanine transaminase (ALT), aspartate aminotransferase (AST) or alkaline phosphatase (ALP) are ＞3.0x upper limit of normal (ULN).
For randomized participants with a Baseline result ＞ULN for ALT, AST, ALP, or total bilirubin but ＜1.5xULN, a Baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in the Case Report form (CRF).
If participant has ＞ULN ALT, AST, or ALP that does not meet the exclusion limit at screening, the tests should be repeated, if possible, prior to dosing to ensure there was no further ongoing clinically relevant increase. In case of a clinically relevant increase as per investigator’s judgment, inclusion of the participant must be discussed with the Medical Monitor.
Study participant has bilirubin ＞1.5xULN at Screening (Visit 1) (unless confirmed Gilbert’s syndrome). If participant has elevations only in total bilirubin, the fractionate bilirubin needs to be checked, to identify possible undiagnosed Gilbert’s syndrome (ie, direct bilirubin ＜35%).
Other Exclusions
30. Participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation with at least some intent to act in the past 6 months as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) at screening.
31. Participant is unable to undergo an MRI scan (eg, hypersensitivity to MRI contrast agents, implanted pacemakers, defibrillators, or other metallic objects on or inside the body that limit performing MRI scans).
For Japan- and France-specific requirements, see Appendix 9 (Section 10.9).
Exclusion Criteria
Exclusion Criteria:
Participant has been diagnosed with a neurological autoimmune disease (including multiple sclerosis (MS) and aquaporin-4 positive neuromyelitis optica spectrum disorder (NMOSD)), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant
Participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator, including participants with a serious infection within 6 weeks prior to the first dose of the investigational medicinal product (IMP)
Participant has a current or medical history of primary immunodeficiency
Participant tests positive for aquaporin-4 antibodies at Screening
Participant has a serum total IgG level ≤ 5.5g/L
Participant has been diagnosed with a neurological autoimmune disease (including multiple sclerosis (MS) and aquaporin-4 positive neuromyelitis optica spectrum disorder (NMOSD)), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant
Participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator, including participants with a serious infection within 6 weeks prior to the first dose of the investigational medicinal product (IMP)
Participant has a current or medical history of primary immunodeficiency
Participant tests positive for aquaporin-4 antibodies at Screening
Participant has a serum total IgG level ≤ 5.5g/L
The Estimated Number of Participants
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Taiwan
10 participants
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Global
104 participants
