A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-associated Disease (MOG-AD)

Study Overview
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Brief Summary
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The purpose of the study is to evalute the efficacy, safety and tolerability of rozanolixizumab for treatment of adult participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD).

Official Title
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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)

Conditions
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Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease (MOG-AD)

Intervention / Treatment
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* Drug: Rozanolixizumab
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant must be ≥18 to ≤89 years of age, at the time of signing the informed consent Confirmed diagnosis of MOG-AD consistent with published diagnostic criteria for MOG-AD 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 Participant must be clinically stable at the time of the Screening Visit and during the Screening Period 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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 89 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rozanolixizumab Arm<br>Participants randomized into this arm will receive rozanolixizumab at pre-specified timepoints. | Drug: Rozanolixizumab<br>* Pharmaceutical form: Solution for infusion Route of administration: subcutaneous infusion Participants will receive pre-specified doses of rozanolixizumab.<br>* Other names: UCB7665;|
| Placebo Comparator: Placebo Arm<br>Participants randomized into this arm will receive placebo at pre-specified timepoints to maintain the blinding. | Other: Placebo<br>* Pharmaceutical form: Solution for infusion Route of administration: subcutaneous infusion Participants will receive placebo.<br>* Other names: PBO;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| For Part A: Time from randomization to first independently centrally adjudicated relapse (TTFR) during the DB Treatment Period | The TTFR (days) will be defined as the interval between the date of randomization and the first date of the objective relapse. During the Double Blind (DB) Treatment Period (Part A); EDB/EWD = End of Double-Blind/Early Withdrawal Visit | Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) | 
| For Part B: Incidence of treatment-emergent adverse events (TEAEs) during OLE Treatment Period | An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. Open-Label Extension (OLE) Treatment Period (Part B); EOS/EWD = End of Study/Early Withdrawal Visit. | OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) | 
| For Part B: Incidence of treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP) during OLE Treatment Period | An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. TEAEs leading to discontinuation of the study are reported. During Part B. | OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| For Part A: Change from Baseline in Low-Contrast Monocular Visual Acuity (Worst Affected Eye) measured by low-contrast Landolt C Broken Rings Chart at the EDB/EWD Visit | Visual acuity is a measurement of the capacity for visual discrimination of fine details. Visual acuity tests are used to determine the smallest characters that can be read on a standardized chart. The Landolt C Broken Ring Chart uses standardized incomplete rings or C, which are positioned in any direction in the chart (up, down, left, right, and 45 degree positions in between). The participant has to be able to indicate where the break of the C is located, by describing the position or by giving gesture. During Part A. | From Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) | 
| For Part A: Disability as assessed by Expanded Disability Status Scale (EDSS) scores at the EDB/EWD Visit (with confirmation at 3 months) | The EDSS is an ordinal clinical rating scale ranging from 0 (normal neurologic examination) to 10 (death) in half-point increments with half steps from 1.0 to 9.5. The higher the value the higher is the level of impairment and disability. During Part A. | Baseline (Week 1), EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) | 
| For Part A: Number of MOG-AD related inpatient hospitalizations during the DB Treatment Period | The total number of MOG-AD related hospitalizations from Baseline through EDB/EWD Visit. During Part A. | Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) | 
| For Part A: Incidence of treatment-emergent adverse events (TEAEs) during the DB Treatment Period | An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. NOTE: An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP. During Part A. | Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) | 
| For Part B: Independently centrally adjudicated annualized relapse rate (ARR) during the DB and OLE Treatment Period | An ARR will be calculated for each participant prior to randomization into the Double-Blind Treatment Period (based on available historical data), and 2 ARRs after randomization to study treatment: first for relapses occurring during the Double-Blind Treatment Period and the second for relapses occurring during the OLE Treatment Period. The relapse episodes for each participant will be recorded throughout the entire study. | Baseline (Week 1) to EOS/EWD Visit (up to OLE Week 52) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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MOG, MOGAD, Rozanolixizumab, Myelin oligodendrocyte glycoprotein, Myelin oligodendrocyte glycoprotein antibody-associated disease