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

• ClinicalTrials.gov Identifier: NCT05063162
• Recruitment Status: Recruiting
• First Posted: September 30, 2021
• Last Update Posted: April 26, 2024
• Sponsor: UCB Biopharma SRL
• Information provided by (Responsible Party): UCB Pharma ( UCB Biopharma SRL )

Tracking Information

• First Submitted Date: September 21, 2021
• First Posted Date: September 30, 2021
• Last Update Posted Date: April 26, 2024
• Actual Study Start Date: February 2, 2022
• Estimated Primary Completion Date: August 10, 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 12, 2023)

• For Part A: Time from randomization to first independently centrally adjudicated relapse (TTFR) during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) ]
  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
• For Part B: Incidence of treatment-emergent adverse events (TEAEs) during OLE Treatment Period [ Time Frame: OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) ]
  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.
• For Part B: Incidence of treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP) during OLE Treatment Period [ Time Frame: OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) ]
  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.

Original Primary Outcome Measures (submitted: September 21, 2021)

• Time from randomization to first independently centrally adjudicated relapse (TTFR) during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to 132 weeks) ]
  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
• Incidence of treatment-emergent adverse events (TEAEs) during OLE Treatment Period [ Time Frame: OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) ]
  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.
• Incidence of treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP) during OLE Treatment Period [ Time Frame: OLE Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) ]
  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.

Current Secondary Outcome Measures (submitted: April 12, 2023)

• 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 [ Time Frame: From Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) ]
  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.
• For Part A: Disability as assessed by Expanded Disability Status Scale (EDSS) scores at the EDB/EWD Visit (with confirmation at 3 months) [ Time Frame: Baseline (Week 1), EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) ]
  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.
• For Part A: Number of MOG-AD related inpatient hospitalizations during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) ]
  The total number of MOG-AD related hospitalizations from Baseline through EDB/EWD Visit. During Part A.
• For Part A: Incidence of treatment-emergent adverse events (TEAEs) during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to approximately 132 weeks) ]
  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.
• For Part B: Independently centrally adjudicated annualized relapse rate (ARR) during the DB and OLE Treatment Period [ Time Frame: Baseline (Week 1) to EOS/EWD Visit (up to OLE Week 52) ]
  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.

Original Secondary Outcome Measures (submitted: September 21, 2021)

• 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 [ Time Frame: From Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) ]
  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.
• Disability as assessed by Expanded Disability Status Scale (EDSS) scores at the EDB/EWD Visit (with confirmation at 3 months) [ Time Frame: Baseline (Week 1), EDB/EWD Visit (up to 132 weeks) ]
  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.
• Number of MOG-AD related inpatient hospitalizations during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) ]
  The total number of inpatient hospitalizations from Baseline through EDB/EWD Visit. During Part A.
• Independently centrally adjudicated annualized relapse rate (ARR) during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) ]
  An on-study ARR will be calculated for each participant based on the confirmed adjudicated relapse(s) observed in the Double-Blind Treatment Period. During Part A.
• Change from Baseline in Low-Contrast Monocular Visual Acuity (Least Affected Eye) measured by low-contrast Landolt C Broken Rings Chart at the EDB/EWD Visit (with confirmation at 3 months) [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) ]
  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.
• Incidence of treatment-emergent adverse events (TEAEs) during the DB Treatment Period [ Time Frame: Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) ]
  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.
• Independently centrally adjudicated annualized relape rate (ARR) during the DB and OLE Treatment Period [ Time Frame: Baseline (Week 1) to EOS/EWD Visit (up to OLE Week 52) ]
  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. During Part A and Part B.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody-associated Disease (MOG-AD)
• Official Title: 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)
• Brief Summary: 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).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 3

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:

MOG001 consists of a Double-Blind (Part A) and an Open-Label Extension Study Period (Part B).

Primary Purpose: Treatment

• Condition: Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease (MOG-AD)

Intervention

• Drug: Rozanolixizumab
  • Pharmaceutical form: Solution for infusion
  • Route of administration: subcutaneous infusion

  Participants will receive pre-specified doses of rozanolixizumab.

  Other Name: UCB7665
• Other: Placebo
  • Pharmaceutical form: Solution for infusion
  • Route of administration: subcutaneous infusion

  Participants will receive placebo.

  Other Name: PBO

Study Arms

• Experimental: Rozanolixizumab Arm
  Participants randomized into this arm will receive rozanolixizumab at pre-specified timepoints.
  Intervention: Drug: Rozanolixizumab
• Placebo Comparator: Placebo Arm
  Participants randomized into this arm will receive placebo at pre-specified timepoints to maintain the blinding.
  Intervention: Other: Placebo

• Publications *: Mader S, Kumpfel T, Meinl E. Pathomechanisms in demyelination and astrocytopathy: autoantibodies to AQP4, MOG, GFAP, GRP78 and beyond. Curr Opin Neurol. 2022 Jun 1;35(3):427-435. doi: 10.1097/WCO.0000000000001052.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: September 21, 2021): 104
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: October 5, 2026
• Estimated Primary Completion Date: August 10, 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 89 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: UCB Cares; 1-844-599-2273 (USA); UCBCares@ucb.com

Contact: UCB Cares; 001 844 599 2273; UCBCares@ucb.com

• Listed Location Countries: Australia, Belgium, Brazil, Czechia, France, Germany, Italy, Japan, Korea, Republic of, Mexico, Portugal, Spain, Sweden, Switzerland, Taiwan, Turkey, Ukraine, United Kingdom, United States

Administrative Information

• NCT Number: NCT05063162
• Other Study ID Numbers: MOG001; 2021-000352-19 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
• Supporting Materials: Study Protocol
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: Clinical Study Report (CSR)
• Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
• URL: http://www.Vivli.org

• Current Responsible Party: UCB Pharma ( UCB Biopharma SRL )
• Original Responsible Party: Same as current
• Current Study Sponsor: UCB Biopharma SRL
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: UCB Cares; 001 844 599 2273

• PRS Account: UCB Pharma
• Verification Date: April 2024