Rozanolixizumab for MOG-AD

(cosMOG Trial)

Recruiting at 103 trial locations

Overseen By UCB Cares

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: UCB Biopharma SRL

Disqualifiers: Multiple sclerosis, NMOSD, Active infection, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

104 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests rozanolixizumab, an experimental drug, to determine its effectiveness for people with MOG-AD, a condition causing nervous system inflammation and symptoms like vision and movement issues. The trial aims to assess the safety and effectiveness of rozanolixizumab compared to a placebo, which contains no active medication. Individuals with MOG-AD who have experienced at least one flare-up in the past year and have a positive test for MOG antibodies may qualify for this study. Participants will be randomly assigned to receive either the treatment or a placebo. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that rozanolixizumab is likely to be safe for humans?

Research has shown that rozanolixizumab is already approved in the USA for other conditions, specifically certain types of generalized myasthenia gravis (gMG). This approval indicates that its safety has been tested to some degree. When used for gMG, most patients have tolerated it well, although some have experienced mild to moderate side effects like headaches and nausea.

The current study's later phase suggests some confidence in its safety from earlier research. However, as with any treatment, monitoring for side effects remains important. Prospective trial participants should discuss any concerns with their healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for MOG-AD?

Unlike the standard treatments for MOG-AD, which often include steroids and immunosuppressants, Rozanolixizumab acts on a different pathway by targeting the neonatal Fc receptor (FcRn). This approach is designed to reduce the levels of pathogenic antibodies in the bloodstream, potentially offering a more targeted treatment option. Researchers are excited about Rozanolixizumab because it could provide a new way to manage MOG-AD with possibly fewer side effects and more precise control over the disease's immune response.

What evidence suggests that rozanolixizumab might be an effective treatment for MOG-AD?

Research shows that rozanolixizumab, which participants in this trial may receive, may help treat conditions where the immune system mistakenly attacks its own tissues. It is already approved in the U.S. for treating certain types of generalized myasthenia gravis, a condition that causes muscle weakness. The drug lowers the levels of harmful antibodies, proteins in the blood that can wrongly attack the body's own cells. Early studies suggest that rozanolixizumab might also be effective for myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD) by targeting these harmful antibodies. This method has succeeded in similar conditions, offering hope for those with MOG-AD. ^{1 2 3 4 6}

Who Is on the Research Team?

UCB Cares

Principal Investigator

001 844 599 2273

Are You a Good Fit for This Trial?

This trial is for adults aged 18-89 with a confirmed diagnosis of MOG-AD, who have had at least one relapse in the past year and tested positive for MOG antibodies recently. They must be stable health-wise and not have other autoimmune diseases or primary immunodeficiency.

Inclusion Criteria

Participant must be clinically stable at the time of the Screening Visit and during the Screening Period

I have been diagnosed with MOG Antibody Disease.

I have had at least one MOG-AD relapse in the past year and a positive MOG antibody test.

Exclusion Criteria

I tested positive for aquaporin-4 antibodies.

Participant has a serum total IgG level ≤ 5.5g/L

I do not have any serious ongoing infections.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-Blind Treatment

Participants receive either rozanolixizumab or placebo in a double-blind manner

up to approximately 132 weeks

Open-Label Extension

Participants receive rozanolixizumab in an open-label manner

up to 52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rozanolixizumab

Trial Overview The study tests Rozanolixizumab's effectiveness and safety against a placebo in treating MOG-AD. Participants will randomly receive either the actual drug or a placebo to compare outcomes.

How Is the Trial Designed?

Group I: Rozanolixizumab Arm Experimental Treatment 1 Intervention

Participants randomized into this arm will receive rozanolixizumab at pre-specified timepoints.

Group II: Placebo Arm Placebo Group 1 Intervention

Participants randomized into this arm will receive placebo at pre-specified timepoints to maintain the blinding.

Rozanolixizumab is already approved in United States, European Union, Japan for the following indications:

Approved in United States as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Approved in European Union as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Approved in Japan as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Who Is Running the Clinical Trial?

UCB Biopharma SRL

Lead Sponsor

Trials

118

Recruited

23,200+

Jean-Christophe Tellier

UCB Biopharma SRL

Chief Executive Officer since 2015

MD from University of Reims Champagne-Ardenne, Rheumatology specialization from University of Paris V, Executive business programs at Harvard and INSEAD

Dr. Iris Loew-Friedrich

UCB Biopharma SRL

Chief Medical Officer since 2014

MD from University of Leuven, PhD in Medical Sciences from University of Leuven

Published Research Related to This Trial

Researchers developed new RORγt inverse agonists that effectively inhibit the production of pro-inflammatory Th17 cytokines, which are linked to autoimmune diseases, by designing compounds with improved potency and physicochemical properties.

Two specific compounds, identified as 10 and 33, demonstrated strong pharmacological activity in both biochemical assays and a rodent model, successfully reducing IL-17 cytokine production after oral administration at a dose of 15 mg/kg.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Synthesis and Biological Evaluation of New Triazolo- and Imidazolopyridine RORγt Inverse Agonists. Hintermann, S., Guntermann, C., Mattes, H., et al.[2018]

A new compound, identified as an RORγt agonist, was developed through structure-based drug design and showed a favorable pharmacokinetic profile in a mouse tumor model.

Despite its promising design and pharmacokinetics, the compound did not demonstrate any significant efficacy in reducing tumor growth at the tested doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Substituted benzyloxytricyclic compounds as retinoic acid-related orphan receptor gamma t (RORγt) agonists. Harikrishnan, LS., Gill, P., Kamau, MG., et al.[2023]

Researchers developed a new lead compound, MG 2778, as an inverse agonist for the RORγt receptor, which is important for treating immune diseases, and created a small library of modified compounds to test their effectiveness.

Among the tested compounds, 19a showed the highest activity in reducing RORγt activity at low micromolar concentrations, although all compounds were less potent than the reference compound ursolic acid, indicating potential for further development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Targeting RORs nuclear receptors by novel synthetic steroidal inverse agonists for autoimmune disorders. Dal Prà, M., Carta, D., Szabadkai, G., et al.[2018]

A new series of biaryl amides were developed as RORγt inhibitors, showing promising oral bioavailability and the ability to penetrate the central nervous system (CNS).

Compounds 9a and 9g exhibited strong in vivo efficacy in a mouse model of experimental autoimmune encephalomyelitis (EAE), demonstrating effective results with once daily oral dosing.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Discovery of Biaryl Amides as Potent, Orally Bioavailable, and CNS Penetrant RORγt Inhibitors. Wang, Y., Cai, W., Cheng, Y., et al.[2020]

A new series of tetrahydroisoquinoline derivatives were developed as potential oral treatments for autoimmune diseases driven by Th17 cells, with compound 15 showing promising results as an inverse agonist for the RORγt receptor.

Compound 15 demonstrated a strong ability to inhibit IL-17A production in mice, indicating its potential efficacy in treating Th17-driven autoimmune conditions, along with an improved pharmacokinetic profile for better absorption and effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Discovery of orally efficacious RORγt inverse agonists. Part 2: Design, synthesis, and biological evaluation of novel tetrahydroisoquinoline derivatives. Kono, M., Oda, T., Tawada, M., et al.[2018]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05063162

NCT05063162 | A Study to Evaluate the Efficacy and ... The purpose of the study is to evalute the efficacy, safety and tolerability of rozanolixizumab for treatment of adult participants with myelin oligodendrocyte ...

2. ucb.com ucb.com/innovation/clinical-studies/clinical-studies-index/Rozanolixizumab-UCB7665

Rozanolixizumab (Rystiggo®) Press Release 11 Apr 2025 Global data show real-world effectiveness of romosozumab for patients at high fracture risk.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10590285/

Rozanolixizumab: First Approval - PMC Rozanolixizumab is the first agent to be approved in the USA for both anti-AChR and anti-MuSK antibody-positive gMG.

4. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-rozanolixizumab-for-adults-with-myelin-oligodendrocyte-glycoprotein-antibody-associated-disease-mog-ad/

Study on the Effectiveness and Safety of Rozanolixizumab ... The purpose of the study is to evaluate how effective and safe Rozanolixizumab is for treating adults with MOGAD.

5. trialx.com trialx.com/clinical-trials/listings/281601/a-study-to-evaluate-the-efficacy-and-safety-of-rozanolixizumab-in-adult-participants-with-myelin-oligodendrocyte-glycoprotein-mog-antibody-associated-disease-mog-ad/

A Study to Evaluate the Efficacy and Safety ... The purpose of the study is to evalute the efficacy, safety and tolerability of rozanolixizumab for treatment of adult participants with myelin ...

6. mayo.edu mayo.edu/research/clinical-trials/cls-20535974

Clinical Trials A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Myelin Oligodendrocyte Glycoprotein (MOG) Antibody- ...