Table of contents

• Trial overview
• Conditions and patient groups
• Study phases and trial designs
• What the trials measure
• Treatment routes and comparators
• Key trials in the data
• How to read the endpoints

Trial overview

The trial data show that Ublituximab is being studied in several interventional trials, which means the research team gives a treatment and then measures what happens.^[1][1][1]

These studies are focused mainly on multiple sclerosis (MS), especially relapsing forms, but one study also includes autoimmune diseases such as myasthenia gravis.^[1][1]

Conditions and patient groups

Most of the listed trials study people with relapsing multiple sclerosis or relapsing remitting multiple sclerosis, where symptoms can flare up and then improve.^[1][1][1]

Some studies include people with primary or secondary progressive multiple sclerosis, which means the disease gets worse over time rather than in clear relapses.^[1]

One trial is designed for pediatric participants, meaning children and teenagers ages 10 to under 18 years with relapsing forms of MS.^[1]

Another trial includes people with autoimmune diseases, including relapsing multiple sclerosis and myasthenia gravis.^[1]

Study phases and trial designs

The trial set includes Phase 1, Phase 2, Phase 3, and Phase 4 studies, showing research at different stages of development and follow-up.^{[1][1][1][1][1][1]}

The Phase 1 study looks at intravenous and subcutaneous administration in autoimmune disease, with a focus on pharmacokinetics and B-cell count.^[1]

The Phase 2 studies include one trial in relapsing remitting MS that measures no evidence of disease activity over 36 months, and another Phase 2 study in progressive MS that evaluates confirmed disability progression.^[1][1]

The Phase 3 studies are larger and look at long-term safety and efficacy, a modified regimen, and subcutaneous administration compared with intravenous administration.^[1][1][1]

The Phase 4 pediatric study focuses on long-term safety and efficacy in children and teenagers with relapsing MS.^[1]

What the trials measure

The main trial goals include safety, efficacy (how well a treatment works), and pharmacokinetics (how the body handles the treatment).^{[1][1][1][1][1]}

Several studies measure MS disease activity using MRI findings such as T1 gadolinium enhancing lesions, T2 lesions, and brain atrophy, which are signs of inflammation or tissue change seen on scans.^[1][1]

Other studies measure relapse outcomes such as annualized relapse rate, which is the number of relapses per person-year.^[1]

Disability outcomes include EDSS changes and confirmed disability progression or improvement. EDSS is a scale used to rate disability in MS.^[1][1][1]

Some trials also measure cognition with the Symbol Digit Modalities Test and function with the Multiple Sclerosis Functional Composite.^[1]

Safety measures include adverse events, infusion-associated events, infectious adverse events, lab changes, vital sign changes, and anti-drug antibodies, which are antibodies the body may make against the treatment.^[1][1][1][1]

Treatment routes and comparators

Some trials study Ublituximab given by intravenous infusion, which means it is given through a vein, while others study subcutaneous injection, which means it is given under the skin.^[1][1][1][1]

One study compares subcutaneous Ublituximab with intravenous Ublituximab, and another compares an autoinjector device with a syringe for subcutaneous use.^[1][1]

The pediatric trial includes a comparison with fingolimod placebo and Ublituximab placebo, which are inactive look-alike treatments used for comparison.^[1]

Some studies also use other medicines around the infusion, such as dexamethasone, paracetamol, cetirizine, methylprednisolone, or diphenhydramine, as listed in the trial records.^[1][1][1]

Key trials in the data

2024-516680-91-00 is a Phase 3 open-label extension study in relapsing MS with 879 participants. It looks at long-term safety and efficacy, including relapses, MRI outcomes, disability, cognition, function, and safety events.^[1]

2024-519284-18-00 is a Phase 3 study called ENHANCE with 800 participants. It tests a modified regimen of Ublituximab, compares pharmacokinetics, and studies people who had a suboptimal experience on prior anti-CD20 therapy.^[1]

2025-521127-73-00 is a Phase 3 study in relapsing forms of MS with 360 participants. It evaluates subcutaneous Ublituximab and compares it with intravenous administration, with a focus on pharmacokinetics, pharmacodynamics, safety, and radiological and clinical effects.^[1]

2025-522257-19-00 is a Phase 4 pediatric study with 248 participants. It studies Ublituximab in children and teenagers with relapsing MS and measures safety, suicidal ideation using the Columbia-Suicide Severity Rating Scale, and B-cell levels.^[1]

2023-509555-13-00 is a Phase 1 study in autoimmune diseases with 164 participants. It evaluates intravenous and subcutaneous Ublituximab and measures pharmacokinetics and B-cell count.^[1]

2024-515470-26-00 is a Phase 2 study in relapsing remitting MS with 90 participants. Its main goal is to see whether there is any evidence of disease activity, called NEDA-3, over 36 months.^[1]

NCT06384976 is a Phase 2 study in primary or secondary progressive MS with 134 participants. It evaluates confirmed disability progression and the efficacy of KYV-101, with Ublituximab listed among the trial interventions.^[1]

How to read the endpoints

An endpoint is the main result a trial wants to measure. It helps show whether the study treatment is working or is safe.^[1][1]

Primary outcome means the most important result the study plans to measure first. In these trials, primary outcomes include disease activity, disability progression, B-cell counts, and treatment safety.^{[1][1][1][1][1][1][1]}

NEDA-3 means no evidence of disease activity, using relapse events, MRI activity, and disability progression. It is a way to see whether MS appears quiet during the study period.^[1][1]

Confirmed disability progression means disability has gotten worse and this change is still present after a set time, not just for a short period.^[1][1][1]

Bioavailability means how much of a treatment enters the body in a form it can use. Some trials compare this between injection methods or devices.^[1][1]