Ublituximab in Pediatric Participants With Relapsing Forms of Multiple Sclerosis (RMS)

Plain English Summary

Study to Assess Effects of Ublituximab in Pediatric Participants With Relapsing Forms of Multiple Sclerosis is a Phase 3 clinical trial sponsored by TG Therapeutics, Inc. studying Relapsing Multiple Sclerosis. Tests the safety and effectiveness of ublituximab in children with relapsing multiple sclerosis (RMS). For children aged 10-17, weighing 25-40 kg, with RMS. Participation involves IV infusions of ublituximab or placebo, and regular check-ups. Alternatives include other MS treatments like fingolimod. The trial aims to enroll 240 participants.

Official Summary

The primary purpose of this study is to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of ublituximab in participants ages 10 to less than (\<)18 years and body weight greater than or equal to (≥)25 kilograms (kg) to less than or equal to (≤)40 kg with RMS (Part A) and to evaluate the non-inferiority of ublituximab compared with fingolimod in pediatric RMS participants with body weight ≥ 25 kg (Part B). The study will further evaluate long-term safety and efficacy of ublituximab in RMS in pediatric participants during its extension period (Part C).

Who Can Participate

Here is what you need to know about eligibility for this trial. Ages 10-17, weighing 25-40 kg, diagnosed with RMS. No other neurological disorders, no prior treatments like corticosteroids or B cell treatments. Must be neurologically stable for at least 30 days. No use of certain anti-arrhythmic drugs or anti-CD20 treatments. This trial is studying Relapsing Multiple Sclerosis, so participants generally need a confirmed diagnosis.

What They're Measuring

Measures include blood levels of ublituximab, changes in B cell counts, and relapse rates, helping patients understand treatment impact. The specific primary outcome measures are: Part A: Area Under the Curve From Week 0 to 24 (AUC0-W24) of Ublituximab (Predose and multiple timepoints up to Week 24); Part A: Maximum Observed Concentration (Cmax) of Ublituximab (Day 1 and Day 15); Part A: Participant B Cell Counts (Up to Week 24); Part B: Annualized Relapse Rate (ARR) (Up to 96 weeks); Part C: Annualized Relapse Rate (ARR) (Up to 168 weeks). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses the need for safe and effective treatments for pediatric RMS, a condition with limited options. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Relapsing Multiple Sclerosis, where improved treatment options are needed.

Investor Insight

Market size is significant, with limited pediatric-specific treatments available, indicating strong potential for approval. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor about your child's eligibility and the risks involved. Participation involves regular IV infusions and check-ups. The trial is being conducted at multiple sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 240 participants

Interventions

• DRUG: Ublituximab — Administered as an intravenous (IV) infusion.
• DRUG: Placebo — Oral capsule.
• DRUG: Placebo — IV infusion.
• DRUG: Fingolimod — Oral capsule.

Primary Outcomes

• Part A: Area Under the Curve From Week 0 to 24 (AUC0-W24) of Ublituximab (Predose and multiple timepoints up to Week 24)
• Part A: Maximum Observed Concentration (Cmax) of Ublituximab (Day 1 and Day 15)
• Part A: Participant B Cell Counts (Up to Week 24)
• Part B: Annualized Relapse Rate (ARR) (Up to 96 weeks)
• Part C: Annualized Relapse Rate (ARR) (Up to 168 weeks)

Secondary Outcomes

• Part A, B and C: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (Part A: Up to Week 24; Part B: Up to 96 weeks; Part C: Up to 168 weeks)
• Part A, B and C: Number of Participants With Change in Columbia-Suicide Severity Rating Scale (C-SSRS ) (Part A: Up to Week 24; Part B: Up to 96 weeks; Part C: Up to 168 weeks)
• Part A: Serum Concentrations of Ublituximab (Up to Week 24)
• Part A and B: Percentage of Participants with Treatment-emergent Anti-drug Antibodies (ADAs) to Ublituximab (Part A: Up to Week 24; Part B: Up to 96 weeks)
• Part A and B: Number of Gadolinium Enhancing (Gd-enhancing) T1 Lesions per Magnetic Resonance Imaging (MRI) Scan (Part A: Up to Week 24; Part B: Up to 96 weeks)

Full Eligibility Criteria

Inclusion Criteria for Part A and Part B:

1. Diagnosis of RMS.
2. EDSS at screening: 0-5.5, inclusive.
3. Neurologic stability for ≥ 30 days prior to screening, and between screening and Week 1 Day 1 (W1D1).

Inclusion Criteria for Part C:

1\. Participants must have completed Part A (Week 24 visit) or Part B (Week 96 visit) to be eligible for Part C.

Exclusion Criteria for Part A and B:

1. Known presence or suspicion of other neurologic disorders that may mimic MS.
2. Prior treatments:

   1. Systemic corticosteroids (\>0.1 milligrams/kilogram/day \[mg/kg/day\], or \>5 milligrams/day \[mg/day\] of prednisone equivalent) or adrenocorticotropic hormone (ACTH) within 30 days prior to the screening MRI scan (note: Topical, ophthalmic, or inhaled corticosteroids are permitted).
   2. High dose intravenous immunoglobulin (IVIG) or subcutaneous IG (SCIG) within 2 months prior to W1D1.
   3. Treatment with anti-CD20 or other B cell directed treatment at any time.
   4. Treatment with alemtuzumab, cladribine, cyclophosphamide, mitoxantrone at any time.

Additional Exclusion Criteria for Part B Only (Relevant to Fingolimod Treatment):

1. Treatment with fingolimod or other sphingosine-1 phosphate-1 (S1P1) modulators at any time.
2. The following antiarrhythmic drugs at Screening: Class Ia anti-arrhythmics.

Exclusion Criteria for Part C:

1\. If the absolute lymphocyte count (ALC) is outside the specified range the participant will not be eligible to receive ublituximab in Part C.

Note: Other protocol-specified inclusion/exclusion criteria may apply

Frequently Asked Questions

Related Conditions

• Relapsing multiple sclerosis (RMS), pediatric MS, neurology, immunotherapy

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