NCT07220252RecruitingPhase 2
Ublituximab in Pediatric Participants With Relapsing Forms of Multiple Sclerosis (RMS)
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- TG Therapeutics, Inc.
- Enrollment
- 240
- Locations
- 2
- Primary Endpoint
- Part A: Area Under the Curve From Week 0 to 24 (AUC0-W24) of Ublituximab
Study Overview
Brief 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).
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 10 Years to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •for Part A and Part B:
- •Diagnosis of RMS.
- •EDSS at screening: 0-5.5, inclusive.
- •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:
- •Known presence or suspicion of other neurologic disorders that may mimic MS.
- •Prior treatments:
- •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).
- •High dose intravenous immunoglobulin (IVIG) or subcutaneous IG (SCIG) within 2 months prior to W1D
- •Treatment with anti-CD20 or other B cell directed treatment at any time.
- •Treatment with alemtuzumab, cladribine, cyclophosphamide, mitoxantrone at any time.
- •Additional Exclusion Criteria for Part B Only (Relevant to Fingolimod Treatment):
- •Treatment with fingolimod or other sphingosine-1 phosphate-1 (S1P1) modulators at any time.
- •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
Arms & Interventions
Part B: Fingolimod
Experimental
Intervention: Fingolimod (Drug)
Part B: IV Placebo
Placebo Comparator
Intervention: Placebo (Drug)
Part B: Placebo
Placebo Comparator
Intervention: Placebo (Drug)
Part C: OLE
Experimental
Intervention: Ublituximab (Drug)
Part A: Ublituximab
Experimental
New Regimen
Intervention: Ublituximab (Drug)
Part B: Ublituximab
Experimental
New Regimen
Intervention: Ublituximab (Drug)
Outcomes
Primary Outcomes
Part A: Area Under the Curve From Week 0 to 24 (AUC0-W24) of Ublituximab
Time Frame: Predose and multiple timepoints up to Week 24
Part A: Maximum Observed Concentration (Cmax) of Ublituximab
Time Frame: Day 1 and Day 15
Part A: Participant B Cell Counts
Time Frame: Up to Week 24
Part B: Annualized Relapse Rate (ARR)
Time Frame: Up to 96 weeks
Part C: Annualized Relapse Rate (ARR)
Time Frame: 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)
- Part A and C: Change From Baseline in Expanded Disability Status Scale (EDSS) Score(Part A: Baseline, up to Week 24; Part C: Baseline, up to 168 weeks)
- Part A and B: Number of New and/or enlarging T2 Hyperintense Lesions (NELs) per MRI Scan(Part A: Up to Week 24; Part B: Up to 96 weeks)
- Past A: Annualized Relapse Rate(Up to Week 24)
- Part B: Pharmacokinetics (PK) Serum Concentration of Ublituximab(Up to Week 96)
- Part C: Time to Confirmed Disability Improvement (CDI)(Up to Week 24)
- Part B: Percentage of Participants with CD19+ B cell counts ≤10 cells/uL(Up to 96 weeks)
- Part B: Annualized Relapse Rate ARR(Up to Week 96)
- Part C: Time to Confirmed Disability Progression (CDP)(Up to Week 24)
Investigators
Study Sites (2)
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