A 2-year Randomized, 3-arm, Double-blind, Non-inferiority Study Comparing the Efficacy and Safety of Ofatumumab and Siponimod Versus Fingolimod in Pediatric Patients With Multiple Sclerosis Followed by an Open-label Extension

Plain English Summary

Efficacy and Safety of Ofatumumab and Siponimod Compared to Fingolimod in Pediatric Patients With Multiple Sclerosis is a Phase 3 clinical trial sponsored by Novartis Pharmaceuticals studying Multiple Sclerosis (MS). This study tests if ofatumumab and siponimod are as effective and safe as fingolimod in treating multiple sclerosis (MS) in young people. It is for patients aged 10 to under 18 years old who have been diagnosed with MS and have had recent disease activity. Participation involves receiving one of three study medications (ofatumumab, siponimod, or fingolimod) or a placebo, with regular check-ups and tests over two years. Alternative treatments for pediatric MS include other disease-modifying therapies, which may have different side effect profiles and administration methods. The trial aims to enroll 129 participants.

Official Summary

Efficacy and safety of ofatumumab and siponimod compared to fingolimod in pediatric patients with multiple sclerosis

Who Can Participate

Here is what you need to know about eligibility for this trial. Children and teenagers aged 10 to under 18 years old with a diagnosis of multiple sclerosis. Must have shown recent signs of MS activity, such as relapses or new lesions on MRI. Cannot have progressive forms of MS, other active immune system diseases, severe heart or kidney problems, or a history of ADEM (Acute disseminated encephalomyelitis). This trial is studying Multiple Sclerosis (MS), so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures how often patients experience MS relapses over two years, indicating how well the treatments control disease activity. The specific primary outcome measures are: Annualized relapse rate (ARR) in target pediatric participants (Baseline up to 24 months). 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 is important because it aims to find effective and safe treatment options for multiple sclerosis in children and adolescents, addressing a gap in understanding how these newer therapies per 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 Multiple Sclerosis (MS), where improved treatment options are needed.

Investor Insight

This trial, sponsored by Novartis, is investigating treatments for a rare but serious condition in a pediatric population, suggesting a focus on expanding the use of their MS therapies to younger pati 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 the potential benefits and risks of each study medication (ofatumumab, siponimod, fingolimod) and what participation entails. Be prepared for regular clinic visits for medication administration, blood tests, and MRI scans to monitor your condition and the treatment's effects. Understand that you may receive the active drug or a placebo, and the study is designed to compare the effectiveness and safety of the different options. The trial is being conducted at 20 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: 129 participants

Interventions

• DRUG: Fingolimod — Fingolimod capsule administered orally once daily at a dose of either 0.5 mg or 0.25 mg (depending on patient's body weight).
• DRUG: Ofatumumab — Ofatumumab as a solution for injection in an autoinjector containing 20 mg ofatumumab (50 mg/mL, 0.4 mL content) for subcutaneous administration. A loading dose at Day1, Day 7 and Day 14 and then injections every 4 weeks/ 6 weeks (depending on patient's body weight).
• DRUG: Siponimod — Siponimod tablet administered orally once daily. Titration period, Day 1 to Day 6, first dose is either 0.1 mg or 0.25 mg up to daily dose of either 0.5 mg, 1 mg or 2 mg (depending on CYP2C9 genotype and body weight).
• OTHER: Fingolimod placebo — Fingolimod matching placebo capsule
• OTHER: Siponimod placebo — Siponimod matching placebo tablet

Primary Outcomes

• Annualized relapse rate (ARR) in target pediatric participants (Baseline up to 24 months)

Secondary Outcomes

• Annualized relapse rate (ARR) as compared to historical interferon β-1a data (Baseline up to 24 months)
• Annualized T2 lesion rate (Baseline up to 24 months)
• Neurofilament light chain (NfL) concentrations (Day 1, Months 3,6,12,18,24)
• Plasma Concentrations of ofatumumab (Day 1, pre-dose for Day 7, Months 2,3,5,6,12,18,24)
• Plasma Concentrations of siponimod (Day 1 (2,3,4,6 h), Day 3 (2,3,4,6 h), pre-dose for Months 1 (pre, 3h), 3,5,12)

Full Eligibility Criteria

Inclusion Criteria:

1. Between 10 to \<18 years of age (i.e., have not yet had their 18th birthday) at randomization
2. Diagnosis of multiple sclerosis
3. EDSS score of 0 to 5.5, inclusive
4. At least one MS relapse/attack during the previous year or two MS relapses in the previous two years prior or evidence of one or more new T2 lesions within 12 months

Exclusion Criteria:

1. Participants with progressive MS
2. Participants with an active, chronic disease of the immune system other than MS
3. Participants meeting the definition of ADEM
4. Participants with severe cardiac disease or significant findings on the screening ECG.
5. Participants with severe renal insufficiency

Trial Locations

• Arkansas Childrens Hosp Rsch Inst, Little Rock, Arkansas, United States
• Childrens Hospital Los Angeles, Los Angeles, California, United States
• Childrens National Medical Center, Washington D.C., District of Columbia, United States
• Axiom Clinical Research of Florida, Tampa, Florida, United States
• Childrens Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
• Medical College of Wisconsin, Milwaukee, Wisconsin, United States
• Novartis Investigative Site, CABA, Argentina
• Novartis Investigative Site, Parkville, Victoria, Australia
• Novartis Investigative Site, Vienna, Austria
• Novartis Investigative Site, Esneux, Belgium
• ...and 10 more locations

Frequently Asked Questions

Related Conditions

• Multiple Sclerosis (MS)
• Pediatric Neurology
• Autoimmune Diseases
• Neurological Disorders
• Inflammatory Diseases
• Central Nervous System (CNS) Diseases
• Immunomodulatory Therapy
• Relapsing-Remitting MS
• Pediatric Autoimmune Diseases
• Neuroinflammation

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