Efficacy and safety of ofatumumab and siponimod compared to fingolimod in pediatric patients with multiple sclerosis
- Conditions
- Multiple Sclerosis in pediatric patientsMedDRA version: 20.1Level: PTClassification code: 10028245Term: Multiple sclerosis Class: 100000004852Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- CTIS2024-511686-11-00
- Lead Sponsor
- ovartis Pharma AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 147
Signed informed consent/assent must be obtained prior to participation in the study, Between 10 to <18 years of age (i.e., have not yet had their 18th birthday) at randomization, A diagnosis of MS as defined by the consensus definition for pediatric MS, Expanded Disability Status Scale (EDSS) score of 0 to 5.5 (inclusive) at Screening, At least one MS relapse/attack during the previous year or two MS relapses in the previous two years prior to screening or evidence of one or more new T2 lesions compared to prior MRI conducted within 12 months prior to randomization (including screening MRI) or one or more Gd-enhancing T1 lesions on MRI conducted within 12 months prior to randomization
Participants with progressive MS, Participants meeting the definition of ADEM •participants meeting criteria for neuromyelitis optica or tested positive for aquaporin 4 (AQP4) at Screening •participants tested positive for anti-MOG at Screening confirmed centrally, Participants with widespread and symmetric white matter alterations in the Screening MRI suggestive of other demyelinating disorders (e.g. metabolic disorders, mitochondrial disorders), Homozygosity for CYP2C9*3, or refusal to test for CYP2C9, Participants with an active, chronic disease (or stable but treated with immune therapy) of the immune system other than MS (e.g. Sjögren’s disease, systemic lupus erythematosus) or with a known immunodeficiency syndrome (acquired immunodeficiency syndrome (AIDS), hereditary immune deficiency, drug-induced immune deficiency) or tested positive for HIV at Screening, Participants with neurological symptoms consistent with progressive multifocal leukoencephalopathy PML or confirmed PML, Participants diagnosed with macular edema during the Screening period, Participants with any other significant condition, as assessed by the investigator, which may preclude participant from participating in the study, Pregnant or nursing (lactating) female participant, Participants with severe active systemic bacterial, viral or fungal infections, including tuberculosis. Treatment initiation should be delayed in participants with an active infection until the infection is resolved, Participants with any severe cardiac disease or significant findings on the screening ECG, Positive results of screening period testing for serological markers for hepatitis A, B, C and E indicating acute or chronic infection, Any other clinically significant laboratory assessment as determined by the Investigator (e.g. significant anemia, neutropenia, thrombocytopenia, signs of impaired bone marrow function, Have received any live or live-attenuated vaccines (including for varicella-zoster virus or measles) within 4 weeks prior to first study drug administration, Participants without acceptable evidence of immunity to varicella-zoster virus, mumps, measles, rubella, diphtheria, tetanus and pertussis at Randomization, Any history of malignancy of any organ system, Participants treated with any of the listed medication as Exclusion Medication within defined timespan
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To demonstrate the non-inferiority of ofatumumab and/or siponimod as compared to fingolimod as assessed by annualized relapse rate (ARR) in the target pediatric MS participants treated for up to 2-years;Secondary Objective: To demonstrate the superiority of ofatumumab and/ or siponimod as compared to historical interferon ß-1a data, assessed by annualized relapse rate (ARR), To evaluate the effects of ofatumumab and/or siponimod versus fingolimod on the number of new or newly enlarging T2 lesions, To evaluate the effects of ofatumumab and/or siponimod versus fingolimod on neurofilament light chain (NfL) concentrations, To evaluate the pharmacokinetic (PK) properties of ofatumumab and siponimod (and its metabolite M17) in pediatric MS patients, To evaluate immunogenicity (ofatumumab), To evaluate the safety and tolerability of ofatumumab and siponimod;Primary end point(s): Annualized relapse rate (ARR of confirmed relapses)
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Annualized relapse rate (ARR of confirmed relapses);Secondary end point(s):Number of new or newly enlarging T2 lesions on MRI per year (annualized T2 lesion rate);Secondary end point(s):Neurofilament light chain (NfL) concentration in serum;Secondary end point(s):Ofatumumab and siponimod and (metabolite M17) plasma concentrations;Secondary end point(s):Proportion of participants with anti-ofatumumab antibodies;Secondary end point(s):Adverse events, Columbia Suicide Severity Rating Scale (C-SSRS), ECG, laboratory and ophthalmological data, pulmonary function tests and vital signs