Multiple Sclerosis in pediatric patients MedDRA version: 20.1 Level: PT Classification code 10028245 Term: Multiple sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Signed informed consent/assent must be obtained prior to participation in the study -Between 10 to =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -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 -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 immunodeficiency, drug-induced immunodeficiency ) 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 severe active systemic bacterial, viral or fungal infections, including tuberculosis -Participants with any severe cardiac disease or significant findings on the screening ECG Any history of malignancy of any organ system -Participants treated with any of the listed medication as Exclusion Medication within defined timespan -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 varicellazoster 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 -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 -other protocol-defined inclusion/exclusion criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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: -Key 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);Timepoint(s) of evaluation of this end point: participants treated up to 2-years | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Annualized relapse rate (ARR of confirmed relapses) -Number of new or newly enlarging T2 lesions on MRI per year (annualized T2 lesion rate) -Neurofilament light chain (NfL) concentration in serum -Ofatumumab and siponimod and(metabolite M17) plasma concentrations -Proportion of participants with antiofatumumab antibodies -Adverse events, Columbia Suicide Severity Rating Scale (C-SSRS), ECG, laboratory and ophthalmological data, pulmonary function tests and vital signs ;Timepoint(s) of evaluation of this end point: For the key secondary endpoint ARR, the endpoint is for participants treated up to 2-years. Information for the timepoints for other secondary endpoints can be found in the study protocol | — |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, Croatia, Czechia, Estonia, France, Germany, Guatemala, India, Israel, Italy, Latvia, Lithuania, Mexico, Poland, Portugal, Romania, Russian Federation, Serbia, Slovakia, Spain, Taiwan, Türkiye, Ukraine, United Kingdom, United States
Contacts
Public ContactMedizinischer Infoservice (MCC)
Novartis Pharma GmbH
Outcome results
None listed