Clinical trial • Phase III • Neurology
SIPONIMOD FUMARIC ACID for Multiple sclerosis
Phase III trial of SIPONIMOD FUMARIC ACID for Multiple sclerosis.
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Multiple sclerosis
- Trial Stage
- Phase III
- Drug Modality
- Small molecule|Monoclonal antibody
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 30-05-2024
- First CTIS Authorization Date
- 09-07-2024
Trial design
Randomised, open-label, fingolimod hydrochloride 0.5 mg oral (comparator arm; max daily dose 0.5 mg as listed)-controlled Phase III trial in Romania, Austria, Italy and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Fingolimod hydrochloride 0.5 mg oral (comparator arm; max daily dose 0.5 mg as listed)
- Target Sample Size
- 77
- Trial Duration For Participant
- 730
Eligibility
Recruits 77 paediatric patients.
- Pregnancy Exclusion
- Pregnant or nursing (breastfeeding) female participant
- Vulnerable Population
- Pediatric population (participants aged 10 to <18 years). Signed informed consent/assent must be obtained prior to participation. Parent/legal guardian consent forms and child/adolescent assent forms are used (separate forms for parent/legal guardian, child assent and adolescent assent are documented). Multiple country-specific ICF and assent documents are available in local languages per country-specific submissions.
Inclusion criteria
- {"criterion_text":"- Signed informed consent/assent must be obtained prior to participation in the study\n- Between 10 to <18 years of age (i.e., have not yet had their 18th birthday) at randomization\n- A diagnosis of MS as defined by the consensus definition for pediatric MS\n- Expanded Disability Status Scale (EDSS) score of 0 to 5.5 (inclusive) at Screening\n- 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"}
Exclusion criteria
- {"criterion_text":"- Participants with progressive MS\n- Participants meeting the definition of ADEM •\tparticipants meeting criteria for neuromyelitis optica or tested positive for aquaporin 4 (AQP4) at Screening •\tparticipants tested positive for anti-MOG at Screening confirmed centrally\n- Participants with widespread and symmetric white matter alterations in the Screening MRI suggestive of other demyelinating disorders (e.g. metabolic disorders, mitochondrial disorders)\n- Homozygosity for CYP2C9*3, or refusal to test for CYP2C9\n- 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\n- Participants with neurological symptoms consistent with progressive multifocal leukoencephalopathy PML or confirmed PML\n- Participants diagnosed with macular edema during the Screening period\n- Participants with any other significant condition, as assessed by the investigator, which may preclude participant from participating in the study\n- Pregnant or nursing (breastfeeding) female participant\n- 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\n- Participants with any severe cardiac disease or significant findings during screening, or on the screening ECG\n- Positive results of screening period testing for serological markers for hepatitis A, B, C and E indicating acute or chronic infection\n- Any other clinically significant laboratory assessment as determined by the Investigator (e.g. significant anemia, neutropenia, thrombocytopenia, signs of impaired bone marrow function\n- Have received any live or live-attenuated vaccines (including for varicella-zoster virus or measles) within 4 weeks prior to first study drug administration\n- Participants without acceptable evidence of immunity to varicella-zoster virus, mumps, measles, rubella, diphtheria, tetanus and pertussis at Randomization\n- Any history of malignancy of any organ system\n- Participants treated with any of the listed medication as Exclusion Medication within defined timespan"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Annualized relapse rate (ARR of confirmed relapses)","definition_or_measurement_approach":"Assessed by annualized relapse rate (ARR) of confirmed relapses"}
Secondary endpoints
- {"endpoint_text":"- Annualized relapse rate (ARR of confirmed relapses)","definition_or_measurement_approach":"Assessed by annualized relapse rate (ARR) of confirmed relapses"}
- {"endpoint_text":"- Number of new or newly enlarging T2 lesions on MRI per year (annualized T2 lesion rate)","definition_or_measurement_approach":"Count of new or newly enlarging T2 lesions on MRI per year (annualized T2 lesion rate)"}
- {"endpoint_text":"- Neurofilament light chain (NfL) concentration in serum","definition_or_measurement_approach":"Measurement of serum neurofilament light chain (NfL) concentration"}
- {"endpoint_text":"- Ofatumumab and siponimod and (metabolite M17) plasma concentrations","definition_or_measurement_approach":"Plasma concentration measurements of ofatumumab, siponimod and metabolite M17"}
- {"endpoint_text":"- Proportion of participants with anti-ofatumumab antibodies","definition_or_measurement_approach":"Assessment of presence of anti-ofatumumab antibodies in participants"}
- {"endpoint_text":"- Adverse events, Columbia Suicide Severity Rating Scale (C-SSRS), ECG, laboratory and ophthalmological data, pulmonary function tests and vital signs","definition_or_measurement_approach":"Safety assessments including adverse event reporting, C-SSRS, ECGs, laboratory tests, ophthalmological evaluations, pulmonary function tests and vital signs"}
Recruitment
- Planned Sample Size
- 77
- Recruitment Window Months
- 121
- Consent Approach
- Signed informed consent and assent required prior to participation. Parent/legal guardian consent forms and child/adolescent assent forms are specified (separate Parent Legal Guardian, Child Assent, Adolescent Assent documents). Country-specific ICF/assent documents are provided in local languages as part of the submissions.
Geography
- Total Number Of Sites
- 26
- Total Number Of Participants
- 64
Romania
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 11-07-2024
- Processing Time Days
- 20
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Spitalul Clinic De Psihiatrie Prof.Dr.Alexandru Obregia
- Department Name
- 3021: Pediatric Neurology
- Contact Person Name
- Dana Cristina Craiu
- Contact Person Email
- dcraiu@yahoo.com
Austria
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 15-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 1
- Number Of Participants
- 4
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- 1011 : Deaprtment Neurology
- Contact Person Name
- Barbara Kornek
- Contact Person Email
- Barbara.kornek@meduniwien.ac.at
Italy
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 15-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- 2413: Dip. di Neuroscienze, Scienze riproduttive ed odontostomatologiche- Centro Sclerosi multipla
- Contact Person Name
- Vincenzo Brescia Morra
- Contact Person Email
- Vincenzo.bresciamorra2@unina.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- 2412: U.O Neurologia
- Contact Person Name
- Laura Papetti
- Contact Person Email
- laura.papetti@opbg.net
Slovakia
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 09-07-2024
- Processing Time Days
- 18
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Narodny Ustav Detskych Chorob
- Department Name
- 3300: Klinika detskej neurológie LFUK a NÚDCH
- Contact Person Name
- Miriam Kolníková
- Contact Person Email
- Miriam.Kolnikova@nudch.eu
Poland
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 18-07-2024
- Processing Time Days
- 27
- Number Of Sites
- 4
- Number Of Participants
- 8
Sites
- Site Name
- Instytut Centrum Zdrowia Matki Polki
- Department Name
- 2812: Klinika Neurologii Rozwojowej i Epileptologii
- Contact Person Name
- Katarzyna Połatyńska
- Contact Person Email
- k.polatynska@gmail.com
- Site Name
- Instytut Pomnik Centrum Zdrowia Dziecka
- Department Name
- 2813: Klinika Neurologii i Epiletoptologii
- Contact Person Name
- Katarzyna Kotulska-Jóźwiak
- Contact Person Email
- k.kotulska@ipczd.pl
- Site Name
- Uniwersytecki Szpital Kliniczny W Poznaniu
- Department Name
- 2810: Oddział Kliniczny Neurologii Dzieci i Młodzieży
- Contact Person Name
- Barbara Steinborn
- Contact Person Email
- bstein@ump.edu.pl
- Site Name
- Uniwersyteckie Centrum Kliniczne
- Department Name
- 2811: Klinika Neurologii Rozwojowej
- Contact Person Name
- Maria Mazurkiewicz-Bełdzińska
- Contact Person Email
- mmazur@gumed.edu.pl
Belgium
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 09-07-2024
- Processing Time Days
- 18
- Number Of Sites
- 2
- Number Of Participants
- 7
Sites
- Site Name
- Centre hospitalier universitaire de Liege
- Department Name
- 1141 : Neurology
- Contact Person Name
- Dominique Dive
- Contact Person Email
- Dominique.dive@chu.ulg.ac.be
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- 1140 : Pediatry
- Contact Person Name
- Hélène Verhelst
- Contact Person Email
- helene.verhelst@ugent.be
Latvia
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 09-07-2024
- Processing Time Days
- 18
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Bernu Kliniska Universitates Slimnica VSIA
- Department Name
- 2510: Neurology Department
- Contact Person Name
- Dace Pretkalnina
- Contact Person Email
- dace.pretkalnina@bkus.lv
Portugal
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 10-07-2024
- Processing Time Days
- 19
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Unidade Local De Saude De Coimbra E.P.E.
- Department Name
- 2910: Centro de Desenvolvimento da Criança
- Contact Person Name
- Filipe Palavra
- Contact Person Email
- uicc@ulscoimbra.min-saude.pt
- Site Name
- Unidade Local De Saude De Sao Jose E.P.E.
- Department Name
- 2911: Neurologia
- Contact Person Name
- Rita Silva
- Contact Person Email
- rita.silva@chlc.min-saude.pt
France
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 11-07-2024
- Processing Time Days
- 20
- Number Of Sites
- 3
- Number Of Participants
- 13
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- 1840: Neurologie Pédiatrie
- Contact Person Name
- Kumaran Deiva
- Contact Person Email
- kumaran.deiva@aphp.fr
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- 1841: Neurologie
- Contact Person Name
- Jerome De Seze
- Contact Person Email
- Jerome.DESEZE@chru-strasbourg.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- 1842: Neuropediatrie
- Contact Person Name
- Pierre Meyer
- Contact Person Email
- p-meyer@chu-montpellier.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 12-07-2024
- Processing Time Days
- 21
- Number Of Sites
- 3
- Number Of Participants
- 10
Sites
- Site Name
- Medical Center - University Of Freiburg
- Department Name
- 1925 : Neuropädiatrie und Muskelerkrankungen
- Contact Person Name
- Matthias Eckenweiler
- Contact Person Email
- matthias.eckenweiler@uniklinik-freiburg.de
- Site Name
- Katholisches Klinikum Bochum gGmbH
- Department Name
- 1921: Kinder und Jugendmedizin
- Contact Person Name
- Thomas Luecke
- Contact Person Email
- thomas.luecke@klinikum-bochum.de
- Site Name
- Universitaetsmedizin Goettingen
- Department Name
- 1920: Kinder und Jugendmedizin/Neuropädiatrie
- Contact Person Name
- Jutta Gaertner
- Contact Person Email
- kinderklinik@med.uni-goettingen.de
Spain
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 10-07-2024
- Processing Time Days
- 19
- Number Of Sites
- 4
- Number Of Participants
- 2
Sites
- Site Name
- Hospital Universitario Ramon Y Cajal
- Department Name
- 3404: Neurología
- Contact Person Name
- Lucienne Costa Frossard Franca
- Contact Person Email
- lufrossard@yahoo.es
- Site Name
- Hospital Universitario Central De Asturias
- Department Name
- 3403: Neurología pediátrica
- Contact Person Name
- Ignacio Malaga Dieguez
- Contact Person Email
- Ignacio.MalagaDieguez@UTSouthwestern.edu
- Site Name
- Hospital Universitario De Cruces
- Department Name
- 3401: Neurología
- Contact Person Name
- Maria del Mar Mendibe Bilbao
- Contact Person Email
- mariadelmar.mendibebilbao@osakidetza.eus
- Site Name
- Hospital Universitario Virgen De La Macarena
- Department Name
- 3402: Neurología
- Contact Person Name
- Sara Eichau Madueno
- Contact Person Email
- saraeichau@gmail.com
Croatia
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 09-07-2024
- Processing Time Days
- 18
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Children's Hospital Zagreb
- Department Name
- 1540: Pediatry (pediatric neurology)
- Contact Person Name
- Jadranka Sekelj Fures
- Contact Person Email
- jadrankasekelj@gmail.com
Estonia
- Earliest CTIS Part Ii Submission Date
- 21-06-2024
- Latest Decision Or Authorization Date
- 15-07-2024
- Processing Time Days
- 24
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- Clinic4U OÜ
- Department Name
- 1721: Neurology
- Contact Person Name
- Katrin Gross-Paju
- Contact Person Email
- katrin.gross-paju@astrakliinik.ee
Sponsor
Primary sponsor
- Full Name
- Novartis Pharma AG
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Parexel International (IRL) Limited
- Responsibilities
- code:12
- Name
- IQVIA Limited
- Responsibilities
- Management of drug supply logistics, dispensing and unblinding
- Name
- Icon Laboratory Services Inc.
- Responsibilities
- Genotyping sample analysis
- Name
- Syneos Health Inc.
- Responsibilities
- code:1
- Name
- Medidata Solutions Inc.
- Responsibilities
- EDC system provider & data storage
- Name
- Labcorp Central Laboratory Services LP
- Responsibilities
- code:4
- Name
- Eresearchtechnology Inc.
- Responsibilities
- Data collection via tablet and hand-held device / Central ECG reading
- Name
- Q Squared Solutions Limited
- Responsibilities
- Central laboratory, clinical chemistry
Third parties
- {"country":"Switzerland","full_name":"Universitaetsspital Basel","duties_or_roles":"EDSS expert consultancy, Neurostatus EDSS rater certification","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"code:12","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Management of drug supply logistics, dispensing and unblinding","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"Genotyping sample analysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Austria","full_name":"Mag. Andreas Raffeiner GmbH","duties_or_roles":"code:8","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Opis S.r.l.","duties_or_roles":"TMF archive Activation sites activities","organisation_type":"Pharmaceutical company"}
- {"country":"Latvia","full_name":"Oribalt Riga SIA","duties_or_roles":"Collection and destruction of the investigational medicinal products","organisation_type":"Pharmaceutical company"}
- {"country":"Poland","full_name":"Eco-Abc Sp. z o. o.","duties_or_roles":"Destruction of the investigational medicinal products","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"Central laboratory, clinical chemistry","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Croatia","full_name":"Medical Intertrade d.o.o.","duties_or_roles":"Obtain license, import study drugs and medical equip, storage&Logistics, support supplies, return&destruction","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Patients","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Romania","full_name":"Alliance Healthcare Romania S.R.L.","duties_or_roles":"Supply and delivery of Non-IMP Destruction of IMP and non-IMP","organisation_type":"Pharmaceutical company"}
- {"country":"Canada","full_name":"Neurorx Research Inc.","duties_or_roles":"Medical image analysis, central reading","organisation_type":"Pharmaceutical company"}
- {"country":"Austria","full_name":"Abf Pharmaceutical Services GmbH","duties_or_roles":"Local depot for storage, distribution of AxMP to sites/ final Rec. + Destruction","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"Packaging of study drug","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Alliance Pharma Inc.","duties_or_roles":"Analysis of neutralizing antibodies (NAb)","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"code:4","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"Medical equipment supplier, management and maintenance","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Phardis S.r.l.","duties_or_roles":"Local equipment storage","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Data collection via tablet and hand-held device","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"EDC system provider & data storage","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"DATAMAP-Gesellschaft fuer Datenmanagement Datenanalyse und Datenpraesentation mbH","duties_or_roles":"Programming supporting DMC","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Latvia","full_name":"Oribalt Riga SIA","duties_or_roles":"IMP destruction","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central ECG reading","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"SGS France","duties_or_roles":"Pharmacokinetics (PK) analyses for: BAF312 and FTY720","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"code:1","organisation_type":"Pharmaceutical company"}
- {"country":"Slovakia","full_name":"Movianto Slovensko s.r.o.","duties_or_roles":"Destruction and storage of returned study medication","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- BAF312
- Active Substance
- SIPONIMOD FUMARIC ACID
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- prodAuthStatus:1
- Frequency
- daily
- Maximum Dose
- 2 mg
- Investigational Product Name
- OFATUMUMAB
- Active Substance
- OFATUMUMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- SUBCUTANEOUS USE
- Route
- SUBCUTANEOUS
- Authorisation Status
- prodAuthStatus:2
- Maximum Dose
- 20 mg
- Investigational Product Name
- FINGOLIMOD HYDROCHLORIDE
- Active Substance
- FINGOLIMOD HYDROCHLORIDE
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Authorisation Status
- prodAuthStatus:2
- Frequency
- daily
- Maximum Dose
- 0.5 mg
- Investigational Product Name
- Placebo to Ofatumumab (OMB157) [Kesimpta]
- Modality
- Other
- Investigational Product Name
- Placebo to Siponimod (BAF312) [Mayzent] film-coated tablets
- Modality
- Other
- Investigational Product Name
- Placebo to Fingolimod (FTY720) [Gilenya] 0.25mg & 0.5mg
- Modality
- Other
Related trials
Other published trials that may interest you.