Clinical trial • Phase III • Neurology|Rare Disease

satralizumab for Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)

Phase III trial of satralizumab for Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD).

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
07-12-2023
First CTIS Authorization Date
31-01-2024

Trial design

Randomised, placebo (matching) - comparator arm is placebo; dose/schedule not specified in provided data-controlled Phase III trial in Italy, Germany, France and others.

Randomised
Yes
Comparator
Placebo (matching) - comparator arm is placebo; dose/schedule not specified in provided data
Target Sample Size
118

Eligibility

Recruits 118 paediatric patients.

Pregnancy Exclusion
6. For women of childbearing potential: participants who agree to remain abstinent or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab
Vulnerable Population
Vulnerable populations are selected (isVulnerablePopulationSelected: true). The trial includes adolescents (documents include assent and adolescent ICF: e.g. "L1_SIS and ICF Assent_12-17 yr", "L1_SIS and ICF Main adolescent") and infant/parent forms (e.g. "L1_SIS and ICF infant form", parental consent forms). Consent/assent handling documented with age-appropriate informed consent and assent forms and parent/guardian consent forms for minors.

Inclusion criteria

  • {"criterion_text":"- 1. Participants who are aged >=12 years at the time of signing Informed Consent Form and confirmed diagnosis of MOGAD with a history of >=1 MOGAD relapse in the 12 months prior to screening or >=2 attacks in the 24 months prior to screening\n- 2. Expanded Disability Status Scale (EDSS) score of 0-6.5 at screening\n- 3. High-contrast visual acuity (HCVA) better than 20/800 in each eye at screening\n- 4. Participants receiving either no ongoing chronic immunosuppressant treatment (IST) for MOGAD at the time of screening or receiving ongoing treatment with azathioprine (AZA), mycophenolate mofetil (MMF), oral corticosteroids (OCS) or a combination of OCS and AZA or MMF prior to and at the time of screening\n- 5. No contraindications to rescue treatments and no contraindications to MRI\n- 6. For women of childbearing potential: participants who agree to remain abstinent or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab"}

Exclusion criteria

  • {"criterion_text":"- 1. Presence of aquaporin-4-antibodies (AQP4-IgG) in the serum\n- 2. History of anti-N-methyl-d-aspartate receptor (NMDAR) encephalitis\n- 3. Any concomitant disease other than MOGAD that may require treatment with ISTs or OCS or intravenous (IV) corticosteroids at doses >20 mg prednisone equivalent per day for >21 days during the study\n- 4. Intravenous immunoglobulins (IVIg) or subcutaneous immunoglobulins ( ScIg) within 4 weeks prior to screening\n- 5. Plasma exchange (PLEX) within 4 weeks prior to screening\n- 6. Systemic corticosteroids, AZA or MMF within 4 weeks prior to screening (if not continued as concomitant IST in the study)"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- 1. Time from randomization to the first occurrence of a MOGAD relapse in the DB treatment period, as determined by an adjudication committee (CEC)","definition_or_measurement_approach":"Time from randomization to first adjudicated MOGAD relapse during the double-blind treatment period; relapse determination by an adjudication committee (CEC)."}

Secondary endpoints

  • {"endpoint_text":"- 1. Rate of adjudicated MOGAD relapses\n- 2. Active lesions on MRI of the neuroaxis (Gd-enhancing or new/enlarging T2 hyperintense lesions measured across the optic nerve, the spinal cord and the brain, including brainstem and cerebellum)\n- 3. Proportion of participants receiving rescue therapy\n- 4. Rate of inpatient hospitalizations (defined as more than an overnight stay, excluding those for elective procedures)\n- 5. Proportion of relapse-free participants at 6-month intervals\n- 6. Change from baseline in total Montreal cognitive assessment (MoCA) score (adolescents only)\n- 7. Incidence, seriousness and severity of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)\n- 8. Change from baseline in targeted vital signs\n- 9. Change from baseline in targeted electrocardiogram (ECG) parameters\n- 10. Change from baseline in targeted clinical laboratory test results\n- 11. Change from baseline in suicidality, as determined by Columbia-Suicide Severity Rating Scale\n- 12. Change from baseline in weight","definition_or_measurement_approach":"1: Rate of adjudicated relapses as determined by adjudication committee. 2: MRI lesions defined as Gd-enhancing or new/enlarging T2 hyperintense lesions measured across optic nerve, spinal cord and brain (including brainstem and cerebellum). 3: Proportion receiving rescue therapy (use captured). 4: Inpatient hospitalizations defined as > overnight stay excluding elective procedures. 5: Relapse-free proportion assessed at 6-month intervals. 6: MoCA change from baseline (adolescents only). 7: AEs graded per NCI CTCAE v5.0. 11: Suicidality assessed by Columbia-Suicide Severity Rating Scale. Other measures are changes from baseline in vitals, ECG, lab results, and weight."}

Recruitment

Planned Sample Size
118
Recruitment Window Months
64
Consent Approach
Age-appropriate informed consent and assent process documented. Adolescent assent forms for 12-17 years (e.g. "L1_SIS and ICF Assent_12-17 yr"), parent/guardian consent forms and infant consent forms are provided (e.g. "L1_SIS and ICF infant form", "L1_SIS and ICF parents"). Documents include dedicated materials for adolescents and parents and specific ICF versions (including OLE materials).

Geography

Total Number Of Sites
24
Total Number Of Participants
73

Italy

Earliest CTIS Part Ii Submission Date
09-01-2024
Latest Decision Or Authorization Date
31-01-2024
Processing Time Days
22
Number Of Sites
7
Number Of Participants
22

Sites

Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
Neuroscienze cliniche Neuroimmunologia e Malattie Neuromuscolari
Principal Investigator Name
Carlo Antozzi
Principal Investigator Email
carlo.antozzi@istituto-besta.it
Contact Person Name
Carlo Antozzi
Site Name
Bambino Gesu Childrens Hospital
Department Name
Neurologia
Principal Investigator Name
Massimiliano Valeriani
Principal Investigator Email
massimiliano.valeriani@opbg.net
Contact Person Name
Massimiliano Valeriani
Site Name
Azienda Ospedaliero-Universitaria San Luigi Gonzaga
Department Name
Neurologia II
Principal Investigator Name
Alessia Di Sapio
Principal Investigator Email
a.disapio@sanluigi.piemonte.it
Contact Person Name
Alessia Di Sapio
Contact Person Email
a.disapio@sanluigi.piemonte.it
Site Name
Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania
Department Name
Clinica Neurologica, Centro Sclerosi Multipla
Principal Investigator Name
Francesco Patti
Principal Investigator Email
patti@unict.it
Contact Person Name
Francesco Patti
Contact Person Email
patti@unict.it
Site Name
Azienda Ospedaliera Universitaria Integrata Verona
Department Name
Neuroscienze Biomedicina e Movimento
Principal Investigator Name
Sara Mariotto
Principal Investigator Email
sara.mariotto@aovr.veneto.it
Contact Person Name
Sara Mariotto
Contact Person Email
sara.mariotto@aovr.veneto.it
Site Name
Ospedale San Raffaele S.r.l.
Department Name
Neurologia Neurofisiologia Neuroriabilitazione-Centro Sclerosi Multipla
Principal Investigator Name
Massimo Filippi
Principal Investigator Email
filippi.massimo@hsr.it
Contact Person Name
Massimo Filippi
Contact Person Email
filippi.massimo@hsr.it
Site Name
Neurological Institute Foundation Casimiro Mondino
Department Name
S.C. Neuroncologia
Principal Investigator Name
Matteo Gastaldi
Principal Investigator Email
matteo.gastaldi@mondino.it
Contact Person Name
Matteo Gastaldi
Contact Person Email
matteo.gastaldi@mondino.it

Germany

Earliest CTIS Part Ii Submission Date
09-01-2024
Latest Decision Or Authorization Date
31-01-2024
Processing Time Days
22
Number Of Sites
7
Number Of Participants
22

Sites

Site Name
Universitaetsklinikum Duesseldorf AöR
Department Name
Klinik fuer Neurologie
Principal Investigator Name
Orhan Aktas
Principal Investigator Email
orhan.aktas@med.uni-duesseldorf.de
Contact Person Name
Orhan Aktas
Site Name
Universitaetsklinikum Mannheim GmbH
Department Name
Neurologische Klinik
Principal Investigator Name
Lucas Schirmer
Principal Investigator Email
lucas.schirmer@medma.uni-heidelberg.de
Contact Person Name
Lucas Schirmer
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
NCRC, Clinical Neuroimmunology
Principal Investigator Name
Tatiana Usnich
Principal Investigator Email
tatiana.usnich@charite.de
Contact Person Name
Tatiana Usnich
Contact Person Email
tatiana.usnich@charite.de
Site Name
Vestische Kinder- und Jugendklinik Datteln
Department Name
Neuropaediatrie
Principal Investigator Name
Kevin Rostasy
Principal Investigator Email
k.rostasy@kinderklinik-datteln.de
Contact Person Name
Kevin Rostasy
Site Name
Universitaetsklinikum Schleswig-Holstein
Department Name
Klinik fuer Neurologie
Principal Investigator Name
Frank Leypoldt
Principal Investigator Email
frank.leypoldt@uksh.de
Contact Person Name
Frank Leypoldt
Contact Person Email
frank.leypoldt@uksh.de
Site Name
Katholisches Klinikum Bochum gGmbH
Department Name
Klinik fuer Neurologie
Principal Investigator Name
Ilya Ayzenberg
Principal Investigator Email
ilya.ayzenberg@rub.de
Contact Person Name
Ilya Ayzenberg
Contact Person Email
ilya.ayzenberg@rub.de
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Institute of Clinical Neuroimmunology
Principal Investigator Name
Tania Kuempfel
Principal Investigator Email
Tania.Kuempfel@med.uni-muenchen.de
Contact Person Name
Tania Kuempfel

France

Earliest CTIS Part Ii Submission Date
09-01-2024
Latest Decision Or Authorization Date
08-03-2024
Processing Time Days
59
Number Of Sites
3
Number Of Participants
11

Sites

Site Name
Bicetre Hospital
Department Name
Neurologie Pédiatrique
Principal Investigator Name
Kumaran DEIVA
Principal Investigator Email
kumaran.deiva@aphp.fr
Contact Person Name
Kumaran DEIVA
Contact Person Email
kumaran.deiva@aphp.fr
Site Name
Hospital Pierre Wertheimer
Department Name
Neurologie
Principal Investigator Name
Romain MARIGNIER
Principal Investigator Email
romain.marignier@chu-lyon.fr
Contact Person Name
Romain MARIGNIER
Contact Person Email
romain.marignier@chu-lyon.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Neurologie
Principal Investigator Name
Jonathan CIRON
Principal Investigator Email
ciron.j@chu-toulouse.fr
Contact Person Name
Jonathan CIRON
Contact Person Email
ciron.j@chu-toulouse.fr

Poland

Earliest CTIS Part Ii Submission Date
26-03-2025
Latest Decision Or Authorization Date
04-05-2025
Processing Time Days
39
Number Of Sites
7
Number Of Participants
18

Sites

Site Name
Instytut Pomnik Centrum Zdrowia Dziecka
Department Name
Klinika Neurologii i Epileptologii
Principal Investigator Name
Katarzyna Kotulska-Jóźwiak
Principal Investigator Email
oddzial.neurologia@ipczd.pl
Contact Person Name
Katarzyna Kotulska-Jóźwiak
Contact Person Email
oddzial.neurologia@ipczd.pl
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Zespół Poradni Specjalistycznych - Botaniczna 3
Principal Investigator Name
Agnieszka Słowik
Principal Investigator Email
neurologiabk@su.krakow.pl
Contact Person Name
Agnieszka Słowik
Contact Person Email
neurologiabk@su.krakow.pl
Site Name
Niepubliczny Zaklad Opieki Zdrowotnej Wielospecjalistyczna Poradnia Lekarska Synapsis Lech Szczechowski
Principal Investigator Name
Lech Szczechowski
Principal Investigator Email
lszczechowski@gmail.com
Contact Person Name
Lech Szczechowski
Contact Person Email
lszczechowski@gmail.com
Site Name
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Department Name
Klinika Neurologii
Principal Investigator Name
Beata Zakrzewska-Pniewska
Principal Investigator Email
kn.csk@uckwum.pl
Contact Person Name
Beata Zakrzewska-Pniewska
Contact Person Email
kn.csk@uckwum.pl
Site Name
Samodzielny Publiczny Szpital Kliniczny Nr 1 Im.Prof.Stanislawa Szyszko Slaskiego Uniwersytetu Medycznego W Katowicach
Department Name
Oddział Neurologiczny
Principal Investigator Name
Monika Adamczyk-Sowa
Principal Investigator Email
neurozab@sum.edu.pl
Contact Person Name
Monika Adamczyk-Sowa
Contact Person Email
neurozab@sum.edu.pl
Site Name
Centrum Medyczne Medyk Sp. z o.o. S.K.
Principal Investigator Name
Halina Bartosik-Psujek
Principal Investigator Email
badaniakliniczne@medyk.rzeszow.pl
Contact Person Name
Halina Bartosik-Psujek
Site Name
Neurocentrum Bydgoszcz Sp. z o.o.
Principal Investigator Name
Robert Bonek
Principal Investigator Email
wyniki@ncbydgoszcz.pl
Contact Person Name
Robert Bonek
Contact Person Email
wyniki@ncbydgoszcz.pl

Sponsor

Primary sponsor

Full Name
F. Hoffmann-La Roche AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
IQVIA Limited
Responsibilities
Monitoring
Name
Ixico Technologies Limited
Responsibilities
Medical image analysis/review - MRI, Ultrasound, etc
Name
Signant Health Global LLC
Responsibilities
Primary/Surrogate Endpoint Test, Centralized Adjudication of Primary Endpoint, Management of clinical outcome assessments instruments
Name
Labcorp Central Laboratory Services S.a.r.l.
Responsibilities
Provision of Kits, Logistics Services, Clinical Haematology, Clinical Chemistry

Third parties

  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Monitoring","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Ixico Technologies Limited","duties_or_roles":"Medical image analysis/review - MRI, Ultrasound, etc","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"Primary/Surrogate Endpoint Test, Centralized Adjudication of Primary Endpoint, Management of clinical outcome assessments instruments","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"Provision of Kits, Logistics Services, Clinical Haematology, Clinical Chemistry","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Satralizumab (RO 533-3787/F01)
Active Substance
satralizumab
Modality
Monoclonal antibody
Authorisation Status
EU/1/21/1559/001 (marketing authorisation present in product information)
Orphan Designation
Yes
Investigational Product Name
RO 533-3787/F01-06 (satralizumab product variant)
Active Substance
satralizumab
Modality
Monoclonal antibody
Authorisation Status
PRD10948861 (product record present; prodAuthStatus indicated)
Combination Treatment
Yes

Related trials

Other published trials that may interest you.