Clinical trial • Phase III • Neurology
satralizumab for Anti-NMDAR encephalitis | Anti-LGI1 encephalitis
Phase III trial of satralizumab for Anti-NMDAR encephalitis | Anti-LGI1 encephalitis.
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Anti-NMDAR encephalitis | Anti-LGI1 encephalitis
- Trial Stage
- Phase III
- Drug Modality
- Monoclonal antibody
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 31-07-2024
- First CTIS Authorization Date
- 06-09-2024
Trial design
Randomised, placebo (matching comparator); no dose or schedule specified in provided data-controlled Phase III trial in Austria, France, Netherlands and others.
- Randomised
- Yes
- Comparator
- Placebo (matching comparator); no dose or schedule specified in provided data
- Biomarker Stratified
- True, anti-NMDAR and anti-LGI1 antibody cohorts
- Target Sample Size
- 134
- Trial Duration For Participant
- 364
Eligibility
Recruits 134 paediatric patients.
- Vulnerable Population
- Vulnerable populations are included (isVulnerablePopulationSelected = true). Age-specific and caregiver documents are provided: infant, adolescent, and parent/caregiver consent forms and 'adult representative' forms are present (eg. 'L1_SISandICF_Infant', 'L1_ICF Main 12-16 years', 'WN43174_Asentimiento 12 a 17 anos', 'L1_SISandICF_Main_parents', 'L1_SISandICF_adult representative'). Consent is obtained from adult participants or from parent/legal guardian for minors; age-appropriate assent is documented for adolescents (assent forms for 12-17 / 12-16 / 15-17 present).
Inclusion criteria
- {"criterion_text":"- Reasonable exclusion of tumor or malignancy before baseline visit (randomization)\n- Onset of autoimmune encephalitis (AIE) symptoms <=9 months before randomization\n- Meet the definition of \"New Onset\" or \"Incomplete Responder\" AIE\n- Diagnosis of probable or definite NMDAR encephalitis\n- Diagnosis of LGI1 encephalitis"}
Exclusion criteria
- {"criterion_text":"- Any untreated teratoma or thymoma at baseline visit (randomization)\n- History of carcinoma or malignancy, unless deemed cured by adequate treatment with no evidence of recurrence for >=5 years before screening\n- For patients with NMDAR AIE, history of negative anti-NMDAR antibody in cerebrospinal fluid (CSF) using a cell-based assay within 9 months of symptom onset\n- Historically known positivity to an intracellular antigen with high cancer association or GAD65\n- Historically known positivity to any cell surface neuronal antibodies other than NMDAR and LGI1, in the absence of NMDAR and LGI1 antibody positivity\n- Confirmed paraneoplastic encephalitis"}
Endpoints
Primary endpoints
- {"endpoint_text":"- 1. NMDAR AIE cohort: Proportion of participants with mRS score improvement >=1 from baseline and no use of rescue therapy at Week 24","definition_or_measurement_approach":"Proportion of participants with a ≥1-point improvement in the Modified Rankin Scale (mRS) from baseline and no use of rescue therapy assessed at Week 24 (NMDAR cohort)."}
- {"endpoint_text":"- 2. LGI1 AIE cohort: Proportion of participants with mRS score improvement >=1 from baseline and no use of rescue therapy at week 52","definition_or_measurement_approach":"Proportion of participants with a ≥1-point improvement in the Modified Rankin Scale (mRS) from baseline and no use of rescue therapy assessed at Week 52 (LGI1 cohort)."}
Secondary endpoints
- {"endpoint_text":"- 1. NMDAR AIE cohort: Time to mRS score improvement >=1 from baseline without use of rescue therapy","definition_or_measurement_approach":"Time from randomization to first occurrence of ≥1-point mRS improvement without subsequent use of rescue therapy (NMDAR cohort)."}
- {"endpoint_text":"- 2. NMDAR AIE cohort: Time to rescue therapy","definition_or_measurement_approach":"Time from randomization to initiation of rescue therapy (NMDAR cohort)."}
- {"endpoint_text":"- 3. NMDAR AIE cohort: Proportion of participants with sustained seizure cessation at Week 24, as defined by 4 consecutive weeks of no seizures maintained until Week 24, and no use of rescue therapy","definition_or_measurement_approach":"Proportion achieving sustained seizure cessation defined as 4 consecutive weeks with no seizures maintained through Week 24 and no rescue therapy use."}
- {"endpoint_text":"- 4. NMDAR AIE cohort: Change in CASE score from baseline at Week 24","definition_or_measurement_approach":"Change from baseline in the Clinical Assessment Scale in Autoimmune Encephalitis (CASE) at Week 24."}
- {"endpoint_text":"- 5. NMDAR AIE cohort: MOCA total score at Week 24","definition_or_measurement_approach":"Montreal Cognitive Assessment (MoCA) total score measured at Week 24."}
- {"endpoint_text":"- 6 . NMDAR AIE cohort: mRS score at Week 24 (as measured on a 7-point scale","definition_or_measurement_approach":"mRS score measured on a 7-point scale at Week 24 (NMDAR cohort)."}
- {"endpoint_text":"- 7. LGI1 AIE cohort: Time to mRS score improvement >=1 from baseline without use of rescue therapy","definition_or_measurement_approach":"Time from randomization to first ≥1-point mRS improvement without rescue therapy (LGI1 cohort)."}
- {"endpoint_text":"- 8. LGI1 AIE cohort: Time to rescue therapy","definition_or_measurement_approach":"Time from randomization to initiation of rescue therapy (LGI1 cohort)."}
- {"endpoint_text":"- 9. LGI1 AIE cohort: Proportion of participants with sustained seizure cessation at Week 24, as defined by 4 consecutive weeks of no seizures maintained until Week 24, and no use of rescue therapy","definition_or_measurement_approach":"Proportion with sustained seizure cessation defined as 4 consecutive weeks without seizures maintained to Week 24 and no rescue therapy use (LGI1 cohort)."}
- {"endpoint_text":"- 10. LGI1 AIE cohort: Change in CASE score from baseline at Week 52","definition_or_measurement_approach":"Change from baseline in CASE score at Week 52 (LGI1 cohort)."}
- {"endpoint_text":"- 11. LGI1 AIE cohort: MoCA total score at Week 52","definition_or_measurement_approach":"MoCA total score measured at Week 52 (LGI1 cohort)."}
- {"endpoint_text":"- 12. LGI1 AIE cohort: RAVLT score at Week 52","definition_or_measurement_approach":"Rey Auditory Verbal Learning Test (RAVLT) score measured at Week 52 (LGI1 cohort)."}
- {"endpoint_text":"- 13. Incidence, seriousness, and severity of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0","definition_or_measurement_approach":"Adverse events assessed for incidence, seriousness and severity; severity graded per NCI CTCAE v5.0."}
- {"endpoint_text":"- 14 . Change from baseline in targeted vital signs, clinical laboratory test results, electrocardiogram (ECG) results, weight, height (<18 years only), and Columbia-Suicide Severity Rating Scale (C-SSRS)","definition_or_measurement_approach":"Change from baseline in selected vital signs, labs, ECG results, weight, height (for participants <18 years only) and C-SSRS assessments."}
Recruitment
- Planned Sample Size
- 134
- Recruitment Window Months
- 55
- Consent Approach
- Informed consent obtained from adult participants or from parent/legal guardian for minors. Age-appropriate assent is used for adolescents (assent documents for 12-16 / 12-17 / 15-17 years are included). Specific caregiver/parent information and consent forms and 'adult representative' forms are provided. Patient information and consent documents exist in multiple languages (English, French, Dutch, Polish, Spanish, German (de-at), Italian, Czech) as evidenced by multiple language versions of SIS/ICF and patient-facing documents.
Geography
- Total Number Of Sites
- 23
- Total Number Of Participants
- 47
Austria
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 08-09-2024
- Processing Time Days
- 25
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Johannes Kepler University Linz
- Department Name
- University Clinic for Neurology
- Contact Person Name
- Gabriele Schwarz
- Contact Person Email
- gabriele.schwarz2@kepleruniklinikum.at
- Site Name
- Medical University Of Vienna
- Department Name
- Department of Neurology
- Contact Person Name
- Paulus Stefan Rommer
- Contact Person Email
- neurostudien@meduniwien.ac.at
France
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 09-09-2024
- Processing Time Days
- 26
- Number Of Sites
- 4
- Number Of Participants
- 7
Sites
- Site Name
- Centre Hospitalier Universitaire De Saint Etienne
- Department Name
- Neurology department
- Contact Person Name
- Jean-Philippe CAMDESSANCHE
- Contact Person Email
- j.philippe.camdessanche@chu-st-etienne.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Neuro-oncology department
- Contact Person Name
- Dimitri PSIMARAS
- Contact Person Email
- dimitri.psimaras@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Neuro-oncology department
- Contact Person Name
- Jerome HONNORAT
- Contact Person Email
- jerome.honnorat@chu-lyon.fr
- Site Name
- Centre Hospitalier Regional Universitaire De Tours
- Department Name
- Neurology and clinical neurophysiology department
- Contact Person Name
- Coline DUWICQUET
- Contact Person Email
- c.duwicquet@chu-tours.fr
Netherlands
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 10-09-2024
- Processing Time Days
- 27
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Neurologie
- Contact Person Name
- Juna de Vries
- Contact Person Email
- trials.neurologie@erasmusmc.nl
Poland
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 05-10-2024
- Processing Time Days
- 52
- Number Of Sites
- 3
- Number Of Participants
- 5
Sites
- Site Name
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
- Department Name
- Oddział Kliniczny Neurologii
- Contact Person Name
- Agnieszka Słowik
- Contact Person Email
- neurologia@su.krakow.pl
- Site Name
- Samodzielny Publiczny Szpital Kliniczny Nr 1 Im.Prof.Stanislawa Szyszko Slaskiego Uniwersytetu Medycznego W Katowicach
- Department Name
- Oddział Neurologiczny
- Contact Person Name
- Monika Adamczyk-Sowa
- Contact Person Email
- sekretariat@szpital.zabrze.pl
- Site Name
- Instytut Psychiatrii I Neurologii
- Department Name
- II Klinika Neurologiczna
- Contact Person Name
- Iwona Kurkowska-Jastrzębska
- Contact Person Email
- neuro2@ipin.edu.pl
Denmark
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 06-09-2024
- Processing Time Days
- 23
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Odense University Hospital
- Department Name
- Neurologisk Afdeling N
- Contact Person Name
- Morten Blaabjerg
- Contact Person Email
- Morten.Blaabjerg1@rsyd.dk
Czechia
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 09-09-2024
- Processing Time Days
- 26
- Number Of Sites
- 2
- Number Of Participants
- 4
Sites
- Site Name
- Fakultni Nemocnice Hradec Kralove
- Department Name
- Neurologická klinika
- Contact Person Name
- Zbyšek Pavelek
- Contact Person Email
- zbysekpavelek@fnhk.cz
- Site Name
- Fakultni Nemocnice V Motole
- Department Name
- Neurologická klinika 2.LF UK a FN Motol
- Contact Person Name
- Martin Elišák
- Contact Person Email
- studie@fnmotol.cz
Spain
- Earliest CTIS Part Ii Submission Date
- 30-06-2025
- Latest Decision Or Authorization Date
- 20-08-2025
- Processing Time Days
- 51
- Number Of Sites
- 3
- Number Of Participants
- 3
Sites
- Site Name
- Hospital Universitario Regional De Malaga
- Department Name
- Neurology
- Contact Person Name
- Pedro Serrano Castro
- Contact Person Email
- pedro.serrano.c@gmail.com
- Site Name
- Hospital General Universitario Gregorio Maranon
- Department Name
- Neurology
- Contact Person Name
- Marisa Martinez Ginés
- Contact Person Email
- marisamgines@hotmail.com
- Site Name
- Hospital Alvaro Cunqueiro
- Department Name
- Neurology
- Contact Person Name
- Inés María González Suárez
- Contact Person Email
- Iines.Maria.Gonzalez.Suarez@sergas.es
Italy
- Earliest CTIS Part Ii Submission Date
- 14-08-2024
- Latest Decision Or Authorization Date
- 17-09-2024
- Processing Time Days
- 34
- Number Of Sites
- 7
- Number Of Participants
- 17
Sites
- Site Name
- Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
- Department Name
- U.O.C. di Neurologia
- Contact Person Name
- Giuseppe Salemi
- Contact Person Email
- giuseppe.salemi@unipa.it
- Site Name
- Ospedale San Raffaele S.r.l.
- Department Name
- Neurologia Neurofisiologia Neuroriabilitazione-Centro Sclerosi Multipla
- Contact Person Name
- Massimo Filippi
- Contact Person Email
- filippi.massimo@hsr.it
- Site Name
- IRCCS Ospedale Policlinico San Martino
- Department Name
- U.O. Clinica Neurologica
- Contact Person Name
- Federico Massa
- Contact Person Email
- federico.massa@hsanmartino.it
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- U.O.C Neurologia 7, Epilettologia Clinica e Sperimentale
- Contact Person Name
- Francesco Deleo
- Contact Person Email
- francesco.deleo@istituto-besta.it
- Site Name
- Azienda Ospedaliera Universitaria Federico II Di Napoli
- Department Name
- Neuroscienze, Scienze Riproduttive e Odontostomatologiche
- Contact Person Name
- Roberta Lanzillo
- Contact Person Email
- roberta.lanzillo@unina.it
- Site Name
- Fondazione Istituto Neurologico Nazionale Casimiro Mondino
- Department Name
- Neuroimmunologia
- Contact Person Name
- Matteo Gastaldi
- Contact Person Email
- matteo.gastaldi@mondino.it
- Site Name
- Azienda Ospedaliera Universitaria Universita' Degli Studi Della Campania Luigi Vanvitelli
- Department Name
- Neurologia I
- Contact Person Name
- Antonio Gallo
- Contact Person Email
- antonio.gallo@unicampania.it
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
- Signant Health Global LLC
- Responsibilities
- Other Third Party Duty
- Name
- Biotrial
- Responsibilities
- Other Third Party Duty
- Name
- Labcorp Central Laboratory Services SARL
- Name
- Parexel Bioanalytical Services Division
- Name
- SRL Inc.
- Name
- Greenphire LLC
- Responsibilities
- Other Third Party Duty
- Name
- Almac Clinical Technologies LLC
Third parties
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Monitoring","organisation_type":"Pharmaceutical company"}
- {"country":"Japan","full_name":"SRL Inc.","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"Other Third Party Duty","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Biotrial","duties_or_roles":"Other Third Party Duty","organisation_type":"Pharmaceutical company"}
- {"country":"South Africa","full_name":"Parexel Bioanalytical Services Division","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Other Third Party Duty","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Satralizumab
- Active Substance
- satralizumab
- Modality
- Monoclonal antibody
- Authorisation Status
- Marketing authorisation present for product PRD9016776: EU/1/21/1559/001 (other test product entry appears to be investigational/unlicensed)
- Orphan Designation
- Yes
- Investigational Product Name
- Satralizumab (sponsor product code RO 533-3787/F01-06)
- Active Substance
- satralizumab
- Modality
- Monoclonal antibody
- Authorisation Status
- Investigational / no marketing authorisation number listed for product PRD10948861
- Investigational Product Name
- Placebo
- Modality
- Other
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