A Study to Test the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis
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|ClinicalTrials.gov Identifier: NCT04875975|
Recruitment Status : Recruiting
First Posted : May 6, 2021
Last Update Posted : March 3, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis||Drug: Rozanolixizumab Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||68 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis|
|Actual Study Start Date :||September 27, 2021|
|Estimated Primary Completion Date :||February 5, 2025|
|Estimated Study Completion Date :||February 5, 2025|
Participants will be randomized to receive a predefined dose of rozanolixizumab.
Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period.
Placebo Comparator: Placebo
Participants will be randomized to receive a dose of placebo.
Subjects will receive placebo in a pre-specified sequence during the Treatment Period.
- Proportion of seizure free study participants at the end of the Treatment Period [ Time Frame: From Baseline until the end of the Treatment Period (Week 25) ]Seizure freedom is defined by 28 consecutive days of no seizures maintained until the end of the Treatment Period
- Change from Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score at the end of the Treatment Period [ Time Frame: From Baseline until the end of the Treatment Period (Week 25) ]The Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) consists of 12 subtests that contribute to 5 age-based domain index scores (immediate memory, visuospatial/constructional, language, attention, delayed memory) that are aggregated for a total scale index score. Higher scores reflect better neurocognitive performance.
- Proportion of participants who required rescue medication due to an absence or loss of clinical benefit during the Treatment Period [ Time Frame: From Baseline until the end of the Treatment Period (Week 25) ]Study participants who require rescue medication due to an absence or loss of clinical benefit will discontinue blinded treatment, and complete the assessments for the Early Discontinuation Visit.
- Time to first occurrence of seizure freedom during the Treatment Period [ Time Frame: From Baseline until the end of the Treatment Period (Week 25) ]The time to first occurrence of seizure freedom (TTFSF) is defined by the number of days after randomization to the first day of the first 28 consecutive days without seizures during the Treatment Period
- Incidence of Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: From Baseline until the End of Study Visit (Week 32) ]An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
- Proportion of participants with a favorable outcome in the Modified Rankin Scale (mRS) during the Treatment Period [ Time Frame: From Baseline until the end of the Treatment Period (Week 25) ]Proportion of participants with a favorable outcome in the Modified Rankin Scale (mRS) during the Treatment Period, where favorable outcome is defined as no worsening for participants with a Baseline mRS score of ≤1 or improvement of ≥1 point for participants with a Baseline mRS score of ≥2
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years to 89 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Study participant must be ≥18 to ≤89 years of age
- Study participant must be seropositive for leucine-rich glioma inactivated 1 (LGI1) antibody
Study participant must have ≥2 seizures/week during the Screening Period or have experienced such seizures that stopped following high dose corticosteroids (500 to 1000 milligram (mg) methylprednisolone (MP) equivalent/day):
- Either faciobrachial dystonic seizures (FBDS) with or without other focal (partial) seizures including focal to bilateral tonic clonic
- Or focal (partial) seizures including focal to bilateral tonic clonic and fulfil the following new-onset Autoimmune encephalitis (AIE) criteria
- Study participant has initiated or re-initiated corticosteroids at a dose of 500 to 1000 mg MP equivalent/day within 42 days prior to randomization. Participants re-initiating corticosteroids are eligible only if re-initiation is due to seizure rebound and within the timeframe outlined. If the study participant has initiated a steroid taper, the study participant cannot receive an oral steroid dose lower than 40mg/day when randomized
- Study participant with onset of disease symptom between 0 to 12 months prior to Screening, per investigator's assessment.
- Study participant weighs at least 35 kg at Screening
A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
i) Not a woman of childbearing potential (WOCBP) OR ii) A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 90 days after the final dose of study treatment
- Study participant has a known hypersensitivity to any components of the study medication or any other anti-neonatal Fc receptor (FcRn) medications.
- Study participant has a confirmed prior diagnosis of epilepsy or new onset seizures that are unrelated to LGI1 autoimmune encephalitis (AIE) or has any known or suspected medical cause for the onset of seizures other than possible AIE
- Study participant has a known active neoplastic disease or history of neoplastic disease within 5 years of study entry
- Study participant has renal impairment, defined as glomerular filtration rate (GFR) <30mL/min/1.73m2 at the Screening Visit
- Study participant has a clinically important active infection (including unresoved or not adequately treated infection) as assessed by investigator
- Study participant has a history of chronic ongoing infections
- Study participant has current unstable liver or biliary disease, per investigator assessment, defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis
- Study participant has a history of solid organ transplant or hematopoietic stem cell transplant
- Study participant has undergone a splenectomy
- Study participant has a current or medical history of primary immune deficiency
- Study participant has received a live vaccination within 4 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of investigational medicinal product (IMP)
- Study participant has previously received rozanolixizumab drug product
- Alanine transaminase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) are >3x upper limit of normal (ULN)
- Study participant has a total IgG level ≤5.5 g/L at the Screening Visit
- Study participant has absolute neutrophil count <1500 cells/mm^3 at the Screening Visit
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04875975
|Contact: UCB Cares||1-844-599-2273 (USA)||UCBCares@ucb.com|
|Contact: UCB Cares||0018445992273||UCBCares@ucb.com|
|Study Director:||UCB Cares||001 844 599 2273 (UCB)|
|Responsible Party:||UCB Biopharma SRL|
|Other Study ID Numbers:||
2019-004778-25 ( EudraCT Number )
|First Posted:||May 6, 2021 Key Record Dates|
|Last Update Posted:||March 3, 2023|
|Last Verified:||March 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.|
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
|Time Frame:||Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.|
|Access Criteria:||Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||No|
Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Central Nervous System Diseases
Nervous System Diseases
Endocrine System Diseases
Physiological Effects of Drugs