A Study to Test the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis

NCT ID: NCT04875975

Last Updated: 2025-05-31

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-09-27
• Study Completion Date: 2024-04-26

Brief Summary

The purpose of the study is to assess the efficacy of rozanolixizumab as measured by seizure freedom, change in cognitive function, use of rescue medication, onset of seizure freedom and to assess safety and tolerability.

Conditions

Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Rozanolixizumab

Participants will be randomized to receive a predefined dose of rozanolixizumab.

Group Type: EXPERIMENTAL

Rozanolixizumab

Intervention Type: DRUG

• Pharmaceutical form: Solution for infusion
• Route of administration: Subcutaneous use

Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period.

Placebo

Participants will be randomized to receive a dose of placebo.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

• Pharmaceutical form: Solution for infusion
• Route of administration: Subcutaneous use

Subjects will receive placebo in a pre-specified sequence during the Treatment Period.

Interventions

Rozanolixizumab

• Pharmaceutical form: Solution for infusion
• Route of administration: Subcutaneous use

Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period.

Intervention Type: DRUG

Placebo

• Pharmaceutical form: Solution for infusion
• Route of administration: Subcutaneous use

Subjects will receive placebo in a pre-specified sequence during the Treatment Period.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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 unresolved or not adequately treated infection) as assessed by investigator, including participants with a serious infection within 6 weeks prior to the first dose of investigational medicinal product (IMP)
• 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 positive tuberculosis (TB) test at the Screening Visit
• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 89 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

UCB Biopharma SRL

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

UCB Cares

Role: STUDY_DIRECTOR

001 844 599 2273 (UCB)

Locations

Aie001 50101

Aurora, Colorado, United States

Aie001 50342

Jacksonville, Florida, United States

Aie001 50243

Boston, Massachusetts, United States

Aie001 50047

Boston, Massachusetts, United States

Aie001 50104

Rochester, Minnesota, United States

Aie001 50298

New York, New York, United States

Aie001 50090

Winston-Salem, North Carolina, United States

Aie001 50311

Cleveland, Ohio, United States

Aie001 50304

Dallas, Texas, United States

Aie001 30027

Melbourne, , Australia

Aie001 40123

Brussels, , Belgium

Aie001 40129

Bordeaux, , France

Aie001 40426

Bron, , France

Aie001 40364

Lille, , France

Aie001 40546

Nancy, , France

Aie001 40132

Nice, , France

Aie001 40019

Paris, , France

Aie001 40286

Toulouse, , France

Aie001 40515

Berlin, , Germany

Aie001 40249

Kiel, , Germany

Aie001 40695

Pavia, , Italy

Aie001 40567

Roma, , Italy

Aie001 40264

Rotterdam, , Netherlands

Aie001 40267

Barcelona, , Spain

Aie001 40341

Málaga, , Spain

Aie001 40168

Nottingham, , United Kingdom

Aie001 40163

Oxford, , United Kingdom

Countries

United States; Australia; Belgium; France; Germany; Italy; Netherlands; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2019-004778-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AIE001

Identifier Type: -

Identifier Source: org_study_id