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

NCT04875975 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: AIE0012019-004778-25
• Study Type: interventional
• Target: 12
• Locations: 9 countries
• Sites: 27

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

Keywords

Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis; Phase 2; Rozanolixizumab

Outcome Measures

Primary Outcomes (1)

• Number of Seizure Free Study Participants at the End of the Treatment

  Seizure freedom was defined as a minimum of 28 consecutive days of no seizures of any type during the treatment and maintained until the end of the treatment (Week 25).

  From Baseline until the end of the Treatment (Week 25)

Secondary Outcomes (5)

• Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Scale Index Score at the End of the Treatment

  From Baseline to Week 5, 13, 21 and 25

• Percentage of Participants With a Favorable Outcome in the Modified Rankin Scale (mRS) During the Treatment

  From Baseline until the end of the Treatment (Week 25)

• Number of Participants Who Required Rescue Medication Due to an Absence or Loss of Clinical Benefit During the Treatment

  From Baseline until the end of the Treatment (Week 25)

• Time to First Occurrence of Seizure Freedom During the Treatment

  From Baseline until the end of the Treatment (Week 25)

• Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)

  From Baseline until the End of Study (Week 32)

Study Arms (2)

Rozanolixizumab

EXPERIMENTAL

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

Drug: Rozanolixizumab

Placebo

PLACEBO COMPARATOR

Participants will be randomized to receive a dose of placebo.

Drug: Placebo

Interventions

Rozanolixizumab DRUG

* Pharmaceutical form: Solution for infusion * Route of administration: Subcutaneous use Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period.

Rozanolixizumab

Placebo DRUG

* Pharmaceutical form: Solution for infusion * Route of administration: Subcutaneous use Subjects will receive placebo in a pre-specified sequence during the Treatment Period.

Placebo

Eligibility Criteria

• Age: 18 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (27)

Aie001 50101

Aurora, Colorado, 80045-2541, United States

Location

Aie001 50342

Jacksonville, Florida, 32224, United States

Location

Aie001 50243

Boston, Massachusetts, 02114-3117, United States

Location

Aie001 50047

Boston, Massachusetts, 02115, United States

Location

Aie001 50104

Rochester, Minnesota, 55905, United States

Location

Aie001 50298

New York, New York, 10016, United States

Location

Aie001 50090

Winston-Salem, North Carolina, 27157, United States

Location

Aie001 50311

Cleveland, Ohio, 44195, United States

Location

Aie001 50304

Dallas, Texas, 75390-8869, United States

Location

Aie001 30027

Melbourne, Australia

Location

Aie001 40123

Brussels, Belgium

Location

Aie001 40129

Bordeaux, France

Location

Aie001 40426

Bron, France

Location

Aie001 40364

Lille, France

Location

Aie001 40546

Nancy, France

Location

Aie001 40132

Nice, France

Location

Aie001 40019

Paris, France

Location

Aie001 40286

Toulouse, France

Location

Aie001 40515

Berlin, Germany

Location

Aie001 40249

Kiel, Germany

Location

Aie001 40695

Pavia, Italy

Location

Aie001 40567

Roma, Italy

Location

Aie001 40264

Rotterdam, Netherlands

Location

Aie001 40267

Barcelona, Spain

Location

Aie001 40341

Málaga, Spain

Location

Aie001 40168

Nottingham, United Kingdom

Location

Aie001 40163

Oxford, United Kingdom

Location

MeSH Terms

Interventions

rozanolixizumab

Limitations and Caveats

Due to the early termination of the recruitment and insufficient participants, it was not feasible to carry out the statistical analyses as originally planned.

Results Point of Contact

• Title: UCB
• Organization: Cares

UCBCares@ucb.com

001 844 599 2273

Study Officials

• UCB Cares

  001 844 599 2273 (UCB)

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2021
• First Posted: May 6, 2021
• Study Start: September 27, 2021
• Primary Completion: March 8, 2024
• Study Completion: April 26, 2024
• Last Updated: May 31, 2025
• Results First Posted: May 31, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, 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.

Locations

IT (2); AU (1); BE (1); FR (7); ES (2); US (9); DE (2); NL (1); GB (2)