Leucine-Rich Glioma Inactivated 1 Autoimmune EncephalitisPhase 2NCT04875975
A Study to Test the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis
A Phase 2 study testing Rozanolixizumab in people with Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis, run by UCB Biopharma SRL at 27 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 89 years
- Conditions the study is for
- Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis
- What is being tested?
- Rozanolixizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 27 sitesin 9 countries
- Study dates
- 27 Sep 2021to 26 Apr 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 89 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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:
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
27 sites in 9 countries.
27 sites in 9 countries
United States9 sites
Aie001 50101
Aurora, Colorado, 80045-2541
Aie001 50243
Boston, Massachusetts, 02114-3117
Aie001 50047
Boston, Massachusetts, 02115
Aie001 50311
Cleveland, Ohio, 44195
Aie001 50304
Dallas, Texas, 75390-8869
Aie001 50342
Jacksonville, Florida, 32224
Aie001 50298
New York, New York, 10016
Aie001 50104
Rochester, Minnesota, 55905
Aie001 50090
Winston-Salem, North Carolina, 27157
France7 sites
Aie001 40129
Bordeaux
Aie001 40426
Bron
Aie001 40364
Lille
Aie001 40546
Nancy
Aie001 40132
Nice
Aie001 40019
Paris
Aie001 40286
Toulouse
About this study
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
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.
- Being tested
- Rozanolixizumab, Placebo
- Keywords
- Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis, Phase 2, Rozanolixizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RozanolixizumabDrug
- Pharmaceutical form: Solution for infusion
- Route of administration: Subcutaneous use
Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period.
PlaceboDrug
- Pharmaceutical form: Solution for infusion
- Route of administration: Subcutaneous use
Subjects will receive placebo in a pre-specified sequence during the Treatment Period.
| Study group | Role | What they receive |
|---|---|---|
| Rozanolixizumab | Experimental | Participants will be randomized to receive a predefined dose of rozanolixizumab. Receives: Drug: Rozanolixizumab |
| Placebo | Placebo comparator | Participants will be randomized to receive a dose of placebo. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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
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 were aggregated for a total scale index score. All index scores have an age-based mean of 100, with a standard deviation (SD) of 15. The total scale score was calculated by taking the mean of the sum of the five index scores. Total possible scale index scores range from 40-135. Higher scores reflect better neurocognitive performance. The total scale index score is the score typically used to reflect global neurocognitive status. Baseline of RBANS is defined as the screening (Visit 1, Week -1) value.
Measured: 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
Percentage of participants with a favorable outcome in mRS during treatment, where favorable outcome defined as no worsening for participants with Baseline mRS score of ≤1 or improvement of ≥1 point for participants with Baseline mRS score of ≥2. The mRS is commonly used scale for measuring degree of disability or dependence in daily activities of people who suffered a stroke or other causes of neurological disability. The scale ranges from 0 (perfect health) to 6 (death).
0-No symptoms at all
- No significant disability despite symptoms; able to carry out all usual activities
- Slight disability; unable to carry out all previous activities, but able to look after own affairs without assistance
- Moderate disability; requiring some help, but able to walk without assistance
- Moderately severe disability; unable to walk and attend to own bodily needs without assistance
- Severe disability; bedridden, incontinent and requiring constant nursing care and attention
- Dead
Measured: 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
Study participants who required rescue medication due to an absence or loss of clinical benefit were discontinued blinded treatment and completed the assessments for the early discontinuation visit.
Measured: From Baseline until the end of the Treatment (Week 25)
Time to First Occurrence of Seizure Freedom During the Treatment
The time to first occurrence of seizure freedom was defined by the number of days after randomization to the first day of the first 28 consecutive days without seizures during the treatment. Time to first occurrence of 28 consecutive days of seizure freedom (days) during the treatment was calculated as date of first day of occurrence of 28 consecutive days of seizure freedom - Date of Randomization + 1.
Measured: From Baseline until the end of the Treatment (Week 25)
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP, whether or not related to the IMP. A TEAE was defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment up to the end of the Treatment and including the 8-week (56 days) Safety-Follow Up (SFU).
Measured: From Baseline until the End of Study (Week 32)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2021
- Study start27 Sep 2021
- Primary completion8 Mar 2024
- Study completion26 Apr 2024
Why it stopped: The study stopped prematurely due to enrolment challenges, the termination was not linked to any safety issues.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Leucine-Rich Glioma Inactivated 1 Autoimmune EncephalitisStudies of the same condition, open and under way.
- More trials run by UCB Biopharma SRLThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma SRL
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04875975
- Sponsor's protocol ID
- AIE001
- Other IDs
- 2019-004778-25, 2019
- Status last verified
- May 2025
- First submitted
- 3 May 2021
- Last updated
- 31 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04875975
- Acronym / sponsor ID
- AIE001
- Keywords
- Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis, Phase 2, Rozanolixizumab
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study stopped prematurely due to enrolment challenges, the termination was not linked to any safety issues.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- UCB Biopharma SRLIndustry (pharma, biotech, devices)
- Study start
- 27 Sep 2021 (actual)
- Primary completion
- 8 Mar 2024 (actual)
- Study completion
- 26 Apr 2024 (actual)
- First posted
- 6 May 2021 (actual)
- Results
- Posted 31 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rozanolixizumab | Experimental | Drug: RozanolixizumabParticipants will be randomized to receive a predefined dose of rozanolixizumab. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will be randomized to receive a dose of placebo. |
Who may qualify
- Ages
- 18 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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):
- 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.
Key exclusion criteria (16 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rozanolixizumab* Pharmaceutical form: Solution for infusion * Route of administration: Subcutaneous use Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period. | Drug | Rozanolixizumab |
| Placebo* Pharmaceutical form: Solution for infusion * Route of administration: Subcutaneous use Subjects will receive placebo in a pre-specified sequence during the Treatment Period. | Drug | Placebo |
Primary outcome
Number of Seizure Free Study Participants at the End of the Treatment
Time frame: From Baseline until the end of the Treatment (Week 25)
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).
5 secondary outcomes
Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Scale Index Score at the End of the Treatment
Time frame: 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
Time frame: 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
Time frame: From Baseline until the end of the Treatment (Week 25)
Time to First Occurrence of Seizure Freedom During the Treatment
Time frame: From Baseline until the end of the Treatment (Week 25)
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From Baseline until the End of Study (Week 32)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273 (UCB)
27 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rozanolixizumab in people with Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis, run by UCB Biopharma SRL at 27 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 31 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, France, Germany, Italy, Spain and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Seizure Free Study Participants at the End of the Treatment (time frame: From Baseline until the end of the Treatment (Week 25)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 May 2025.