Trial Outcomes & Findings for A Study to Test the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis (NCT NCT04875975)

Last Updated: 2025-05-31

Results Overview

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).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

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

Results posted on

2025-05-31

Participant Flow

The study started to enroll participants in September 2021 and concluded in April 2024.

The Participant Flow refers to the Randomized Set (RS).

Participant milestones

Participant milestones
Measure	Placebo Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Overall Study STARTED	6	6
Overall Study COMPLETED	2	0
Overall Study NOT COMPLETED	4	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Overall Study Adverse Event	1	2
Overall Study Lack of Efficacy	2	2
Overall Study Permanently left due to seizure recurrence	1	0
Overall Study Relapse With Insults	0	1
Overall Study Permanently left due to new seizure recurrence	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=6 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.	Total n=12 Participants Total of all reporting groups
Age, Continuous	60.7 Years STANDARD_DEVIATION 12.4 • n=5 Participants	70.7 Years STANDARD_DEVIATION 11.4 • n=7 Participants	65.7 Years STANDARD_DEVIATION 12.5 • n=5 Participants
Age, Customized 18 - <65 years	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Age, Customized 65 - <85 years	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Age, Customized >=85 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Race/Ethnicity, Customized White	5 Participants n=5 Participants	4 Participants n=7 Participants	9 Participants n=5 Participants
Race/Ethnicity, Customized Missing	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	5 Participants n=5 Participants	4 Participants n=7 Participants	9 Participants n=5 Participants

PRIMARY outcome

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

Population: The Randomized Set (RS) consisted of all enrolled study participants who were randomized to treatment arms.

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).

Outcome measures

Outcome measures
Measure	Placebo n=6 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Number of Seizure Free Study Participants at the End of the Treatment	1 Participants	0 Participants

SECONDARY outcome

Timeframe: From Baseline to Week 5, 13, 21 and 25

Population: The Randomized Set (RS) consisted of all enrolled study participants who were randomized to treatment arms. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Here, number analyzed signifies participants who were evaluable at specified time points.

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.

Outcome measures

Outcome measures
Measure	Placebo n=4 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Scale Index Score at the End of the Treatment Week 13	7.0 score on a scale Standard Deviation 2.9	12.3 score on a scale Standard Deviation 7.9
Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Scale Index Score at the End of the Treatment Week 25	NA score on a scale Standard Deviation NA As pre-specified in the SAP, mean and SD will only be calculated if number of participants are equal to 3 or greater than 3. Here, Mean and SD were not calculated due to less than 3 participants.	NA score on a scale Standard Deviation NA As pre-specified in the SAP, mean and SD will only be calculated if number of participants are equal to 3 or greater than 3. Here, Mean and SD were not calculated due to less than 3 participants.
Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Scale Index Score at the End of the Treatment Week 5	4.5 score on a scale Standard Deviation 2.6	2.2 score on a scale Standard Deviation 11.3
Change From Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Scale Index Score at the End of the Treatment Week 21	7.0 score on a scale Standard Deviation 7.3	NA score on a scale Standard Deviation NA As pre-specified in the SAP, mean and SD will only be calculated if number of participants are equal to 3 or greater than 3. Here, Mean and SD were not calculated due to less than 3 participants.

SECONDARY outcome

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

Population: The Randomized Set (RS) consisted of all enrolled study participants who were randomized to treatment arms. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Analysis datasets were not generated an output when the n \< 3, therefore, no data obtained and reported.

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 1. No significant disability despite symptoms; able to carry out all usual activities 2. Slight disability; unable to carry out all previous activities, but able to look after own affairs without assistance 3. Moderate disability; requiring some help, but able to walk without assistance 4. Moderately severe disability; unable to walk and attend to own bodily needs without assistance 5. Severe disability; bedridden, incontinent and requiring constant nursing care and attention 6. Dead

Outcome measures

Outcome measures
Measure	Placebo n=2 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=1 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Percentage of Participants With a Favorable Outcome in the Modified Rankin Scale (mRS) During the Treatment	NA percentage of participants Analysis datasets were not generated an output when the n \< 3, therefore, no data obtained and reported.	NA percentage of participants Analysis datasets were not generated an output when the n \< 3, therefore, no data obtained and reported.

SECONDARY outcome

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

Population: The Randomized Set (RS) consisted of all enrolled study participants who were randomized to treatment arms.

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.

Outcome measures

Outcome measures
Measure	Placebo n=6 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Number of Participants Who Required Rescue Medication Due to an Absence or Loss of Clinical Benefit During the Treatment	3 Participants	2 Participants

SECONDARY outcome

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

Population: The Randomized Set (RS) consisted of all enrolled study participants who were randomized to treatment arms.

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.

Outcome measures

Outcome measures
Measure	Placebo n=6 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Time to First Occurrence of Seizure Freedom During the Treatment	0.1 Weeks Interval 0.1 to 8.4	0.1 Weeks Interval 0.1 to 8.3

SECONDARY outcome

Timeframe: From Baseline until the End of Study (Week 32)

Population: The Safety Set (SS) consisted of all randomized study participants who received at least one dose of IMP.

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).

Outcome measures

Outcome measures
Measure	Placebo n=6 Participants Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 Participants Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)	100 percentage of participants	100 percentage of participants

Adverse Events

Placebo

Serious events: 3 serious events

Other events: 6 other events

Deaths: 0 deaths

Rozanolixizumab (RLZ)

Serious events: 3 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Placebo n=6 participants at risk Participants received placebo as a subcutaneous (sc) infusion once a week (Q1W) for 25 weeks.	Rozanolixizumab (RLZ) n=6 participants at risk Participants received rozanolixizumab as a sc infusion Q1W for 25 weeks.
Infections and infestations COVID-19	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Infections and infestations Psoas abscess	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Infections and infestations Bacteraemia	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Nervous system disorders Encephalitis autoimmune	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Nervous system disorders Peripheral sensory neuropathy	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Psychiatric disorders Mania	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Psychiatric disorders Suicidal ideation	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.
Vascular disorders Deep vein thrombosis	16.7% 1/6 • Number of events 1 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.	0.00% 0/6 • From Baseline until the End of Study (up to Week 32) 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) SFU. The Safety Set consisted of all randomized study participants who received at least one dose of IMP.

Other adverse events

Additional Information

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