Trial Outcomes & Findings for Efficacy of Ocrelizumab in Autoimmune Encephalitis (NCT NCT03835728)

NCT ID: NCT03835728

Last Updated: 2021-10-19

Results Overview

The number of participants who had clinical worsening within 12 months.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

12 months

Results posted on

2021-10-19

Participant Flow

Failed to meet target enrollment and study was discontinued

Participant milestones

Participant milestones
Measure	Treatment Arm Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.
Overall Study STARTED	2	1
Overall Study COMPLETED	2	1
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy of Ocrelizumab in Autoimmune Encephalitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment Arm n=2 Participants Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm n=1 Participants Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.	Total n=3 Participants Total of all reporting groups
Age, Customized Age	47.5 years n=5 Participants	44 years n=7 Participants	44 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	2 participants n=5 Participants	1 participants n=7 Participants	3 participants n=5 Participants
Encephalitis antibody NMDAR Ab	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Encephalitis antibody LGI1 Ab	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

The number of participants who had clinical worsening within 12 months.

Outcome measures

Outcome measures
Measure	Treatment Arm n=2 Participants Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm n=1 Participants Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.
Number of Participants Who Had Clinical Worsening	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 12 months

Population: There were no treatment failures in the treatment arm. Thus, there was no data for this secondary outcome measure in the treatment arm

Definition of clinical worsening (treatment failure): 1. Clinician or patient/caregiver perception of clinical decline 2. Worsening of patient/family reported IADL (by one point or more) 3. One of the following additional features: * Significant worsening of Texas Functional Living Scale (by ≥ 5 T points, 0.5 st deviation) * Other clinical worsening leading to hospitalization

Outcome measures

Outcome measures
Measure	Treatment Arm Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm n=1 Participants Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.
Time to Treatment Failure	—	12 weeks

SECONDARY outcome

Timeframe: Baseline, 6 month

Population: Participant in placebo arm met study endpoint prior to 6 month outcome measure so change in TFLS score at 6 months was not analyzable for efficacy purposes.

Change in TFLS T-score (Texas Functional Living Scale) scores at 6 months compared to baseline. - A performance-based measure of functional competence designed to assess instrumental activities of daily living (e.g., managing money) that are thought to be more susceptible to cognitive change than basic activities of daily living (e.g., dressing). Content and Structure: The TFLS is comprised of 24 items divided into four subscales assessing abilities related to Time, Money and Calculation, Communication, and Memory. Many items provide a range of possible points allowing the instrument to account for the varying levels of functioning that may be observed in clinical populations. Total raw score ranges between 0 and 50 with standardized T-score values between 20 and 66. The higher the score, the better the performance. Change in TFLS T-score was used in this study

Outcome measures

Outcome measures
Measure	Treatment Arm n=2 Participants Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.
Change in TFLS T-score (Texas Functional Living Scale) Score at 6 Months	12.75 score on a scale Interval 3.0 to 22.5	—

SECONDARY outcome

Timeframe: Baseline, 12 months

Population: Participant in placebo arm met study endpoint prior to 6 month outcome measure so change in TFLS score at 12 months was not analyzable for efficacy purposes.

Change in TFLS T-score (Texas Functional Living Scale) scores at 12 months compared to baseline. - A performance-based measure of functional competence designed to assess instrumental activities of daily living (e.g., managing money) that are thought to be more susceptible to cognitive change than basic activities of daily living (e.g., dressing). Content and Structure: The TFLS is comprised of 24 items divided into four subscales assessing abilities related to Time, Money and Calculation, Communication, and Memory. Many items provide a range of possible points allowing the instrument to account for the varying levels of functioning that may be observed in clinical populations. Total raw score ranges between 0 and 50 with standardized T-score values between 20 and 66. The higher the score, the better the performance. Change in TFLS T-score was used in this study

Outcome measures

Outcome measures
Measure	Treatment Arm n=2 Participants Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.
Change in TFLS T Score (Texas Functional Living Scale) Score at 12 Months	24.5 score on a scale Interval 3.0 to 46.0	—

Adverse Events

Treatment Arm

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Treatment Placebo Arm

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment Arm n=2 participants at risk Ocrelizumab will be administered 3 times over a 1 year study period. Subjects will receive a dose of 300 mg at week 0 (baseline) and again at week 2. The final dose of 600 mg will be administered at week 24. Ocrelizumab: Subjects will be randomized in a 1:1 fashion to receive infusion of Ocrelizumab (2 doses at 300 mg and 1 dose at 600 mg) or matched placebo. The 2 300 mg doses will be administered at day 2 and day 14. The 600 mg dose will be administered during the 6 month visit. The drug will be administered via infusion three times throughout the trial period: after the initial screening, at two weeks from initial infusion, and at 6 months.	Treatment Placebo Arm n=1 participants at risk Saline will be used as the matching placebo Saline: This will be the matching placebo used in the study.
Skin and subcutaneous tissue disorders rash	100.0% 2/2 • Number of events 2 • 1 year	100.0% 1/1 • Number of events 1 • 1 year
Hepatobiliary disorders Elevated transaminases	50.0% 1/2 • Number of events 1 • 1 year	0.00% 0/1 • 1 year
Nervous system disorders Seizures	100.0% 2/2 • Number of events 2 • 1 year	0.00% 0/1 • 1 year

Additional Information

Dr. Steven Vernino

UT Southwestern, Dept of Neurology

Phone: 214-645-8800

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place