Trial Outcomes & Findings for Ublituximab for Acute Neuromyelitis Optica (NMO) Relapses (NCT NCT02276963)

Last Updated: 2019-06-06

Results Overview

The Kurtzke Expanded Disability Status Scale (EDSS) was developed to measure the disability status of subjects with demyelinating disease. It allows an objective quantification of the level of functioning that could be widely and reproducibly used by researchers and health care providers. The EDSS provides a total score on a scale that ranges from 0 to 10 where 0 is normal and 10 is deceased. Increasing disability is reflected in an increasing EDSS score.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

6 participants

Primary outcome timeframe

On admission to the hospital on day 1, on discharge 5-21 days later and on follow up at 90 days

Results posted on

2019-06-06

Participant Flow

This was a single center, phase I open label study of ublituximab in NMOSD patients with acute relapses. Patients aged 18-65 who presented to the Johns Hopkins Hospital between January and September 2016 with new neurologic symptoms were eligible for enrollment if they demonstrated a new contrast-enhancing lesion on MRI.

There were no pre-assignment events.

Participant milestones

Participant milestones
Measure	Ublituximab Plus Glucocorticoids Ublituximab 450 mg intravenously once on day 1, plus glucocorticoids 1000 mg intravenously daily on days 1-5 Ublituximab: Monoclonal antibody that specifically binds to the trans-membrane antigen CD20, which induces immune response that causes lysis of B cells.
Overall Study STARTED	6
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ublituximab for Acute Neuromyelitis Optica (NMO) Relapses

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ublituximab Plus Glucocorticoids n=5 Participants Ublituximab 450 mg intravenously once on day 1, plus glucocorticoids 1000 mg intravenously daily on days 1-5 Ublituximab: Monoclonal antibody that specifically binds to the trans-membrane antigen CD20, which induces immune response that causes lysis of B cells.
Age, Continuous	44.4 years STANDARD_DEVIATION 11.5 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants
AQP4 IgG serostatus AQP4 IgG seropositive	5 Participants n=5 Participants
AQP4 IgG serostatus AQP4 IgG seronegative	0 Participants n=5 Participants
Duration of disease	12.2 years STANDARD_DEVIATION 4.5 • n=5 Participants
Number of previous relapses	4.8 relapses STANDARD_DEVIATION 1.3 • n=5 Participants
Background immunotherapy None	2 Participants n=5 Participants
Background immunotherapy Mycophenolate mofetil	2 Participants n=5 Participants
Background immunotherapy Rituximab	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: On admission to the hospital on day 1, on discharge 5-21 days later and on follow up at 90 days

Population: 5 subjects had EDSS measurements at the first three time points: baseline, admission and discharge. 3 subjects had EDSS scores with an additional EDSS measurement at 90-day follow up.

Outcome measures

Outcome measures
Measure	Ublituximab Plus Glucocorticoids n=5 Participants Ublituximab 450 mg intravenously once on day 1, plus glucocorticoids 1000 mg intravenously daily on days 1-5 Ublituximab: Monoclonal antibody that specifically binds to the trans-membrane antigen CD20, which induces immune response that causes lysis of B cells.
Change in Neurological Disability - Expanded Disability Scale Score Baseline	4.0 EDSS unit score Interval 2.25 to 5.5
Change in Neurological Disability - Expanded Disability Scale Score Admission	6.5 EDSS unit score Interval 5.25 to 7.5
Change in Neurological Disability - Expanded Disability Scale Score Discharge	6.5 EDSS unit score Interval 4.75 to 7.5
Change in Neurological Disability - Expanded Disability Scale Score 90-day follow up	4.0 EDSS unit score Interval 2.0 to 8.0

Adverse Events

Ublituximab Plus Glucocorticoids

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ublituximab Plus Glucocorticoids n=5 participants at risk Ublituximab 450 mg intravenously once on day 1, plus glucocorticoids 1000 mg intravenously daily on days 1-5 Ublituximab: Monoclonal antibody that specifically binds to the trans-membrane antigen CD20, which induces immune response that causes lysis of B cells.
Musculoskeletal and connective tissue disorders Headache/body ache	60.0% 3/5 • Number of events 3 • 90 days Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Blood and lymphatic system disorders Leukopenia	20.0% 1/5 • Number of events 1 • 90 days Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.

Additional Information

Michael Levy

Johns Hopkins University

Phone: 443-287-4412

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place