Trial Outcomes & Findings for C1-esterase Inhibitor (Cinryze) for Acute Treatment of Neuromyelitis Optica Exacerbation (NCT NCT01759602)

Results Overview

Over the course of hospitalization for the acute NMO exacerbations, subjects will be monitored daily for frequency of adverse events.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

10 participants

Primary outcome timeframe

5-21 days

Results posted on

2014-07-18

Participant Flow

Participant milestones

Participant milestones
Measure	C1-esterase Inhibitor (Cinryze) This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

C1-esterase Inhibitor (Cinryze) for Acute Treatment of Neuromyelitis Optica Exacerbation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	C1-esterase Inhibitor (Cinryze) n=10 Participants This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Age, Continuous	41 years n=99 Participants
Sex: Female, Male Female	8 Participants n=99 Participants
Sex: Female, Male Male	2 Participants n=99 Participants

PRIMARY outcome

Timeframe: 5-21 days

Over the course of hospitalization for the acute NMO exacerbations, subjects will be monitored daily for frequency of adverse events.

Outcome measures

Outcome measures
Measure	C1-esterase Inhibitor (Cinryze) n=10 Participants This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Number of Adverse Safety Events During Hospitalization	0 adverse safety events

SECONDARY outcome

Timeframe: 5-21 days

Outcome measures

Outcome measures
Measure	C1-esterase Inhibitor (Cinryze) n=10 Participants This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Frequency of Serious Adverse Events.	0 serious adverse events

SECONDARY outcome

Timeframe: 5-21 days

Outcome measures

Outcome measures
Measure	C1-esterase Inhibitor (Cinryze) n=10 Participants This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Percentage of Subjects Withdrawing Due to Adverse Events.	0 percentage of subjects withdrawing

SECONDARY outcome

Timeframe: 5-21 days

ALT elevations were considered "mild" if they rose to less than 4-fold baseline levels.

Outcome measures

Outcome measures
Measure	C1-esterase Inhibitor (Cinryze) n=10 Participants This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Change From Baseline in Hematology, Chemistry, and Urinalysis Parameters. ALT Level, mild elevation	1 participants
Change From Baseline in Hematology, Chemistry, and Urinalysis Parameters. Hematology, abnormal	0 participants
Change From Baseline in Hematology, Chemistry, and Urinalysis Parameters. Urinalysis, abnormal	0 participants

SECONDARY outcome

Timeframe: participants were followed for the duration of hospital stay ranging from 5-21 days, an average of 13 days; EDSS assessment was administered at discharge

The Kurtzke Expanded Disability Status Scale (EDSS) was developed to measure the disability status of patients with multiple sclerosis. 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. The first levels 1.0 to 4.5 refer to people with a high degree of ambulatory ability and the subsequent levels 5.0 to 9.5 refer to the loss of ambulatory ability. The range of main categories include (0) = normal neurologic exam; to (5) = ambulatory without aid or rest for 200 meters; disability severe enough to impair full daily activities; to (10) = death due to MS. In addition, it also provides eight subscale measurements called Functional System (FS) scores.

Outcome measures

Outcome measures
Measure	C1-esterase Inhibitor (Cinryze) n=10 Participants This is a phase 1b open-label, interventional proof-of-concept study in patients with neuromyelitis optica (NMO) in which all subjects will receive 3 daily infusions of 2000 Units of intravenous CINRYZE at the onset of an NMO exacerbation in addition to standard of care high-dose steroids, plus an additional 2 infusions of 1000 Units of intravenous CINRYZE during a second treatment phase with plasma exchange, if necessary. C1-esterase inhibitor (Cinryze)
Expanded Disability Status Score (EDSS)	2.5 EDSS scores Interval 0.0 to 8.0

Adverse Events

C1-esterase Inhibitor (Cinryze)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Michael Levy, MD, PhD

Johns Hopkins University

Phone: 443-287-4412

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place