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Trial record 3 of 52 for:    Devic syndrome

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

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ClinicalTrials.gov Identifier: NCT01759602
Recruitment Status : Completed
First Posted : January 3, 2013
Results First Posted : July 18, 2014
Last Update Posted : July 18, 2014
Sponsor:
Collaborator:
ViroPharma
Information provided by (Responsible Party):
Michael Levy, Johns Hopkins University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Neuromyelitis Optica
Intervention Drug: C1-esterase inhibitor (Cinryze)
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description

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)

Period Title: Overall Study
Started 10
Completed 10
Not Completed 0
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description

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 Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants
41
(21 to 63)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
8
  80.0%
Male
2
  20.0%
1.Primary Outcome
Title Number of Adverse Safety Events During Hospitalization
Hide Description Over the course of hospitalization for the acute NMO exacerbations, subjects will be monitored daily for frequency of adverse events.
Time Frame 5-21 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description:

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 Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: adverse safety events
0
2.Secondary Outcome
Title Frequency of Serious Adverse Events.
Hide Description [Not Specified]
Time Frame 5-21 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description:

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 Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: serious adverse events
0
3.Secondary Outcome
Title Percentage of Subjects Withdrawing Due to Adverse Events.
Hide Description [Not Specified]
Time Frame 5-21 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description:

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 Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: percentage of subjects withdrawing
0
4.Secondary Outcome
Title Change From Baseline in Hematology, Chemistry, and Urinalysis Parameters.
Hide Description ALT elevations were considered "mild" if they rose to less than 4-fold baseline levels.
Time Frame 5-21 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description:

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 Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: participants
ALT Level, mild elevation 1
Hematology, abnormal 0
Urinalysis, abnormal 0
5.Secondary Outcome
Title Expanded Disability Status Score (EDSS)
Hide Description

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.

Time Frame 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
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description:

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 Number of Participants Analyzed 10
Median (Full Range)
Unit of Measure: EDSS scores
2.5
(0 to 8)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title C1-esterase Inhibitor (Cinryze)
Hide Arm/Group Description

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)

All-Cause Mortality
C1-esterase Inhibitor (Cinryze)
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
C1-esterase Inhibitor (Cinryze)
Affected / at Risk (%)
Total   0/10 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
C1-esterase Inhibitor (Cinryze)
Affected / at Risk (%)
Total   0/10 (0.00%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Michael Levy, MD, PhD
Organization: Johns Hopkins University
Phone: 443-287-4412
EMail: mlevy@jhmi.edu
Layout table for additonal information
Responsible Party: Michael Levy, Johns Hopkins University
ClinicalTrials.gov Identifier: NCT01759602     History of Changes
Other Study ID Numbers: NA_00078437
First Submitted: December 29, 2012
First Posted: January 3, 2013
Results First Submitted: May 28, 2014
Results First Posted: July 18, 2014
Last Update Posted: July 18, 2014