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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 October 2017
Main ID:  NCT01759602
Date of registration: 29/12/2012
Prospective Registration: Yes
Primary sponsor: Michael Levy
Public title: C1-esterase Inhibitor (Cinryze) for Acute Treatment of Neuromyelitis Optica Exacerbation
Scientific title: Phase 1b Study of C1-esterase Inhibitor (Cinryze) With Standard of Care for Acute Treatment of Neuromyelitis Optica Exacerbations
Date of first enrolment: January 2013
Target sample size: 10
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT01759602
Study type:  Interventional
Study design:   
Phase:  Phase 1
Countries of recruitment
United States
Contacts
Name:     Michael Levy, MD, PhD
Address: 
Telephone:
Email:
Affiliation:  Johns Hopkins University
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Able and willing to provide written informed consent.

2. 18-65 years of age.

3. New acute optic neuritis and/or transverse myelitis. A clinical event is defined as an
episode of inflammation in the spinal cord and/or optic nerve leading to neurologic
symptoms not ascribed to another disease process.

4. Diagnosis of NMO according to the 2006 revisions of the Wingerchuk diagnostic criteria
for NMO (Wingerchuk, 2006), or AQP4 positive NMOSD per the EFNS Guidelines. For NMO,
subjects must have two absolute criteria:

1. optic neuritis

2. myelitis and at least two of three supportive criteria:

3. presence of a contiguous spinal cord MRI lesion extending over three or more
vertebral segments,

4. MRI criteria NOT satisfying the revised McDonald diagnostic criteria for MS
[Polman, 2011]

5. NMO-IgG (AQP4) in serum. For NMOSD, subjects must have longitudinally extensive
transverse myelitis (LETM) recurrent isolated optic neuritis (RION)/bilateral
optic neuritis (BON), or opticospinal multiple sclerosis (OSMS) that is AQP4
antibody positive.

5. A female subject is eligible to enter the study if she is:

A. Not pregnant or nursing; B. Of non-childbearing potential (i.e. women who have had a
hysterectomy, are post-menopausal, which is defined as >2 years without menses or, in
female subjects who have been post-menopausal for <2 years, must be confirmed with Follicle
Stimulating Hormone (FSH) and estradiol levels), have both ovaries surgically removed or
have current documented tubal ligation) OR Of child-bearing potential (i.e. women with
functional ovaries and no documented impairment of oviductal or uterine function that would
cause sterility). This category includes women with oligomenorrhoea (even severe), women
who are perimenopausal or have just begun to menstruate.

C. Subject has a negative serum pregnancy test at screening and agrees to one of the
following:

1. Complete abstinence from intercourse for the period from consent into the study until
6 months after the last dose of investigational product; or,

2. Consistent and correct use of one of the following acceptable methods of birth control
for the period from consent into the study until 6 months after the last dose of
investigational product:

i. Oral contraceptives (either combined or progesterone only) ii. Injectable progesterone
iii. Levonorgestrel implants iv. Estrogenic vaginal ring v. Percutaneous contraceptive
patches vi. Intrauterine device (IUD) or intrauterine system (IUS) with a documented
failurerate of <1% per year vii. Male partner sterilization (vasectomy with documentation
of azoospermia) prior to the female subject's entry into the study; this male must be the
sole partner for the subject viii. Double barrier method: condom and an occlusive cap
(diaphragm or cervical/vault caps) with a vaginal spermicidal agent
(foam/gel/film/cream/suppository).

Exclusion Criteria:

1. Current evidence or known history of clinically significant infection including:

1. Chronic or ongoing active infectious disease requiring long term systemic
treatment such as, but not limited to: PML, chronic renal infection, chronic
chest infection with bronchiectasis, tuberculosis, or active hepatitis C.

2. Previous serious opportunistic or atypical infections.

3. History of positive serology for hepatitis B.

4. Prior history, or suspicion, of tuberculosis (TB)

5. History of positive serology for HIV.

2. History of clinically significant CNS trauma (e.g. traumatic brain injury, cerebral
contusion, spinal cord compression).

3. History or presence of myelopathy due to spinal cord compression by disc or vertebral
disease.

4. Past or current history of medically significant adverse effects (including allergic
reactions) from:

a. Corticosteroids

5. Past or current malignancy, except for

1. Cervical carcinoma Stage 1B or less

2. Non-invasive basal cell and squamous cell skin carcinoma

3. Cancer diagnoses with a duration of complete response (remission) >5 years

4. A history of hematologic malignancy excludes a subject from participation,
regardless of response.

6. Significant concurrent, uncontrolled medical condition including, but not limited to,
cardiac, renal, hepatic, hematological, gastrointestinal, endocrine, immunodeficiency
syndrome, pulmonary, cerebral, psychiatric, or neurological disease which could affect
the subject's safety, impair the subject's reliable participation in the trial, impair
the evaluation of endpoints, or necessitate the use of medication not allowed by the
protocol, as determined by the PI of the study.

7. Use of an investigational drug or other experimental therapy for a condition other
than NMO within 4 weeks, 5 pharmacokinetic half lives or duration of biological effect
(whichever is longer) prior to screening.

8. Current participation in any other interventional clinical trial. Participation in
non-interventional trial requires approval of the protocol by investigator.



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All
Health Condition(s) or Problem(s) studied
Neuromyelitis Optica
Intervention(s)
Drug: C1-esterase inhibitor (Cinryze)
Primary Outcome(s)
Number of Adverse Safety Events During Hospitalization [Time Frame: 5-21 days]
Secondary Outcome(s)
Change From Baseline in Hematology, Chemistry, and Urinalysis Parameters. [Time Frame: 5-21 days]
Expanded Disability Status Score (EDSS) [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]
Frequency of Serious Adverse Events. [Time Frame: 5-21 days]
Percentage of Subjects Withdrawing Due to Adverse Events. [Time Frame: 5-21 days]
Secondary ID(s)
NA_00078437
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
ViroPharma
Ethics review
Results
Results available: Yes
Date Posted: 18/07/2014
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT01759602
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