Phase 1
N=10
Efficacy of Bevacizumab (Avastin) in Treatment of Acute NMO Exacerbations
Neuromyelitis Optica · Neuromyelitis Optica Spectrum Disorder
Bottom Line
View on ClinicalTrials.gov: NCT01777412 ↗Enrolled (actual)
10
Serious AEs
10.0%
Results posted
May 2015
Primary outcome: Primary: Baseline Expanded Disability Status Score (EDSS) — 3.5 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Bevacizumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Johns Hopkins University
- Primary completion
- Feb 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Baseline Expanded Disability Status Score (EDSS) |
3.5 | — |
| PRIMARY Safety Assessment and Side Effects |
1; 0 | — |
| PRIMARY Follow-Up Expanded Disability Status Score (EDSS) |
3.0 | — |
Summary
This is a phase 1b interventional trial of bevacizumab (Avastin®) to evaluate the tolerability/safety and preliminary efficacy of bevacizumab (Avastin®) as add-on therapy for treatment of acute optic neuritis and/or transverse myelitis in neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD). A single infusion of Avastin® is added to standard-of-care high dose steroids and an additional dose of Avastin® is added to plasma exchange (if necessary). The primary outcomes are clinical changes in the Expanded Disability Severity Scale, Timed 25-foot Walk and Low Contrast Visual Acuity, MRI parameters and safety.
Eligibility Criteria
Subjects eligible for enrollment must meet all of the following criteria:
- Able and willing to provide written informed consent.
- 18-70 years of age.
- 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.
- Known or suspected diagnosis of NMO according to the 2006 revisions of the Wingerchuk diagnostic criteria for NMO or AQP4 positive NMOSD per the EFNS Guidelines. For NMO, subjects must have two absolute criteria:
- optic neuritis
- myelitis and at least two of three supportive criteria:
- presence of a contiguous spinal cord MRI lesion extending over three or more vertebral segments,
- MRI criteria NOT satisfying the revised McDonald diagnostic criteria for MS [Polman, 2011]
- 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
- 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 postmenopausal, which is defined as >2 years without menses (female subjects who have been post-menopausal for 5 years
- A history of hematologic malignancy excludes a subject from participation, regardless of response.
- Recent major surgery within the last 28 days.
- 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.
- 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.
- Current participation in any other interventional clinical trial. Participation in non-interventional trial requires approval of the protocol by investigator.
Data sourced from ClinicalTrials.gov (NCT01777412). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.