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Neuromyelitis OpticaPhase 1NCT01777412

Efficacy of Bevacizumab (Avastin) in Treatment of Acute NMO Exacerbations

A Phase 1 study testing Bevacizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Johns Hopkins University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Bevacizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
Jun 2013to May 2015
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Subjects eligible for enrollment must meet all of the following criteria:

  1. Able and willing to provide written informed consent.
  2. 18-70 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. 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:
  5. optic neuritis
  6. myelitis and at least two of three supportive criteria:
  7. presence of a contiguous spinal cord MRI lesion extending over three or more vertebral segments,
  8. MRI criteria NOT satisfying the revised McDonald diagnostic criteria for MS [Polman, 2011]
  9. 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
  10. A female subject is eligible to enter the study if she is:
  11. 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 <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,
  12. Of childbearing 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. The subject must have a negative serum pregnancy test at screening and agrees to one of the following:
  13. Complete abstinence from intercourse for the period from consent into the study until 6 months after the last dose of investigational product; or, ii. 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:
  14. Oral contraceptives (either combined or progesterone only)
  15. Injectable progesterone
  16. Levonorgestrel implants
  17. Estrogenic vaginal ring
  18. Percutaneous contraceptive patches
  19. Intrauterine device (IUD) or intrauterine system (IUS) with a documented failure rate of <1% per year
  20. 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
  21. Double barrier method: condom and an occlusive cap (diaphragm or cervical/vault caps) with a vaginal spermicidal agent (foam/gel/film/cream/suppository).

Subjects meeting any of the following criteria are not eligible and cannot enroll in the study:

  1. Evidence or history of clinically significant infection including:
  2. 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.
  3. Positive test for HBsAg.
  4. Prior history, or suspicion, of tuberculosis (TB)
  5. History of positive serology for HIV.
  6. Past or current malignancy, except for
  7. Cervical carcinoma Stage 1B or less
  8. Non-invasive basal cell and squamous cell skin carcinoma
  9. Cancer diagnoses with a duration of complete response (remission) >5 years
  10. A history of hematologic malignancy excludes a subject from participation, regardless of response.
  11. Recent major surgery within the last 28 days.
  12. 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.
  13. 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.
  14. Current participation in any other interventional clinical trial. Participation in non-interventional trial requires approval of the protocol by investigator.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287

Other trials in United States

About this study

Official title: An Open-label Phase 1b Study of Avastin® (Bevacizumab) for the Treatment of Acute Optic Neuritis and/or Transverse Myelitis in Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD).

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.

Read the detailed description

Study Objective: The overall objective is to evaluate the tolerability/safety and efficacy of adding bevacizumab (Avastin®) to standard of care therapy in improving clinical and radiologic outcomes of acute optic neuritis and/or transverse myelitis in neuromyelitis optica and neuromyelitis optica spectrum disorders.

Primary Objective: To compare the clinical and radiographic outcome following acute optic neuritis and/or transverse myelitis in NMO/NMOSD in patients who receive 1-2 doses of 10 mg/kg dose of bevacizumab (Avastin®) in addition to standard medical therapy.

Secondary Objectives:

  • To determine the effect of Avastin on NMO clinical scores (Expanded Disability Status Scale, Timed 25-foot Walk and Low Contrast Visual Acuity [LCVA]).
  • To evaluate the safety and tolerability of a 10 mg/kg dose of intravenous Avastin.
  • To determine the frequency of adverse events with Avastin in this patient population.
  • To determine the effect of Avastin on MRI lesion size and extent.

The duration of the investigation is 1-2 years.

Being tested
Bevacizumab
Keywords
NMO, NMOSD, NMO-IgG, Aquaporin-4, AQP4, anti-AQP4
Medical subject headings
Neuromyelitis Optica

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT01777412
Study groupRoleWhat they receive
BevacizumabExperimentalBevacizumab 10 mg/kg intravenous infusion at onset of exacerbation and, if needed, a second time during the plasma exchange phase.
Receives: Drug: Bevacizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Baseline Expanded Disability Status Score (EDSS)

    EDSS

    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.

    Measured: Admission to hospital

  2. Safety Assessment and Side Effects

    Frequency and severity of adverse events and side effects. Serious adverse events are considered those which are life threatening, lead to hospitalization and related to the drug. Side effects are considered minor effects of the experimental drug that do not significantly impact the care of the patient with the experimental drug.

    Measured: 91 days

  3. Follow-Up Expanded Disability Status Score (EDSS)

    EDSS

    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.

    Measured: Follow-up visit 91 days after admission

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jan 2013 (estimated)
  2. Study startJun 2013
  3. Primary completionFeb 2015
  4. Study completionMay 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01777412
Sponsor's protocol ID
Avastin_NMO_1
Status last verified
Aug 2015
First submitted
21 Jan 2013
Last updated
21 Aug 2015
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Bevacizumab in people with Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder, run by Johns Hopkins University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 May 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Baseline Expanded Disability Status Score (EDSS) (time frame: Admission to hospital). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2015.

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