A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder

  • STATUS
    Recruiting
  • End date
    Dec 24, 2024
  • participants needed
    15
  • sponsor
    Alexion Pharmaceuticals
Updated on 24 February 2021
Investigator
Alexion Pharmaceuticals, Email for Complete Site List
Primary Contact
Georgetown University (5.2 mi away) Contact
+28 other location
immunosuppressive agents
immunosuppressive therapy
human chorionic gonadotropin
eculizumab
aquaporin 4

Summary

The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to < 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).

Details
Condition Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders
Treatment Eculizumab
Clinical Study IdentifierNCT04155424
SponsorAlexion Pharmaceuticals
Last Modified on24 February 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age between 2 yrs and 17 yrs?
Gender: Male or Female
Do you have any of these conditions: Neuromyelitis Optica or Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders or Neuromyelitis Optica Spectrum Disorder?
Do you have any of these conditions: Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders or Neuromyelitis Optica or Neuromyelitis Optica Spectrum Disorder?
Do you have any of these conditions: Neuromyelitis Optica Spectrum Disorder or Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders or Neuromyelitis Optica?
Male or female participants aged 2 years to < 18 years with body weight 10 kilograms (kg)
Vaccinated against Neisseria meningitidis within 3 years prior to, or at the time of initiating eculizumab. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics until 2 weeks after the vaccination
Documented vaccination against haemophilus influenzae type b and streptococcus pneumoniae infections at least 2 weeks prior to dosing as per local and country-specific immunization guidelines for the appropriate age group
Anti-aquaporin-4 antibody-positive and diagnosis of NMOSD as defined by the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria
Historical Relapse Rate of at least 2 relapses in the last 2 years, and with at least 1 relapse in the year prior to Screening
EDSS score 7
Participants who enter the study receiving supportive immunosuppressive therapies (ISTs) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration
Female participants of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin) at Screening and follow protocol-specified contraception guidance for avoiding pregnancy while on treatment and for 5 months after the last dose of eculizumab
Male participants with a female spouse/partner of childbearing potential or a pregnant or breastfeeding spouse or partner must agree to use double barrier contraception (male condom plus appropriate barrier method for the female partner) while on treatment and for at least 5 months after the last dose of eculizumab

Exclusion Criteria

Parent or legal guardian is an Alexion employee
Pregnant, breastfeeding, or intending to conceive during the course of the study
Participants known to be human immunodeficiency virus positive or with congenital immunodeficiency
Unresolved meningococcal or other serious infection
Any unresolved acute or chronic systemic bacterial or other infection that is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics
Use of rituximab or other biologicals such as tocilizumab within 6 months prior to Screening
Use of mitoxantrone within 3 months prior to Screening
Use of intravenous immunoglobulin or plasma exchange within 3 weeks prior to Screening
Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening
Has previously received treatment with eculizumab or other complement inhibitors
Clear my responses

How to participate?

Step 1 Connect with a site
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar
Name

Primary Contact

site
Name

Phone Email

0/250
Please verify that you are not a bot.

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note