Neuromyelitis OpticaNCT03766347
Pediatric NMOSD Observational Study
An observational study of Neuromyelitis Optica and NMO Spectrum Disorder, run by Mayo Clinic at 1 site in United States.
Trial summary
This study is being done to develop a database of pediatric patients in order to study the cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD) in pediatric patients. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Neuromyelitis Optica, NMO Spectrum Disorder
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Feb 2018to 3 Apr 2024
- How long will I take part?
- 1 Year
- Phase
- Observational
- Are there results?
- None posted in the registry
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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Any pediatric patients that have tested positive for AQP4 antibody regardless of geographic location.
Who may be able to join
- AQP4 positive
- Ability to give informed consent by patient or caregiver
Who cannot join
- Inability to complete required forms via phone, mail, or email.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study
This study is being done to develop a database of pediatric patients in order to study the cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD) in pediatric patients.
Read the detailed description
This study is being done to collect information on the natural history of NMOSD in pediatric AQP4-IgG seropositive patients. A major restriction in performing drug studies in pediatric patients with NMOSD is limited information on the course of the disease in these patients. Collecting clinical information over the course of a 1 year observational study would inform on the natural history of the disease in these patients. A repository of pediatric patients with rare diseases can increase knowledge on the natural history of the specific disease, assist in identifying appropriate patients fulfilling specified criteria for drug studies and potentially serve as a control group.
Timepoints: Baseline, 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point).
Baseline data:
- Demographics [age/sex/ethnicity],
- Clinical presentation information including date of initial diagnosis,
- Clinical phenotype
- Immunotherapy used current and past,
- Family history of autoimmune diseases,
- Serological data results
- Radiologic data as available
Self-Report Assessments will be:
- Current impairment, as measured by the expanded disability status scale (EDSS) score self-reported using Ratzker (1997) EDSS Self Report form,
- Quality of life as measured by the EQ-5D and Varni's (1998) PedsQL over the phone/mail/email.
At follow-up visits 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point):
- Attacks/relapses
- Any hospitalizations
- Confirm medications and update records if changes
Self-Report Assessments at follow-up will be:
- Current impairment, as measured by the EDSS score self-reported using Ratzker (1997) EDSS Self Report form,
- Quality of life as measured by the EQ-5D and PedsQL over the phone/mail/email.
- Conditions
- Neuromyelitis Optica, NMO Spectrum Disorder
- Keywords
- Pediatric, Aquaporin-4, AQP4, NMOSD, NMO, Neuromyelitis Optica
- 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 group | Role | What they receive |
|---|---|---|
| Pediatric Neuromyelitis Optica | Participants under the age of 18 that are positive for Aquaporin-4 antibody. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to relapse
Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.
Measured: One year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2018
- Study start1 Feb 2018
- Primary completion3 Apr 2024
- Study completion3 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis OpticaStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 100 participants (actual)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Responsible party
- Principal investigator, Sean Pittock, Mayo Clinic
- Investigators
- Sean J Pittock, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03766347
- Sponsor's protocol ID
- 17-005618
- Status last verified
- Apr 2024
- First submitted
- 4 Dec 2018
- Last updated
- 10 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study
- ClinicalTrials.gov ID
- NCT03766347
- Acronym / sponsor ID
- 17-005618
- Conditions
- Neuromyelitis Optica, NMO Spectrum Disorder
- Keywords
- Pediatric, Aquaporin-4, AQP4, NMOSD, NMO, Neuromyelitis Optica
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- Alexion Pharmaceuticals, Inc.
- Study start
- 1 Feb 2018 (actual)
- Primary completion
- 3 Apr 2024 (actual)
- Study completion
- 3 Apr 2024 (actual)
- First posted
- 6 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being done to develop a database of pediatric patients in order to study the cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD) in pediatric patients.
Detailed description
This study is being done to collect information on the natural history of NMOSD in pediatric AQP4-IgG seropositive patients. A major restriction in performing drug studies in pediatric patients with NMOSD is limited information on the course of the disease in these patients. Collecting clinical information over the course of a 1 year observational study would inform on the natural history of the disease in these patients. A repository of pediatric patients with rare diseases can increase knowledge on the natural history of the specific disease, assist in identifying appropriate patients fulfilling specified criteria for drug studies and potentially serve as a control group.
Timepoints: Baseline, 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point).
Baseline data:
- Demographics [age/sex/ethnicity],
- Clinical presentation information including date of initial diagnosis,
- Clinical phenotype
- Immunotherapy used current and past,
- Family history of autoimmune diseases,
- Serological data results
- Radiologic data as available
Self-Report Assessments will be:
- Current impairment, as measured by the expanded disability status scale (EDSS) score self-reported using Ratzker (1997) EDSS Self Report form,
- Quality of life as measured by the EQ-5D and Varni's (1998) PedsQL over the phone/mail/email.
At follow-up visits 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point):
- Attacks/relapses
- Any hospitalizations
- Confirm medications and update records if changes
Self-Report Assessments at follow-up will be:
- Current impairment, as measured by the EDSS score self-reported using Ratzker (1997) EDSS Self Report form,
- Quality of life as measured by the EQ-5D and PedsQL over the phone/mail/email.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 1 Year
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric Neuromyelitis Optica | – | –Participants under the age of 18 that are positive for Aquaporin-4 antibody. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Any pediatric patients that have tested positive for AQP4 antibody regardless of geographic location.
Key inclusion criteria (2 listed)
- AQP4 positive
- Ability to give informed consent by patient or caregiver
Key exclusion criteria (1 listed)
- Inability to complete required forms via phone, mail, or email.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Time to relapse
Time frame: One year.
Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2024.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sean J Pittock, M.D. principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuromyelitis Optica and NMO Spectrum Disorder, run by Mayo Clinic 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?
The sponsor lists 2 inclusion and 1 exclusion criteria. 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 10 Apr 2024. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Time to relapse (time frame: One year.). 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 10 Apr 2024.