Autoimmune GFAP AstrocytopathyGFAPNCT04463550
GFAP Auto-immunity : a French Cohort Study
An observational study of Autoimmune GFAP Astrocytopathy, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Glial fibrillary acidic protein (GFAP)-Immunoglobulin G (IgG) have recently been described as a biomarker of a novel inflammatory central nervous system (CNS) disorder, termed autoimmune GFAP astrocytopathy. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Autoimmune GFAP Astrocytopathy
- What is being tested?
- Description and analysis
- How many people
- 38 participants
- Where
- 1 siteFrance
- Study dates
- 1 Sep 2019to 1 Nov 2020
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients GFAP-IgG positive in serum and/or CSF
Who may be able to join
- Positive GFAP-Ab in serum and/or CSF tested by immunohistochemistry on mouse brain slices and confirmed by cell-based assay (CBA) of HEK293 cells expressing GFAP.
- Diagnosis and follow-up in France
- No age limit : from 0 to unlimited age
Who cannot join
- Patients GFAP-IgG negative in serum and CSF
- Absence of complete clinicopathological data
- Foreign follow-up
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Hospices Civils de Lyon
Bron, 69250
Romain MARIGNIER, 04-72-35-75-22, romain.marignier@chu-lyon.fr
Géraldine PICARD, 04 72 35 58 42, geraldine.picard@chu-lyon.fr
About this study
Glial fibrillary acidic protein (GFAP)-Immunoglobulin G (IgG) have recently been described as a biomarker of a novel inflammatory central nervous system (CNS) disorder, termed autoimmune GFAP astrocytopathy. Thus far, four major clinical series have been published (two from Mayo Clinic USA, one from Italy and one from China). GFAP-IgG detected in serum or in cerebrospinal fluid, by tissue-based assay and confirmed by cell-based assay, are associated with encephalitis or meningoencephalitis of acute or subacute onset, less frequently with myelitis or optic disk edema. The characteristic MRI feature is brain linear perivascular radial gadolinium enhancement in the white matter perpendicular to the ventricle, consistent with the immunohistochemical staining pattern of GFAP in rodent brain sections. Approximately 20% of reported cases are associated with a neoplasm (ovarian teratoma mostly). Coexisting neural autoantibodies are described in some patients, N-methyl-D-aspartate (NMDA)-receptor (R)-IgG mostly, followed by aquaporin 4 (AQP4)-IgG. The disease is usually corticosteroid responsive although relapse can occur. In contrast, Chinese patients display poorer outcomes. Pathophysiology is not well understood but the intracellular antigen location makes GFAP-IgG unlikely pathogenic whereas animal models and neuropathologic data suggest a T-cell immune-mediated disorder.
The aim of the investigators is to report the first French cohort of patients GFAP-IgG positive. Investigators retrospectively assessed clinical, immunological and radiological features, treatment response and outcomes.
- Conditions
- Autoimmune GFAP Astrocytopathy
- Being tested
- Description and analysis
- Keywords
- Acidic Protein IgG, astrocytopathy, meningoencephalomyelitis, perivascular-radial parenchymal enhancement, autoimmune, paraneoplasic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Description and analysisOther
Retrospective, non-interventional study, using clinical, biological, radiological and therapeutic data collected during the initial diagnosis and follow-up.
| Study group | Role | What they receive |
|---|---|---|
| Patients GFAP-IgG positive in serum and/or CSF | Patients developing clinical autoimmune encephalitis or meningoencephalomyelitis with anti-GFAP antibodies, managed by the National Reference Center for Paraneoplastic Syndromes and Autoimmune Encephalitis or the National Reference Center for Centre de référence for Neuro-inflammatory diseases of the brain and the spinal cord at the Neurological Hospital of Bron. Receives: Other: Description and analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Report retrospectively clinical data of patients GFAP-IgG positive.
Describe prodromes (yes or no), neurologic signs and clinical course (acute/subacute - yes or
no - or progressive onset - yes or no), if admitted in intensive care units (yes or no), retrospectively provided by
the treating physicians using a structured questionnaire.or progressive onset), if admitted in intensive care units, neoplastic and dysimmune associated diseases and T cell dysregulation condition .
Measured: 13 months
Secondary outcomes (2)
Describe GFAP-antibody test results.
positivity of GFAP α -IgG in cerebrospinal fluid, in serum and isoform type at diagnostic and follow up.
positivity of GFAP α -IgG in cerebrospinal fluid, in serum and isoform type at diagnostic and follow up.
Measured: 13 months
demographic data of patients GFAP-IgG positive : age at onset
age at onset (years) retrospectively provided by the treating
physicians using a structured questionnaire
Measured: 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2020
- Study start1 Sep 2019
- Primary completion1 Jan 2020
- Study completion1 Nov 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune GFAP AstrocytopathyStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Retrospective
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Romain MARIGNIER, Study director, Centre de référence des maladies inflammatoires rares du cerveau et de la moelle, Lyon, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04463550
- Sponsor's protocol ID
- GFAP
- Acronym
- GFAP
- Status last verified
- Jun 2020
- First submitted
- 17 Apr 2020
- Last updated
- 9 Jul 2020
- 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 9 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- GFAP Auto-immunity : a French Cohort Study
- ClinicalTrials.gov ID
- NCT04463550
- Acronym / sponsor ID
- GFAP / GFAP
- Conditions
- Autoimmune GFAP Astrocytopathy
- Keywords
- Acidic Protein IgG, astrocytopathy, meningoencephalomyelitis, perivascular-radial parenchymal enhancement, autoimmune, paraneoplasic
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 1 Sep 2019 (actual)
- Primary completion
- 1 Jan 2020 (actual)
- Study completion
- 1 Nov 2020 (estimated)
- First posted
- 9 Jul 2020 (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.
Glial fibrillary acidic protein (GFAP)-Immunoglobulin G (IgG) have recently been described as a biomarker of a novel inflammatory central nervous system (CNS) disorder, termed autoimmune GFAP astrocytopathy. Thus far, four major clinical series have been published (two from Mayo Clinic USA, one from Italy and one from China). GFAP-IgG detected in serum or in cerebrospinal fluid, by tissue-based assay and confirmed by cell-based assay, are associated with encephalitis or meningoencephalitis of acute or subacute onset, less frequently with myelitis or optic disk edema. The characteristic MRI feature is brain linear perivascular radial gadolinium enhancement in the white matter perpendicular to the ventricle, consistent with the immunohistochemical staining pattern of GFAP in rodent brain sections. Approximately 20% of reported cases are associated with a neoplasm (ovarian teratoma mostly). Coexisting neural autoantibodies are described in some patients, N-methyl-D-aspartate (NMDA)-receptor (R)-IgG mostly, followed by aquaporin 4 (AQP4)-IgG. The disease is usually corticosteroid responsive although relapse can occur. In contrast, Chinese patients display poorer outcomes. Pathophysiology is not well understood but the intracellular antigen location makes GFAP-IgG unlikely pathogenic whereas animal models and neuropathologic data suggest a T-cell immune-mediated disorder.
The aim of the investigators is to report the first French cohort of patients GFAP-IgG positive. Investigators retrospectively assessed clinical, immunological and radiological features, treatment response and outcomes.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients GFAP-IgG positive in serum and/or CSF | – | Other: Description and analysisPatients developing clinical autoimmune encephalitis or meningoencephalomyelitis with anti-GFAP antibodies, managed by the National Reference Center for Paraneoplastic Syndromes and Autoimmune Encephalitis or the National Reference Center for Centre de référence for Neuro-inflammatory diseases of the brain and the spinal cord at the Neurological Hospital of Bron. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients GFAP-IgG positive in serum and/or CSF
Key inclusion criteria (3 listed)
- Positive GFAP-Ab in serum and/or CSF tested by immunohistochemistry on mouse brain slices and confirmed by cell-based assay (CBA) of HEK293 cells expressing GFAP.
- Diagnosis and follow-up in France
- No age limit : from 0 to unlimited age
Key exclusion criteria (3 listed)
- Patients GFAP-IgG negative in serum and CSF
- Absence of complete clinicopathological data
- Foreign follow-up
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Description and analysisRetrospective, non-interventional study, using clinical, biological, radiological and therapeutic data collected during the initial diagnosis and follow-up. | Other | Patients GFAP-IgG positive in serum and/or CSF |
Primary outcome
Report retrospectively clinical data of patients GFAP-IgG positive.
Time frame: 13 months
Describe prodromes (yes or no), neurologic signs and clinical course (acute/subacute - yes or no - or progressive onset - yes or no), if admitted in intensive care units (yes or no), retrospectively provided by the treating physicians using a structured questionnaire.or progressive onset), if admitted in intensive care units, neoplastic and dysimmune associated diseases and T cell dysregulation condition .
2 secondary outcomes
Describe GFAP-antibody test results.
Time frame: 13 months
demographic data of patients GFAP-IgG positive : age at onset
Time frame: 13 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2020.
- Individual participant dataSharing planned: No
Related trials for Autoimmune GFAP Astrocytopathy
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China National Registry of Neuro-Inflammatory Diseases
Multiple Sclerosis, NMO Spectrum Disorder
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.
Romain MARIGNIER study director
Centre de référence des maladies inflammatoires rares du cerveau et de la moelle, Lyon, France
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 Autoimmune GFAP Astrocytopathy, run by Hospices Civils de Lyon at 1 site in France.
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 3 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 9 Jul 2020. 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 France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Romain MARIGNIER, Dr (04-72-35-75-22, romain.marignier@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Report retrospectively clinical data of patients GFAP-IgG positive. (time frame: 13 months). 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 9 Jul 2020.