Autoimmune GFAP Astrocytopathy Clinical Trial
— GFAPOfficial title:
GFAP Auto-immunity : a French Cohort Study
| NCT number | NCT04463550 |
| Other study ID # | GFAP |
| Secondary ID | |
| Status | Recruiting |
| Phase | |
| First received | |
| Last updated | |
| Start date | September 1, 2019 |
| Est. completion date | November 1, 2020 |
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.
| Status | Recruiting |
| Enrollment | 38 |
| Est. completion date | November 1, 2020 |
| Est. primary completion date | January 1, 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | N/A and older |
| Eligibility |
Inclusion Criteria: - 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 Exclusion Criteria: - Patients GFAP-IgG negative in serum and CSF - Absence of complete clinicopathological data - Foreign follow-up |
| Country | Name | City | State |
|---|---|---|---|
| France | Hospices Civils de Lyon | Bron |
| Lead Sponsor | Collaborator |
|---|---|
| Hospices Civils de Lyon |
France,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | demographic data of patients GFAP-IgG positive : phenotype | Report if caucasian (yes or no) retrospectively provided by the treating physicians using a structured questionnaire |
13 months | |
| Other | demographic data of patients GFAP-IgG positive : sex | Report if female sex (yes or no) retrospectively provided by the treating physicians using a structured questionnaire physicians using a structured questionnaire |
13 months | |
| Other | associated conditions of patients GFAP-IgG positive | Report if neoplastic and dysimmune associated diseases and T cell dysregulation condition (yes or no) physicians using a structured questionnaire physicians using a structured questionnaire |
13 months | |
| Other | Report cerebrospinal fluid white cell count | white cell count (/mm3) | 13 months | |
| Other | Report cerebrospinal fluid protein level | protein level (g/L) | 13 months | |
| Other | Report cerebrospinal fluid glucose level | glucose level (mmol/L) | 13 months | |
| Other | Report number of oligoclonal bands in cerebrospinal fluid | number of oligoclonal bands | 13 months | |
| Other | Describe MRI features. | Head and medullar MRI at diagnostic and follow up were reviewed by a neuroradiologist. scale) at each relapse and last follow up. | 13 months | |
| Other | Report other paraclinic findings : electroencephalographic results. | Describe electroencephalographic results at diagnostic and follow up when done . | 13 months | |
| Other | Report other paraclinic findings : electromyographic results. | Describe electromyographic results at diagnostic and follow up when done . | 13 months | |
| Other | Report ophthalmic exam : visual acuity | Describe visual acuity (/10) at diagnostic and follow up when done . | 13 months | |
| Other | Report ophthalmic exam : ocular fundus. | Describe ocular fundus at diagnostic and follow up results when done . | 13 months | |
| Other | Report ophthalmic exam : visual field. | Describe visual field at diagnostic and follow up when done . | 13 months | |
| Other | Report ophthalmic exam : optical coherence tomography. | Describe optical coherence tomography (RNFL thickness) at diagnostic and follow up when done . | 13 months | |
| Other | Report histologic findings. | Describe histologic results at diagnostic and follow up when done . | 13 months | |
| Other | Report treatments and response. | Describe acute and long-term treatments administered and response. | 13 months | |
| Other | Report outcome. | Describe outcome (modified Rankin scale) at each relapse and last follow up. | 13 months | |
| Primary | 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 . |
13 months | |
| Secondary | Describe GFAP-antibody test results. | positivity of GFAP a -IgG in cerebrospinal fluid, in serum and isoform type at diagnostic and follow up. positivity of GFAP a -IgG in cerebrospinal fluid, in serum and isoform type at diagnostic and follow up. |
13 months | |
| Secondary | demographic data of patients GFAP-IgG positive : age at onset | age at onset (years) retrospectively provided by the treating physicians using a structured questionnaire |
13 months |